SEC Filing - Regenxbio Inc

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Amendment No.2 to Form S-1

As filed with the Securities and Exchange Commission on September 15, 2015.

Registration No. 333-206430

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

Amendment No. 2

FORM S-1

REGISTRATION STATEMENT

Under

THE SECURITIES ACT OF 1933

REGENXBIO INC.

(Exact Name of Registrant as Specified in its Charter)

Delaware

2836

47-1851754

(State or Other Jurisdiction of

Incorporation or Organization)

(Primary Standard Industrial

Classification Code Number)

(I.R.S. Employer

Identification Number)

9712 Medical Center Drive, Suite 100

Rockville, MD 20850

(240) 552-8181

(Address, including zip code and telephone number, including area code, of registrant’s principal executive offices)

Kenneth T. Mills

Chief Executive Officer

REGENXBIO Inc.

9712 Medical Center Drive, Suite 100

Rockville, MD 20850

(240) 552-8181

(Name, address, including zip code and telephone number, including area code, of agent for service)

Copies to:

Jay K. Hachigian, Esq.

Richard R. Hesp, Esq.

Keith J. Scherer, Esq.

Albert W. Vanderlaan, Esq.

Gunderson Dettmer Stough

Villeneuve Franklin & Hachigian, LLP

One Marina Park Drive, Suite 900

Boston, MA 02210

Telephone: (617) 648-9100

Telecopy: (617) 648-9199

Sara Garon Berl, Esq.

Vice President, General Counsel and Secretary

REGENXBIO Inc.

9712 Medical Center Drive, Suite 100

Rockville, MD 20850

Telephone: (240) 552-8181

Richard D. Truesdell, Jr., Esq.

Sophia Hudson, Esq.

Davis Polk & Wardwell LLP

450 Lexington Avenue

New York, NY 10017

Telephone: (212) 450-4000

Approximate date of commencement of proposed sale to the public: As soon as practicable after the effective date of this Registration Statement.

If any of the securities being registered on this Form are to be offered on a delayed or continuous basis pursuant to Rule 415 under the Securities Act of 1933, check the following box. ¨

If this Form is filed to register additional securities for an offering pursuant to Rule 462(b) under the Securities Act, please check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering. ¨

If this Form is a post-effective amendment filed pursuant to Rule 462(c) under the Securities Act, check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering. ¨

If this Form is a post-effective amendment filed pursuant to Rule 462(d) under the Securities Act, check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering. ¨

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting company. See the definitions of “large accelerated filer,” “accelerated filer” and “smaller reporting company” in Rule 12b-2 of the Exchange Act. (Check one):


Large accelerated filer	¨	Accelerated filer	¨
Non-accelerated filer	x (Do not check if a smaller reporting company)	Smaller reporting company	¨

The Registrant hereby amends this Registration Statement on such date or dates as may be necessary to delay its effective date until the Registrant shall file a further amendment which specifically states that this Registration Statement shall thereafter become effective in accordance with Section 8(a) of the Securities Act of 1933 or until the Registration Statement shall become effective on such date as the Commission, acting pursuant to such Section 8(a), may determine.

Explanatory Note

The sole purpose of this Amendment No. 2 to the Registration Statement on Form S-1 (Registration No. 333-206430), as amended, is to amend Exhibits 10.3, 10.9, 10.11, 10.12, 10.14, 10.15, 10.16, 10.17, 10.18, 10.19, 10.21, 10.22, 10.23, 10.24, 10.25 and 10.27. Accordingly, this Amendment No. 2 consists only of the facing page, this explanatory note, and Part II of the Registration Statement. The Prospectus, constituting Part I of the Registration Statement, is unchanged and has therefore been omitted.

PART II

INFORMATION NOT REQUIRED IN THE PROSPECTUS

Item 13. Other Expenses of Issuance and Distribution.

The following table presents the costs and expenses, other than underwriting discounts and commissions, payable in connection with the sale of common stock being registered. All amounts are estimates except the SEC registration fee, the FINRA filing fee, and the NASDAQ listing fee. Except as otherwise noted, all the expenses below will be paid by us.


SEC registration fee	$	14,106
FINRA filing fee		18,708
NASDAQ Listing fee		225,000
Printing and engraving expenses		395,000
Legal fees and expenses		2,200,000
Accounting fees and expenses		330,000
Blue sky fees and expenses		5,000
Transfer agent fees		10,000
Miscellaneous fees and expenses		102,186

Total	$	3,300,000

Item 14. Indemnification of Directors and Officers.

In connection with the completion of this offering, the Registrant’s restated certificate of incorporation will contain provisions that eliminate, to the maximum extent permitted by the General Corporation Law of the State of Delaware, the personal liability of the Registrant’s directors for monetary damages for breach of their fiduciary duties as directors. The Registrant’s amended and restated bylaws to be in effect immediately prior to the completion of this offering provide that the Registrant must indemnify its directors and officers and may indemnify its employees and other agents to the fullest extent permitted by the General Corporation Law of the State of Delaware.

Sections 145 and 102(b)(7) of the General Corporation Law of the State of Delaware provide that a corporation may indemnify any person made a party to an action by reason of the fact that he or she was a director, officer, employee or agent of the corporation or is or was serving at the request of a corporation against expenses (including attorneys’ fees), judgments, fines and amounts paid in settlement actually and reasonably incurred by him or her in connection with such action if he or she acted in good faith and in a manner he or she reasonably believed to be in, or not opposed to, the best interests of the corporation and, with respect to any criminal action or proceeding, had no reasonable cause to believe his or her conduct was unlawful, except that, in the case of an action by or in right of the corporation, no indemnification may generally be made in respect of any claim as to which such person is adjudged to be liable to the corporation.

The Registrant has entered into indemnification agreements with its directors and executive officers, in addition to the indemnification provided for in its amended and restated bylaws, and intends to enter into indemnification agreements with any new directors and executive officers in the future.

The Registrant has purchased and intends to maintain insurance on behalf of any person who is or was a director or officer of the Registrant against any loss arising from any claim asserted against him or her and incurred by him or her in any such capacity, subject to certain exclusions.

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The Underwriting Agreement, the form of which is attached as Exhibit 1.1 hereto, provides for indemnification by the underwriters of the Registrant and its executive officers and directors, and by the Registrant of the underwriters, for certain liabilities, including liabilities arising under the Securities Act and affords certain rights of contribution with respect thereto.

See also “Undertakings” set out in response to Item 17 herein.

Item 15. Recent Sales of Unregistered Securities.

Set forth below is information regarding the shares of common stock and preferred stock issued, and options granted, by us since September 15, 2012 that were not registered under the Securities Act of 1933.

(1)

Under the ReGenX Biosciences, LLC (our predecessor entity) 2009 Equity Plan, we granted an aggregate of 6,420,000 of Class B Units at a distribution threshold of $0.02382 per unit as profits interests to certain of our employees.

(2)

In October 2013, we issued and sold an aggregate of 95,314,803 Series B Preferred Units (pre-Conversion) to investors for an aggregate purchase price of approximately $7.9 million.

(3)

In September 2014, we converted from a Delaware limited liability company named ReGenX Biosciences, LLC (the LLC) to a Delaware corporation named REGENXBIO Inc. (the Conversion). Pursuant to the Conversion, we issued (i) 2,642,963 shares of common stock upon the conversion of 132,148,224 Class A units of the LLC, (ii) 1,906,295 shares of Series A Preferred Stock upon the conversion of 119,656,372 Series A Preferred units of the LLC and (iii) 2,393,127 shares of Series B Preferred Stock upon the conversion of 95,314,803 shares of Series B Preferred units of the LLC.

(4)

Under our 2014 Stock Plan, (i) from September 2014 to November 2014, we granted stock options to purchase an aggregate of 2,132,400 shares of our common stock at an exercise price of $0.85 per share to certain of our employees, officers, consultants and advisors and (ii) in May 2015, we granted stock options to purchase an aggregate of 1,063,900 shares of our common stock at an exercise price of $3.76 per share to certain of our employees, officers, consultants and advisors.

(5)

In January 2015, we issued and sold an aggregate of 4,631,774 shares of Series C convertible preferred stock to investors for an aggregate purchase price of $30.0 million.

(6)

In May 2015, we issued and sold an aggregate of 7,366,849 shares of Series D convertible preferred stock to investors for an aggregate purchase price of $70.5 million.

(7)

In October 2014, an advisor exercised an option to purchase 1,900 shares of our common stock.

(8)

In March 2015, a former employee exercised an option to purchase 7,800 shares of our common stock.

(9)

In May 2015, an employee exercised an option to purchase 100,000 shares of our common stock.

(10)

In July 2015, an employee exercised an option to purchase 10,150 shares of our common stock.

The offers, sales, grants and issuances of the securities described in paragraphs (1), (7), (8), (9) and (10) were deemed to be exempt from registration under the Securities Act in reliance on Rule 701. The recipients of such securities were our employees, officers, bona fide consultants and advisors and received the securities under the ReGenX Biosciences, LLC 2009 Equity Plan. Appropriate legends were affixed to the securities issued in these transactions. Each of the recipients of securities in these transactions had adequate access, through employment, business or other relationships, to information about us.

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The offer, sale, and issuance of the securities described in paragraphs (2), (3), (5) and (6) were deemed to be exempt from registration under the Securities Act in reliance on Section 4(a)(2) of the Securities Act in that the issuance of the securities to the accredited investors did not involve a public offering. The recipients of the securities in these transactions acquired the securities for investment only and not with a view to or for sale in connection with any distribution thereof, and appropriate legends were affixed to the securities issued in these transactions. The recipients of the securities in these transactions were accredited investors under Rule 501 of Regulation D.

The offers, sales, grants and issuances of the securities described in paragraph (4) were deemed to be exempt from registration under the Securities Act in reliance on Rule 701. The recipients of such securities were our employees, officers, bona fide consultants and advisors and received the securities under our 2014 Stock Plan. Appropriate legends were affixed to the securities issued in these transactions. Each of the recipients of securities in these transactions had adequate access, through employment, business or other relationships, to information about us.

Item 16. Exhibits and Financial Statement Schedules.

(a) Exhibits

The exhibits to the registration statement are listed in the Exhibit Index attached hereto and incorporated by reference herein.

(b) Financial Statement Schedules

Schedules not listed above have been omitted because the information required to be set forth therein is not applicable or is shown in the financial statements or notes thereto.

Item 17. Undertakings.

Insofar as indemnification for liabilities arising under the Securities Act of 1933 may be permitted to directors, officers and controlling persons of the registrant pursuant to the foregoing provisions, or otherwise, the registrant has been advised that, in the opinion of the SEC, such indemnification is against public policy as expressed in the Securities Act of 1933, and is, therefore, unenforceable. In the event that a claim for indemnification against such liabilities (other than the payment by the registrant of expenses incurred or paid by a director, officer or controlling person of the registrant in the successful defense of any action, suit or proceeding) is asserted by such director, officer or controlling person in connection with the securities being registered, the registrant will, unless in the opinion of its counsel the matter has been settled by controlling precedent, submit to a court of appropriate jurisdiction the question whether such indemnification by it is against public policy as expressed in the Securities Act of 1933, and will be governed by the final adjudication of such issue.

The undersigned Registrant hereby undertakes to provide the underwriters, at the closing specified in the underwriting agreement, certificates in such denominations and registered in such names as required by the underwriters to permit prompt delivery to each purchaser.

The undersigned registrant hereby undertakes that:

(1)

For purposes of determining any liability under the Securities Act of 1933, the information omitted from a form of prospectus filed as part of this registration statement in reliance upon Rule 430A and contained in the form of prospectus filed by the Registrant pursuant to Rule 424(b)(1) or (4) or 497(h) under the Securities Act of 1933 shall be deemed to be part of this registration statement as of the time it was declared effective.

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(2)

For the purpose of determining any liability under the Securities Act of 1933, each post-effective amendment that contains a form of prospectus shall be deemed to be a new registration statement relating to the securities offered therein, and the offering of such securities at that time shall be deemed to be the initial bona fide offering thereof.

(3)

For the purpose of determining liability under the Securities Act to any purchaser, each prospectus filed pursuant to Rule 424(b) as part of a registration statement relating to an offering, other than registration statements relying on Rule 430B or other than prospectuses filed in reliance on Rule 430A, shall be deemed to be part of and included in the registration statement as of the date it is first used after effectiveness. Provided, however, that no statement made in a registration statement or prospectus that is part of the registration statement or made in a document incorporated or deemed incorporated by reference into the registration statement or prospectus that is part of the registration statement will, as to a purchaser with a time of contract of sale prior to such first use, supersede or modify any statement that was made in the registration statement or prospectus that was part of the registration statement or made in any such document immediately prior to such date of first use.

(4)

In a primary offering of securities of the undersigned registrant pursuant to this registration statement, regardless of the underwriting method used to sell the securities to the purchaser, if the securities are offered or sold to such purchaser by means of any of the following communications, the undersigned registrant will be a seller to the purchaser and will be considered to offer or sell such securities to such purchaser:

(i)

Any preliminary prospectus or prospectus of the undersigned registrant relating to the offering required to be filed pursuant to Rule 424;

(ii)

Any free writing prospectus relating to the offering prepared by or on behalf of the undersigned registrant or used or referred to by the undersigned registrant;

(iii)

The portion of any other free writing prospectus relating to the offering containing material information about the undersigned registrant or its securities provided by or on behalf of the undersigned registrant; and

(iv)

Any other communication that is an offer in the offering made by the undersigned registrant to the purchaser.

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SIGNATURES

Pursuant to the requirements of the Securities Act of 1933, the registrant has duly caused this Registration Statement to be signed on its behalf by the undersigned, thereunto duly authorized, in the City of Rockville, State of Maryland, on this 15^th day of September, 2015.


REGENXBIO INC.

By:		/s/ Kenneth T. Mills
		Kenneth T. Mills
		President and Chief Executive Officer

Pursuant to the requirements of the Securities Act of 1933, as amended, this Registration Statement has been signed by the following persons in the capacities and on the dates indicated:


Signature	Title	Date

/s/ Kenneth T. Mills Kenneth T. Mills	Chief Executive Officer, President and Director (Principal Executive Officer)	September 15, 2015

/s/ Vittal Vasista Vittal Vasista	Chief Financial Officer (Principal Financial and Accounting Officer)	September 15, 2015

* Donald J. Hayden, Jr.	Chairman of the Board of Directors	September 15, 2015

* Luke M. Beshar	Director	September 15, 2015

* Edgar G. Engleman, M.D.	Director	September 15, 2015

* Allan M. Fox	Director	September 15, 2015

* A.N. “Jerry” Karabelas, Ph.D.	Director	September 15, 2015

* Camille Samuels	Director	September 15, 2015

*By:

/s/ Kenneth T. Mills

Kenneth T. Mills

Attorney-in-Fact

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EXHIBIT INDEX


Exhibit		Description

1.1‡		Form of Underwriting Agreement

3.1‡		Restated Certificate of Incorporation, as amended (currently in effect)

3.2‡		Bylaws (currently in effect)

3.3‡		Form of Restated Certificate of Incorporation (to be effective immediately prior to the closing of this offering)

3.4‡		Form of Amended and Restated Bylaws (to be effective immediately prior to the closing of this offering)

4.1‡		Specimen stock certificate evidencing the shares of common stock

4.2‡		Amended and Restated Investors’ Rights Agreement dated as of May 15, 2015

5.1‡		Opinion of Gunderson Dettmer Stough Villeneuve Franklin & Hachigian, LLP

10.1‡		Form of Indemnity Agreement for directors and officers

10.2+‡		2014 Stock Plan, as amended

10.3+		2015 Equity Incentive Plan and form of option agreement thereunder

10.4+‡		2015 Employee Stock Purchase Plan

10.5+‡		Employment Agreement effective as of June 30, 2015 between the Registrant and Kenneth T. Mills.

10.6+‡		Employment Agreement effective as of June 30, 2015 between the Registrant and Stephen Yoo, M.D.

10.7+‡		Employment Agreement effective as of June 30, 2015 between the Registrant and Vittal Vasista

10.8+‡		Independent Director Compensation Policy

10.9†		License Agreement effective February 24, 2009 between the Registrant and The Trustees of the University of Pennsylvania

10.10†‡		First Amendment to License Agreement dated March 6, 2009 between the Registrant and The Trustees of the University of Pennsylvania

10.11†		Second Amendment to License Agreement effective September 9, 2014 between the Registrant and The Trustees of the University of Pennsylvania

10.12†		License Agreement dated March 6, 2009 between the Registrant and SmithKline Beecham Corporation d/b/a GlaxoSmithKline

10.13‡		Amendment to License Agreement dated April 15, 2009 between the Registrant and SmithKline Beecham Corporation d/b/a GlaxoSmithKline

10.14†		License Agreement dated April 10, 2014 between the Registrant and AAVLife

10.15†		License Agreement dated July 9, 2013 between the Registrant and Audentes Therapeutics, Inc.

10.16†		License Agreement dated March 21, 2014 between the Registrant and AveXis, Inc.

10.17†		License Agreement dated November 22, 2010 between the Registrant and Baxalta US Inc. (as assignee of Baxter Healthcare Corporation, as assignee of Chatham Therapeutics, LLC)

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Exhibit		Description

10.18†		License Agreement dated October 30, 2013 between the Registrant and Dimension Therapeutics, Inc.

10.19†		First Amendment to License Agreement dated June 18, 2014 between the Registrant and Dimension Therapeutics, Inc.

10.20†‡		Second Amendment to License Agreement dated September 29, 2014 between the Registrant and Dimension Therapeutics, Inc.

10.21†		Option and License Agreement dated March 10, 2015 between the Registrant and Dimension Therapeutics, Inc.

10.22†		License Agreement dated March 5, 2014 between the Registrant and Laboratorios Del Dr. Esteve, S.A.

10.23†		License Agreement dated December 2, 2013 between the Registrant and Lysogene Société par Actions Simplifiée

10.24†		License Agreement dated May 28, 2014 between the Registrant and Voyager Therapeutics, Inc.

10.25†		Exclusive Patent License Agreement dated November 10, 2014 between the Registrant and the Regents of the University of Minnesota

10.26‡		Lease dated March 6, 2015 between the Registrant and BMR-Medical Center Drive LLC

10.27†		Development, Manufacturing, and Testing Standard Terms and Conditions dated April 3, 2015 between the Registrant and WuXi AppTec, Inc.

10.28†‡		Cooperation Agreement dated May 28, 2015 between the Registrant and WuXi AppTec, Inc.

10.29+‡		REGENXBIO Inc. Management Cash Incentive Plan

10.30‡		Board of Managers Agreement dated February 6, 2013 between the Registrant and Donald J. Hayden, Jr.

16.1‡		Letter from Baker Tilly Virchow Krause, LLP addressed to the SEC provided in connection with the change in independent accountant

23.1‡		Consent of PricewaterhouseCoopers LLP, independent registered public accounting firm

23.2‡		Consent of Gunderson Dettmer Stough Villeneuve Franklin & Hachigian, LLP (included in Exhibit 5.1)

24.1‡		Power of Attorney (included on signature page)

+	Indicates management contract or compensatory plan.

†	Portions of this exhibit (indicated by asterisks) have been omitted pursuant to a request for confidential treatment. The omitted portions of this exhibit have been filed with the SEC.

‡	Previously filed.

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EX-10.3

Exhibit 10.3

REGENXBIO INC.

2015 EQUITY INCENTIVE PLAN

(AS ADOPTED ON JUNE 17, 2015)

REGENXBIO INC.

2015 EQUITY INCENTIVE PLAN

ARTICLE 1. INTRODUCTION.

The Board adopted the Plan to become effective immediately, although no Awards may be granted prior to the Registration Date. The purpose of the Plan is to promote the long-term success of the Company and the creation of stockholder value by (a) encouraging Service Providers to focus on critical long-range corporate objectives, (b) encouraging the attraction and retention of Service Providers with exceptional qualifications and (c) linking Service Providers directly to stockholder interests through increased stock ownership. The Plan seeks to achieve this purpose by providing for Awards in the form of Options (which may constitute ISOs or NSOs), SARs, Restricted Shares, Stock Units and Performance Cash Awards.

ARTICLE 2. ADMINISTRATION.

2.1 General. The Plan may be administered by the Board or one or more Committees. Each Committee shall have the authority and be responsible for such functions as have been assigned to it.

2.2 Section 162(m). To the extent an Award is intended to qualify as “performance-based compensation” within the meaning of Code Section 162(m), the Plan will be administered by a Committee of two or more “outside directors” within the meaning of Code Section 162(m).

2.3 Section 16. To the extent desirable to qualify transactions hereunder as exempt under Exchange Act Rule 16b-3, the transactions contemplated hereunder will be approved by the entire Board or a Committee of two or more “non-employee directors” within the meaning of Exchange Act Rule 16b-3.

2.4 Powers of Administrator. Subject to the terms of the Plan, and in the case of a Committee, subject to the specific duties delegated to the Committee, the Administrator shall have the authority to (a) select the Service Providers who are to receive Awards under the Plan, (b) determine the type, number, vesting requirements and other features and conditions of such Awards, (c) determine whether and to what extent any Performance Goals have been attained, (d) interpret the Plan and Awards granted under the Plan, (e) make, amend and rescind rules relating to the Plan and Awards granted under the Plan, including rules relating to sub-plans established for the purposes of satisfying applicable foreign laws or for qualifying for favorable tax treatment under applicable foreign laws, (f) impose such restrictions, conditions or limitations as it determines appropriate as to the timing and manner of any resales by a Participant of any Common Shares issued pursuant to an Award, including restrictions under an insider trading policy and restrictions as to the use of a specified brokerage firm for such resales, and (g) make all other decisions relating to the operation of the Plan and Awards granted under the Plan.

2.5 Effect of Administrator’s Decisions. The Administrator’s decisions, determinations and interpretations shall be final and binding on all Participants and any other holders of Awards.

2.6 Governing Law. The Plan shall be governed by, and construed in accordance with, the laws of the State of Delaware (except its choice-of-law provisions).

ARTICLE 3. SHARES AVAILABLE FOR GRANTS.

3.1 Basic Limitation. Common Shares issued pursuant to the Plan may be authorized but unissued shares or treasury shares. The aggregate number of Common Shares issued under the Plan shall not exceed the sum of (a) 2,025,000 shares (subject to adjustment pursuant to a stock split to be effected prior to the IPO Date), (b) the number of Common Shares reserved under the Predecessor Plan that are not issued or subject to outstanding awards under the Predecessor Plan on the Registration Date, (c) any Common Shares subject to outstanding options under the Predecessor Plan on the Registration Date that subsequently expire or lapse unexercised and Common Shares issued pursuant to awards granted under the Predecessor Plans that are outstanding on the Registration Date and that are subsequently forfeited to or repurchased by the Company and (d) the additional Common Shares described in Articles 3.2 and 3.3; provided, however, that no more than 6,015,300 Common Shares (subject to adjustment pursuant to a stock split to be effected prior to the IPO Date), in the aggregate, shall be added to the Plan pursuant to clauses (b) and (c). The number of Common Shares that are subject to Stock Awards outstanding at any time under the Plan may not exceed the number of Common Shares that then remain available for issuance under the Plan. The numerical limitations in this Article 3.1 shall be subject to adjustment pursuant to Article 9.

3.2 Annual Increase in Shares. As of the first business day of each fiscal year of the Company during the term of the Plan, commencing on January 1, 2016, the aggregate number of Common Shares that may be issued under the Plan shall automatically increase by a number equal to the least of (a) 4% of the total number of Common Shares outstanding on December 31 of the prior year, or (b) a number of Common Shares determined by the Board.

3.3 Shares Returned to Reserve. To the extent that Options, SARs or Stock Units granted under this Plan are forfeited or expire for any other reason before being exercised or settled in full, the Common Shares subject to such Options, SARs or Stock Units shall again become available for issuance under the Plan. If SARs are exercised, then only the number of Common Shares (if any) actually issued to the Participant in settlement of such SARs shall reduce the number available under Article 3.1 and the balance shall again become available for issuance under the Plan. If Stock Units are settled, then only the number of Common Shares (if any) actually issued to the Participant in settlement of such Stock Units shall reduce the number available under Article 3.1 and the balance shall again become available for issuance under the Plan. If Restricted Shares or Common Shares issued upon the exercise of Options or otherwise under the Plan are reacquired by the Company pursuant to a forfeiture provision, repurchase right or for any other reason prior to the shares having become vested, then such Common Shares shall again become available for issuance under the Plan. Common Shares applied to pay

the Exercise Price of Options or to satisfy tax withholding obligations related to any Award shall again become available for issuance under the Plan. To the extent that an Award is settled in cash rather than Common Shares, the cash settlement shall not reduce the number of Shares available for issuance under the Plan.

3.4 Awards Not Reducing Share Reserve in Article 3.1. Any dividend equivalents paid or credited under the Plan with respect to Stock Units shall not be applied against the number of Common Shares that may be issued under the Plan, whether or not such dividend equivalents are converted into Stock Units. In addition, Common Shares subject to Substitute Awards granted by the Company shall not reduce the number of Common Shares that may be issued under Article 3.1, nor shall shares subject to Substitute Awards again be available for Awards under the Plan in the event of any forfeiture, expiration or cash settlement of such Substitute Awards.

3.5 Share Limits. Subject to adjustment in accordance with Article 9:

(a) The aggregate number of Common Shares subject to Options and SARs that may be granted under this Plan during any calendar year to any one Participant shall not exceed 1,500,000 Common Shares (subject to adjustment pursuant to a stock split to be effected prior to the IPO Date), except that the Company may grant to a new Employee in the calendar year in which his or her Service as an Employee first commences Options and/or SARs that cover (in the aggregate) up to an additional 500,000 Common Shares (subject to adjustment pursuant to a stock split to be effected prior to the IPO Date);

(b) The aggregate number of Common Shares subject to Restricted Share awards and Stock Units that may be granted under this Plan during any calendar year to any one Participant shall not exceed 1,500,000 Shares (subject to adjustment pursuant to a stock split to be effected prior to the IPO Date), except that the Company may grant to a new Employee in the calendar year in which his or her Service as an Employee first commences Restricted Share awards and Stock Units that cover (in the aggregate) up to an additional 500,000 Common Shares (subject to adjustment pursuant to a stock split to be effected prior to the IPO Date);

(c) No Participant shall be paid more than $1 million in cash in any calendar year pursuant to Performance Cash Awards granted under the Plan;

(d) No more than 6,015,300 Common Shares (subject to adjustment pursuant to a stock split to be effected prior to the IPO Date) plus the additional Common Shares described in Article 3.2 may be issued under the Plan upon the exercise of ISOs; and

(e) The maximum aggregate grant date fair value (as determined in accordance with Financial Accounting Standards Board Accounting Standards Codification Topic 718, or any successor thereto) of Options and SARs that may be granted under this Plan to an Outside Director as compensation for services as an Outside Director during a calendar year shall not exceed $500,000.

ARTICLE 4. ELIGIBILITY.

4.1 Incentive Stock Options. Only Employees who are common-law employees of the Company, a Parent or a Subsidiary shall be eligible for the grant of ISOs. In addition, an Employee who owns more than 10% of the total combined voting power of all classes of outstanding stock of the Company or any of its Parents or Subsidiaries shall not be eligible for the grant of an ISO unless the additional requirements set forth in Code Section 422(c)(5) are satisfied.

4.2 Other Awards. Awards other than ISOs may only be granted to Service Providers.

ARTICLE 5. OPTIONS.

5.1 Stock Option Agreement. Each grant of an Option under the Plan shall be evidenced by a Stock Option Agreement between the Optionee and the Company. Such Option shall be subject to all applicable terms of the Plan and may be subject to any other terms that are not inconsistent with the Plan. The Stock Option Agreement shall specify whether the Option is intended to be an ISO or an NSO. The provisions of the various Stock Option Agreements entered into under the Plan need not be identical.

5.2 Number of Shares. Each Stock Option Agreement shall specify the number of Common Shares subject to the Option, which number shall adjust in accordance with Article 9.

5.3 Exercise Price. Each Stock Option Agreement shall specify the Exercise Price, which shall not be less than 100% of the Fair Market Value of a Common Share on the date of grant. The preceding sentence shall not apply to an Option that is a Substitute Award granted in a manner that would satisfy the requirements of Code Section 409A and, if applicable, Code Section 424(a).

5.4 Exercisability and Term. Each Stock Option Agreement shall specify the date or event when all or any installment of the Option is to become vested and/or exercisable. The Stock Option Agreement shall also specify the term of the Option; provided that, except to the extent necessary to comply with applicable foreign law, the term of an Option shall in no event exceed 10 years from the date of grant. A Stock Option Agreement may provide for accelerated vesting and/or exercisability upon certain specified events and may provide for expiration prior to the end of its term in the event of the termination of the Optionee’s Service.

5.5 Death of Optionee. After an Optionee’s death, any vested and exercisable Options held by such Optionee may be exercised by his or her beneficiary or beneficiaries. Each Optionee may designate one or more beneficiaries for this purpose by filing the prescribed form with the Company. A beneficiary designation may be changed by filing the prescribed form with the Company at any time before the Optionee’s death. If no beneficiary was designated or if no designated beneficiary survives the Optionee, then any vested and exercisable Options held by the Optionee may be exercised by his or her estate.

5.6 Modification or Assumption of Options. Within the limitations of the Plan, the Administrator may modify, reprice, extend or assume outstanding options or may accept the cancellation of outstanding options (whether granted by the Company or by another issuer) in return for the grant of new Options for the same or a different number of shares and at the same or a different exercise price or in return for the grant of a different type of Award. The foregoing notwithstanding, no modification of an Option shall, without the consent of the Optionee, impair his or her rights or obligations under such Option.

5.7 Buyout Provisions. The Administrator may at any time (a) offer to buy out for a payment in cash or cash equivalents an Option previously granted or (b) authorize an Optionee to elect to cash out an Option previously granted, in either case at such time and based upon such terms and conditions as the Administrator shall establish.

5.8 Payment for Option Shares. The entire Exercise Price of Common Shares issued upon exercise of Options shall be payable in cash or cash equivalents at the time when such Common Shares are purchased. In addition, the Administrator may, in its sole discretion and to the extent permitted by applicable law, accept payment of all or a portion of the Exercise Price through any one or a combination of the following forms or methods:

(a) Subject to any conditions or limitations established by the Administrator, by surrendering Common Shares that are already owned by the Optionee with a Fair Market Value on the date of surrender equal to the aggregate exercise price of the Common Shares as to which such Option will be exercised;

(b) By delivering (on a form prescribed by the Company) an irrevocable direction to a securities broker approved by the Company to sell all or part of the Common Shares being purchased under the Plan and to deliver all or part of the sales proceeds to the Company;

(c) Subject to such conditions and requirements as the Administrator may impose from time to time, through a net exercise procedure; or

(d) Through any other form or method consistent with applicable laws, regulations and rules.

ARTICLE 6. STOCK APPRECIATION RIGHTS.

6.1 SAR Agreement. Each grant of a SAR under the Plan shall be evidenced by a SAR Agreement between the Optionee and the Company. Such SAR shall be subject to all applicable terms of the Plan and may be subject to any other terms that are not inconsistent with the Plan. The provisions of the various SAR Agreements entered into under the Plan need not be identical.

6.2 Number of Shares. Each SAR Agreement shall specify the number of Common Shares to which the SAR pertains, which number shall adjust in accordance with Article 9.

6.3 Exercise Price. Each SAR Agreement shall specify the Exercise Price, which shall in no event be less than 100% of the Fair Market Value of a Common Share on the date of grant. The preceding sentence shall not apply to a SAR that is a Substitute Award granted in a manner that would satisfy the requirements of Code Section 409A.

6.4 Exercisability and Term. Each SAR Agreement shall specify the date when all or any installment of the SAR is to become vested and exercisable. The SAR Agreement shall also specify the term of the SAR; provided that except to the extent necessary to comply with applicable foreign law, the term of a SAR shall not exceed 10 years from the date of grant. A SAR Agreement may provide for accelerated vesting and exercisability upon certain specified events and may provide for expiration prior to the end of its term in the event of the termination of the Optionee’s Service.

6.5 Exercise of SARs. Upon exercise of a SAR, the Optionee (or any person having the right to exercise the SAR after his or her death) shall receive from the Company (a) Common Shares, (b) cash or (c) a combination of Common Shares and cash, as the Administrator shall determine. The amount of cash and/or the Fair Market Value of Common Shares received upon exercise of SARs shall, in the aggregate, not exceed the amount by which the Fair Market Value (on the date of surrender) of the Common Shares subject to the SARs exceeds the Exercise Price. If, on the date when a SAR expires, the Exercise Price is less than the Fair Market Value on such date but any portion of such SAR has not been exercised or surrendered, then such SAR shall automatically be deemed to be exercised as of such date with respect to such portion. A SAR Agreement may also provide for an automatic exercise of the SAR on an earlier date.

6.6 Death of Optionee. After an Optionee’s death, any vested and exercisable SARs held by such Optionee may be exercised by his or her beneficiary or beneficiaries. Each Optionee may designate one or more beneficiaries for this purpose by filing the prescribed form with the Company. A beneficiary designation may be changed by filing the prescribed form with the Company at any time before the Optionee’s death. If no beneficiary was designated or if no designated beneficiary survives the Optionee, then any vested and exercisable SARs held by the Optionee at the time of his or her death may be exercised by his or her estate.

6.7 Modification or Assumption of SARs. Within the limitations of the Plan, the Administrator may modify, reprice, extend or assume outstanding SARs or may accept the cancellation of outstanding SARs (whether granted by the Company or by another issuer) in return for the grant of new SARs for the same or a different number of shares and at the same or a different exercise price or in return for the grant of a different type of Award. The foregoing notwithstanding, no modification of a SAR shall, without the consent of the Optionee, impair his or her rights or obligations under such SAR.

ARTICLE 7. RESTRICTED SHARES.

7.1 Restricted Stock Agreement. Each grant of Restricted Shares under the Plan shall be evidenced by a Restricted Stock Agreement between the recipient and the Company.

Such Restricted Shares shall be subject to all applicable terms of the Plan and may be subject to any other terms that are not inconsistent with the Plan. The provisions of the various Restricted Stock Agreements entered into under the Plan need not be identical.

7.2 Payment for Awards. Restricted Shares may be sold or awarded under the Plan for such consideration as the Administrator may determine, including (without limitation) cash, cash equivalents, property, cancellation of other equity awards, full-recourse promissory notes, past services and future services, and such other methods of payment as are permitted by applicable law.

7.3 Vesting Conditions. Each Award of Restricted Shares may or may not be subject to vesting and/or other conditions as the Administrator may determine. Vesting shall occur, in full or in installments, upon satisfaction of the conditions specified in the Restricted Stock Agreement. Such conditions, at the Administrator’s discretion, may include one or more Performance Goals. A Restricted Stock Agreement may provide for accelerated vesting upon certain specified events.

7.4 Voting and Dividend Rights. The holders of Restricted Shares awarded under the Plan shall have the same voting, dividend and other rights as the Company’s other stockholders, unless the Administrator otherwise provides. A Restricted Stock Agreement, however, may require that any cash dividends paid on Restricted Shares (a) be accumulated and paid when such Restricted Shares vest, or (b) be invested in additional Restricted Shares. Such additional Restricted Shares shall be subject to the same conditions and restrictions as the shares subject to the Stock Award with respect to which the dividends were paid. In addition, unless the Administrator provides otherwise, if any dividends or other distributions are paid in Common Shares, such Common Shares shall be subject to the same restrictions on transferability and forfeitability as the Restricted Shares with respect to which they were paid.

ARTICLE 8. STOCK UNITS.

8.1 Stock Unit Agreement. Each grant of Stock Units under the Plan shall be evidenced by a Stock Unit Agreement between the recipient and the Company. Such Stock Units shall be subject to all applicable terms of the Plan and may be subject to any other terms that are not inconsistent with the Plan. The provisions of the various Stock Unit Agreements entered into under the Plan need not be identical.

8.2 Payment for Awards. To the extent that an Award is granted in the form of Stock Units, no cash consideration shall be required of the Award recipients.

8.3 Vesting Conditions. Each Award of Stock Units may or may not be subject to vesting, as determined by the Administrator. Vesting shall occur, in full or in installments, upon satisfaction of the conditions specified in the Stock Unit Agreement. Such conditions, at the Administrator’s discretion, may include one or more Performance Goals. A Stock Unit Agreement may provide for accelerated vesting upon certain specified events.

8.4 Voting and Dividend Rights. The holders of Stock Units shall have no voting rights. Prior to settlement or forfeiture, Stock Units awarded under the Plan may, at the Administrator’s discretion, provide for a right to dividend equivalents. Such right entitles the

holder to be credited with an amount equal to all cash dividends paid on one Common Share while the Stock Unit is outstanding. Dividend equivalents may be converted into additional Stock Units. Settlement of dividend equivalents may be made in the form of cash, in the form of Common Shares, or in a combination of both. Prior to distribution, any dividend equivalents shall be subject to the same conditions and restrictions as the Stock Units to which they attach.

8.5 Form and Time of Settlement of Stock Units. Settlement of vested Stock Units may be made in the form of (a) cash, (b) Common Shares or (c) any combination of both, as determined by the Administrator. The actual number of Stock Units eligible for settlement may be larger or smaller than the number included in the original Award, based on predetermined performance factors, including Performance Goals. Methods of converting Stock Units into cash may include (without limitation) a method based on the average Fair Market Value of Common Shares over a series of trading days. Vested Stock Units shall be settled in such manner and at such time(s) as specified in the Stock Unit Agreement. Until an Award of Stock Units is settled, the number of such Stock Units shall be subject to adjustment pursuant to Article 9.

8.6 Death of Recipient. Any Stock Units that become payable after the recipient’s death shall be distributed to the recipient’s beneficiary or beneficiaries. Each recipient of Stock Units under the Plan may designate one or more beneficiaries for this purpose by filing the prescribed form with the Company. A beneficiary designation may be changed by filing the prescribed form with the Company at any time before the Award recipient’s death. If no beneficiary was designated or if no designated beneficiary survives the Award recipient, then any Stock Units that become payable after the recipient’s death shall be distributed to the recipient’s estate.

8.7 Modification or Assumption of Stock Units. Within the limitations of the Plan, the Administrator may modify or assume outstanding stock units or may accept the cancellation of outstanding stock units (whether granted by the Company or by another issuer) in return for the grant of new Stock Units for the same or a different number of shares or in return for the grant of a different type of Award. The foregoing notwithstanding, no modification of a Stock Unit shall, without the consent of the Participant, impair his or her rights or obligations under such Stock Unit.

8.8 Creditors’ Rights. A holder of Stock Units shall have no rights other than those of a general creditor of the Company. Stock Units represent an unfunded and unsecured obligation of the Company, subject to the terms and conditions of the applicable Stock Unit Agreement.

ARTICLE 9. ADJUSTMENTS; DISSOLUTIONS AND LIQUIDATIONS; CORPORATE TRANSACTIONS.

9.1 Adjustments. In the event of a subdivision of the outstanding Common Shares, a declaration of a dividend payable in Common Shares or a combination or consolidation of the outstanding Common Shares (by reclassification or otherwise) into a lesser number of Common Shares, corresponding proportionate adjustments shall automatically be made in each of the following:

(a) The number and kind of shares available for issuance under Article 3, including the numerical share limits in Articles 3.1, 3.2 and 3.5;

(b) The number and kind of shares covered by each outstanding Option, SAR and Stock Unit; and

(c) The Exercise Price applicable to each outstanding Option and SAR, and the repurchase price, if any, applicable to Restricted Shares.

In the event of a declaration of an extraordinary dividend payable in a form other than Common Shares in an amount that has a material effect on the price of Common Shares, a recapitalization, a spin-off or a similar occurrence, the Administrator shall make such adjustments as it, in its sole discretion, deems appropriate in one or more of the foregoing. Any adjustment in the number of and kind of shares subject to an Award under this Article 9.1 shall be rounded down to the nearest whole share, although the Administrator in its sole discretion may make a cash payment in lieu of a fractional share. Except as provided in this Article 9, a Participant shall have no rights by reason of any issuance by the Company of stock of any class or securities convertible into stock of any class, any subdivision or consolidation of shares of stock of any class, the payment of any stock dividend or any other increase or decrease in the number of shares of stock of any class.

9.2 Dissolution or Liquidation. To the extent not previously exercised or settled, Options, SARs and Stock Units shall terminate immediately prior to the dissolution or liquidation of the Company.

9.3 Corporate Transactions. In the event that the Company is a party to a merger, consolidation, or a Change in Control (other than one described in Article 14.6(d)), all Common Shares acquired under the Plan and all Awards outstanding on the effective date of the transaction shall be treated in the manner described in the definitive transaction agreement (or, in the event the transaction does not entail a definitive agreement to which the Company is party, in the manner determined by the Administrator, with such determination having final and binding effect on all parties), which agreement or determination need not treat all Awards (or portions thereof) in an identical manner. Unless an Award Agreement provides otherwise, the treatment specified in the transaction agreement or by the Administrator shall include (without limitation) one or more of the following with respect to each outstanding Award:

(a) The continuation of such outstanding Awards by the Company (if the Company is the surviving entity);

(b) The assumption of such outstanding Awards by the surviving entity or its parent, provided that the assumption of an Option or a SAR shall comply with applicable tax requirements;

(c) The substitution by the surviving entity or its parent of an equivalent award for outstanding Awards (including, but not limited to, an award to acquire the same consideration paid to the holders of Common Shares in the transaction), provided that the substitution of an Option or a SAR shall comply with applicable tax requirements;

(d) The cancellation of outstanding Options and SARs without payment of any consideration. The Optionees shall be able to exercise such Options and SARs (to the extent the Options and SARs are vested or become vested as of the effective date of the transaction) during a period of not less than five full business days preceding the closing date of the transaction, unless (i) a shorter period is required to permit a timely closing of the transaction and (ii) such shorter period still offers the Optionees a reasonable opportunity to exercise such Options and SARs. Any exercise of such Options and SARs during such period may be contingent on the closing of the transaction;

(e) Full exercisability of outstanding Options and SARs and full vesting of the Common Shares subject to Options and SARs, followed by cancellation of such Options and SARs. The full exercisability of such Options and SARs and full vesting of such Common Shares may be contingent on the closing of the transaction. The Optionees shall be able to exercise such Options and SARs during a period of not less than five full business days preceding the closing date of such merger or consolidation, unless (i) a shorter period is required to permit a timely closing of such merger or consolidation and (ii) such shorter period still offers the Optionees a reasonable opportunity to exercise such Options and SARs. Any exercise of such Options and SARs during such period may be contingent on the closing of such merger or consolidation;

(f) The cancellation of the Options and SARs and a payment to the Optionee with respect to each Share subject to the portion of the Award that is vested as of the transaction date equal to the excess of (A) the value, as determined by the Administrator in its absolute discretion, of the property (including cash) received by the holder of a Common Share as a result of the transaction, over (B) the per-share Exercise Price of the Option or SAR (such excess, the “Spread”). Such payment shall be made in the form of cash, cash equivalents, or securities of the surviving entity or its parent having a value equal to the Spread. In addition, any escrow, holdback, earn-out or similar provisions in the transaction agreement may apply to such payment to the same extent and in the same manner as such provisions apply to the holders of Common Shares, but only to the extent the application of such provisions does not adversely affect the status of the Option or SAR as exempt from Code Section 409A. If the Spread applicable to an Option or SAR is zero or a negative number, then the Option or SAR may be cancelled without making a payment to the Optionee;

(g) The cancellation of outstanding Stock Units and a payment to the holder thereof with respect to each Common Share subject to the Stock Unit (whether or not such Stock Unit is then vested) equal to the value, as determined by the Administrator in its absolute discretion, of the property (including cash) received by the holder of a Common Share as a result of the transaction (the “Transaction Value”). Such payment shall be made in the form of cash, cash equivalents, or securities of the surviving entity or its parent having a value equal to the Transaction Value. In addition, such payment may be subject to vesting based on the Participant’s continuing Service, provided that the vesting schedule shall not be less favorable to the Participant than the schedule under which such Stock Units would have vested, and if required under applicable tax rules, such payment may be deferred until the settlement date specified in the Stock Unit Agreement. In addition, any escrow, holdback, earn-out or similar provisions in the transaction agreement may apply to such payment to the

same extent and in the same manner as such provisions apply to the holders of Common Shares. In the event that a Stock Unit is subject to Code Section 409A, the payment described in this clause (g) shall be made on the settlement date specified in the applicable Stock Unit Agreement, provided that settlement may be accelerated in accordance with Treasury Regulation Section 1.409A-3(j)(4); or

(h) The assignment of any reacquisition or repurchase rights held by the Company in respect of an Award of Restricted Shares to the surviving entity or its parent, with corresponding proportionate adjustments made to the price per share to be paid upon exercise of any such reacquisition or repurchase rights.

For avoidance of doubt, the Administrator shall have the discretion, exercisable either at the time an Award is granted or at any time while the Award remains outstanding, to provide for the acceleration of vesting upon the occurrence of a Change in Control, whether or not the Award is to be assumed or replaced in the transaction, or in connection with a termination of the Participant’s Service following a transaction.

Any action taken under this Article 9.3 shall either preserve an Award’s status as exempt from Code Section 409A or comply with Code Section 409A.

ARTICLE 10. OTHER AWARDS.

10.1 Performance Cash Awards. A Performance Cash Award is a cash award that may be granted subject to the attainment of specified Performance Goals during a Performance Period. A Performance Cash Award may also require the completion of a specified period of continuous Service. The length of the Performance Period, the Performance Goals to be attained during the Performance Period, and the degree to which the Performance Goals have been attained shall be determined conclusively by the Administrator. Each Performance Cash Award shall be set forth in a written agreement or in a resolution duly adopted by the Administrator which shall contain provisions determined by the Administrator and not inconsistent with the Plan. The terms of various Performance Cash Awards need not be identical.

10.2 Awards Under Other Plans. The Company may grant awards under other plans or programs. Such awards may be settled in the form of Common Shares issued under this Plan. Such Common Shares shall be treated for all purposes under the Plan like Common Shares issued in settlement of Stock Units and shall, when issued, reduce the number of Common Shares available under Article 3.

ARTICLE 11. LIMITATION ON RIGHTS.

11.1 Retention Rights. Neither the Plan nor any Award granted under the Plan shall be deemed to give any individual a right to remain a Service Provider. The Company and its Parents, Subsidiaries and Affiliates reserve the right to terminate the Service of any Service Provider at any time, with or without cause, subject to applicable laws, the Company’s certificate of incorporation and by-laws and a written employment agreement (if any).

11.2 Stockholders’ Rights. Except as set forth in Article 7.4 or 8.4 above, a Participant shall have no dividend rights, voting rights or other rights as a stockholder with

respect to any Common Shares covered by his or her Award prior to the time when a stock certificate for such Common Shares is issued or, if applicable, the time when he or she becomes entitled to receive such Common Shares by filing any required notice of exercise and paying any required Exercise Price. No adjustment shall be made for cash dividends or other rights for which the record date is prior to such time, except as expressly provided in the Plan.

11.3 Regulatory Requirements. Any other provision of the Plan notwithstanding, the obligation of the Company to issue Common Shares under the Plan shall be subject to all applicable laws, rules and regulations and such approval by any regulatory body as may be required. The Company reserves the right to restrict, in whole or in part, the delivery of Common Shares pursuant to any Award prior to the satisfaction of all legal requirements relating to the issuance of such Common Shares, to their registration, qualification or listing or to an exemption from registration, qualification or listing. The inability of the Company to obtain authority from any regulatory body having jurisdiction, which authority is deemed necessary by the Company’s counsel to be necessary to the lawful issuance and sale of any Common Shares hereunder, will relieve the Company of any liability in respect of the failure to issue or sell such Common Shares as to which such requisite authority will not have been obtained.

11.4 Transferability of Awards. The Administrator may, in its sole discretion, permit transfer of an Award in a manner consistent with applicable law. Unless otherwise determined by the Administrator, Awards shall be transferable by a Participant only by (a) beneficiary designation, (b) a will or (c) the laws of descent and distribution. An ISO may only be transferred by will or by the laws of descent and distribution and may be exercised during the lifetime of the Optionee only by the Optionee or by the Optionee’s guardian or legal representative.

11.5 Other Conditions and Restrictions on Common Shares. Any Common Shares issued under the Plan shall be subject to such forfeiture conditions, rights of repurchase, rights of first refusal, other transfer restrictions and such other terms and conditions as the Administrator may determine. Such conditions and restrictions shall be set forth in the applicable Award Agreement and shall apply in addition to any restrictions that may apply to holders of Common Shares generally. In addition, Common Shares issued under the Plan shall be subject to such conditions and restrictions imposed either by applicable law or by Company policy, as adopted from time to time, designed to ensure compliance with applicable law or laws with which the Company determines in its sole discretion to comply including in order to maintain any statutory, regulatory or tax advantage.

ARTICLE 12. TAXES.

12.1 General. As a condition to an Award under the Plan, a Participant or his or her successor shall make arrangements satisfactory to the Company for the satisfaction of any federal, state, local or foreign withholding tax obligations that arise in connection with any Award granted under the Plan. The Company shall not be required to issue any Common Shares or make any cash payment under the Plan until such obligations are satisfied.

12.2 Share Withholding. To the extent that applicable law subjects a Participant to tax withholding obligations, the Administrator may permit such Participant to satisfy all or part

of such obligations by having the Company withhold all or a portion of any Common Shares that otherwise would be issued to him or her or by surrendering all or a portion of any Common Shares that he or she previously acquired. Such Common Shares shall be valued at their Fair Market Value on the date when they are withheld or surrendered. Any payment of taxes by assigning Common Shares to the Company may be subject to restrictions including any restrictions required by SEC, accounting or other rules.

12.3 Section 162(m) Matters. The Administrator, in its sole discretion, may determine whether an Award is intended to qualify as “performance-based compensation” within the meaning of Code Section 162(m). The Administrator may grant Awards that are based on Performance Goals but that are not intended to qualify as performance-based compensation. With respect to any Award that is intended to qualify as performance-based compensation, the Administrator shall designate the Performance Goal(s) applicable to, and the formula for calculating the amount payable under, an Award within 90 days following commencement of the applicable Performance Period (or such earlier time as may be required under Code Section 162(m)), and in any event at a time when achievement of the applicable Performance Goal(s) remains substantially uncertain. Prior to the payment of any Award that is intended to constitute performance-based compensation, the Administrator shall certify in writing whether and the extent to which the Performance Goal(s) were achieved for such Performance Period. The Administrator shall have the right to reduce or eliminate (but not to increase) the amount payable under an Award that is intended to constitute performance-based compensation.

12.4 Section 409A Matters. Except as otherwise expressly set forth in an Award Agreement, it is intended that Awards granted under the Plan either be exempt from, or comply with, the requirements of Code Section 409A. To the extent an Award is subject to Code Section 409A (a “409A Award”), the terms of the Plan, the Award and any written agreement governing the Award shall be interpreted to comply with the requirements of Code Section 409A so that the Award is not subject to additional tax or interest under Code Section 409A, unless the Administrator expressly provides otherwise. A 409A Award shall be subject to such additional rules and requirements as specified by the Administrator from time to time in order for it to comply with the requirements of Code Section 409A. In this regard, if any amount under a 409A Award is payable upon a “separation from service” to an individual who is considered a “specified employee” (as each term is defined under Code Section 409A), then no such payment shall be made prior to the date that is the earlier of (i) six months and one day after the Participant’s separation from service or (ii) the Participant’s death, but only to the extent such delay is necessary to prevent such payment from being subject to Code Section 409A(a)(1).

12.5 Limitation on Liability. Neither the Company nor any person serving as Administrator shall have any liability to a Participant in the event an Award held by the Participant fails to achieve its intended characterization under applicable tax law.

ARTICLE 13. FUTURE OF THE PLAN.

13.1 Term of the Plan. The Plan, as set forth herein, shall become effective on the Registration Date. The Plan shall remain in effect until the earlier of (a) the date when the Plan is terminated under Article 13.2 or (b) the 10th anniversary of the date when the Board adopted the Plan.

13.2 Amendment or Termination. The Board may, at any time and for any reason, amend or terminate the Plan. No Awards shall be granted under the Plan after the termination thereof. The termination of the Plan, or any amendment thereof, shall not affect any Award previously granted under the Plan.

13.3 Stockholder Approval. An amendment of the Plan shall be subject to the approval of the Company’s stockholders only to the extent required by applicable laws, regulations or rules.

ARTICLE 14. DEFINITIONS.

14.1 “Administrator” means the Board or any Committee administering the Plan in accordance with Article 2.

14.2 “Affiliate” means any entity other than a Subsidiary, if the Company and/or one or more Subsidiaries own not less than 50% of such entity.

14.3 “Award” means any award granted under the Plan, including as an Option, a SAR, a Restricted Share, a Stock Unit or a Performance Cash Award.

14.4 “Award Agreement” means a Stock Option Agreement, an SAR Agreement, a Restricted Stock Agreement, a Stock Unit Agreement or such other agreement evidencing an Award granted under the Plan.

14.5 “Board” means the Company’s Board of Directors, as constituted from time to time.

14.6 “Change in Control” means:

(a) Any “person” (as such term is used in Sections 13(d) and 14(d) of the Exchange Act) becomes the “beneficial owner” (as defined in Rule 13d-3 of the Exchange Act), directly or indirectly, of securities of the Company representing more than fifty percent (50%) of the total voting power represented by the Company’s then-outstanding voting securities;

(b) The consummation of the sale or disposition by the Company of all or substantially all of the Company’s assets;

(c) The consummation of a merger or consolidation of the Company with or into any other entity, other than a merger or consolidation which would result in the voting securities of the Company outstanding immediately prior thereto continuing to represent (either by remaining outstanding or by being converted into voting securities of the surviving entity or its parent) more than fifty percent (50%) of the total voting power represented by the voting securities of the Company or such surviving entity or its parent outstanding immediately after such merger or consolidation; or

(d) Individuals who are members of the Board (the “Incumbent Board”) cease for any reason to constitute at least a majority of the members of the Board over a period of 12 months; provided, however, that if the appointment or election (or nomination for election) of any new Board member was approved or recommended by a majority vote of the members of the Incumbent Board then still in office, such new member shall, for purposes of this Plan, be considered as a member of the Incumbent Board.

A transaction shall not constitute a Change in Control if its sole purpose is to change the state of the Company’s incorporation or to create a holding company that will be owned in substantially the same proportions by the persons who held the Company’s securities immediately before such transaction. In addition, if a Change in Control constitutes a payment event with respect to any Award which provides for a deferral of compensation and is subject to Code Section 409A, then notwithstanding anything to the contrary in the Plan or applicable Award Agreement the transaction with respect to such Award must also constitute a “change in control event” as defined in Treasury Regulation Section 1.409A-3(i)(5) to the extent required by Code Section 409A.

14.7 “Code” means the Internal Revenue Code of 1986, as amended.

14.8 “Committee” means a committee of one or more members of the Board, or of other individuals satisfying applicable laws, appointed by the Board to administer the Plan.

14.9 “Common Share” means one share of the common stock of the Company.

14.10 “Company” means REGENXBIO Inc., a Delaware corporation.

14.11 “Consultant” means a consultant or adviser who provides bona fide services to the Company, a Parent, a Subsidiary or an Affiliate as an independent contractor and who qualifies as a consultant or advisor under Instruction A.1.(a)(1) of Form S-8 under the Securities Act of 1933, as amended.

14.12 “Employee” means a common-law employee of the Company, a Parent, a Subsidiary or an Affiliate.

14.13 “Exchange Act” means the Securities Exchange Act of 1934, as amended.

14.14 “Exercise Price,” in the case of an Option, means the amount for which one Common Share may be purchased upon exercise of such Option, as specified in the applicable Stock Option Agreement. “Exercise Price,” in the case of a SAR, means an amount, as specified in the applicable SAR Agreement, which is subtracted from the Fair Market Value of one Common Share in determining the amount payable upon exercise of such SAR.

14.15 “Fair Market Value” means the closing price of a Common Share on any established stock exchange or a national market system on the applicable date or, if the applicable date is not a trading day, on the last trading day prior to the applicable date, as reported in a source that the Administrator deems reliable. If Common Shares are no longer traded on an established stock exchange or a national market system, the Fair Market Value shall be determined by the Administrator in good faith on such basis as it deems appropriate. The Administrator’s determination shall be conclusive and binding on all persons.

14.16 “ISO” means an incentive stock option described in Code Section 422(b).

14.17 “NSO” means a stock option not described in Code Sections 422 or 423.

14.18 “Option” means an ISO or NSO granted under the Plan and entitling the holder to purchase Common Shares.

14.19 “Optionee” means an individual or estate holding an Option or SAR.

14.20 “Outside Director” means a member of the Board who is not an Employee.

14.21 “Parent” means any corporation (other than the Company) in an unbroken chain of corporations ending with the Company, if each of the corporations other than the Company owns stock possessing 50% or more of the total combined voting power of all classes of stock in one of the other corporations in such chain. A corporation that attains the status of a Parent on a date after the adoption of the Plan shall be considered a Parent commencing as of such date.

14.22 “Participant” means an individual or estate holding an Award.

14.23 “Performance Cash Award” means an award of cash granted under Article 10.1 of the Plan.

14.24 “Performance Goal” means a goal established by the Administrator for the applicable Performance Period based on one or more of the performance criteria set forth in Appendix A. Depending on the performance criteria used, a Performance Goal may be expressed in terms of overall Company performance or the performance of a business unit, division, Subsidiary, Affiliate or an individual. A Performance Goal may be measured either in absolute terms or relative to the performance of one or more comparable companies or one or more relevant indices. The Administrator may adjust the results under any performance criterion to exclude any of the following events that occurs during a Performance Period: (a) asset write-downs, (b) litigation, claims, judgments or settlements, (c) the effect of changes in tax laws, accounting principles or other laws or provisions affecting reported results, (d) accruals for reorganization and restructuring programs, (e) extraordinary, unusual or non-recurring items, (f) exchange rate effects for non-U.S. dollar denominated net sales and operating earnings, or (g) statutory adjustments to corporate tax rates; provided, however, that if an Award is intended to qualify as “performance-based compensation” within the meaning of Code Section 162(m), such adjustment(s) shall only be made to the extent consistent with Code Section 162(m).

14.25 “Performance Period” means a period of time selected by the Administrator over which the attainment of one or more Performance Goals will be measured for the purpose of determining a Participant’s right to a Performance Cash Award or an Award of Restricted Shares or Stock Units that vests based on the achievement of Performance Goals. Performance Periods may be of varying and overlapping duration, at the discretion of the Administrator.

14.26 “Plan” means this REGENXBIO Inc. 2015 Equity Incentive Plan, as amended from time to time.

14.27 “Predecessor Plan” means the Company’s 2014 Stock Plan, as amended.

14.28 “Registration Date” means the effective date of the registration statement filed by the Company with the Securities and Exchange Commission pursuant to Form S-1.

14.29 “Restricted Share” means a Common Share awarded under the Plan.

14.30 “Restricted Stock Agreement” means the agreement between the Company and the recipient of a Restricted Share that contains the terms, conditions and restrictions pertaining to such Restricted Share.

14.31 “SAR” means a stock appreciation right granted under the Plan.

14.32 “SAR Agreement” means the agreement between the Company and an Optionee that contains the terms, conditions and restrictions pertaining to his or her SAR.

14.33 “Service” means service as an Employee, Outside Director or Consultant.

14.34 “Service Provider” means any individual who is an Employee, Outside Director or Consultant.

14.35 “Stock Award” means any award of an Option, a SAR, a Restricted Share or a Stock Unit under the Plan.

14.36 “Stock Option Agreement” means the agreement between the Company and an Optionee that contains the terms, conditions and restrictions pertaining to his or her Option.

14.37 “Stock Unit” means a bookkeeping entry representing the equivalent of one Common Share, as awarded under the Plan.

14.38 “Stock Unit Agreement” means the agreement between the Company and the recipient of a Stock Unit that contains the terms, conditions and restrictions pertaining to such Stock Unit.

14.39 “Subsidiary” means any corporation (other than the Company) in an unbroken chain of corporations beginning with the Company, if each of the corporations other than the last corporation in the unbroken chain owns stock possessing 50% or more of the total combined voting power of all classes of stock in one of the other corporations in such chain. A corporation that attains the status of a Subsidiary on a date after the adoption of the Plan shall be considered a Subsidiary commencing as of such date

14.40 “Substitute Awards” means Awards or Common Shares issued by the Company in assumption of, or substitution or exchange for, Awards previously granted, or the right or obligation to make future awards, in each case by a corporation acquired by the Company or any Affiliate or with which the Company or any Affiliate combines to the extent permitted by NASDAQ Marketplace Rule 5635 or any successor thereto.

APPENDIX A

PERFORMANCE CRITERIA

The Administrator may establish Performance Goals derived from one or more of the following criteria when it makes Awards of Restricted Shares or Stock Units that vest entirely or in part on the basis of performance or when it makes Performance Cash Awards:



• Earnings (before or after taxes)		• Sales or revenue (using a measure thereof that complies with Section 162(m))

• Earnings per share		• Expense or cost reduction

• Earnings before interest, taxes and depreciation		• Working capital

• Earnings before interest, taxes, depreciation and amortization		• Economic value added (or an equivalent metric)

• Total stockholder return		• Market share

• Return on equity or average stockholders’ equity		• Cash measures including cash flow and cash balance

• Return on assets, investment or capital employed		• Operating cash flow

• Operating income		• Cash flow per share

• Gross margin		• Share price

• Operating margin		• Debt reduction

• Net operating income		• Customer satisfaction

• Net operating income after tax		• Stockholders’ equity

• Return on operating revenue		• Contract awards or backlog

• Objective corporate or individual strategic goals		• Objective individual performance goals

• To the extent that an Award is not intended to comply with Code Section 162(m), other measures of performance selected by the Administrator

EX-10.9

Exhibit 10.9

CONFIDENTIAL TREATMENT REQUESTED

UNIVERSITY of PENNSYLVANIA

License Agreement

This License Agreement (this “Agreement”) is between The Trustees of the University of Pennsylvania, a Pennsylvania nonprofit corporation (“Penn”), and ReGenX, LLC, a Delaware limited liability company (“Company”). This Agreement is being signed on February 20, 2009 (the “Execution Date”). This Agreement will be effective on February 24, 2009 (the “Effective Date”).

BACKGROUND

Penn owns certain intellectual property developed by Dr. James M. Wilson, M.D., Ph.D. of Penn’s School of Medicine (“Dr. Wilson”) relating to a gene therapy technology platform based on certain novel adeno associated viruses discovered by Dr. Wilson at Penn. Penn also owns certain letters patent and/or applications, including provisional patent applications, for letters patent relating to the intellectual property. The Company desires to obtain an exclusive license under the patent rights and related know how to exploit the intellectual property relating to the gene therapy technology platform. Company also desires to fund further research by Dr. Wilson under a separate sponsored research agreement and to obtain an exclusive option under such sponsored research agreement to negotiate for an exclusive license in any intellectual property created, conceived or reduced to practice pursuant to such SRA. Penn has determined that the exploitation of the intellectual property by Company is in the best interest of Penn and is consistent with its educational and research missions and goals.

In consideration of the mutual obligations contained in this Agreement, and intending to be legally bound, the parties agree as follows:

1. LICENSE

1.1 License Grants.

(a) License to Patent Rights. Subject to the limitations set forth in Section 1.1(b) Penn hereby grants to Company an exclusive, worldwide license under the Patent Rights to make, have made, use, import, offer for sale and sell Licensed Products in the Field of Use during the Term (as such terms may be defined in Section 1.2)(the “Patent License”). The Patent License includes the right to sublicense as permitted by this Agreement.

(b) Limitations With Respect to Certain Patent Rights.

(i) The parties acknowledge that with respect to the patents and patent applications listed on Exhibit B-1 only (the “**** Licensed Patents”), Penn has already granted a nonexclusive license to **** pursuant to the agreement identified in Exhibit B-1 (the “**** License”). Accordingly, the rights granted to Penn herein with respect to the **** Licensed Patents only shall be nonexclusive for so long as such preexisting license grant remains in effect. Penn shall have no right to grant or authorize any third party to grant any further rights or licenses in the Field of Use with respect to the **** Licensed Patents during the Term and the rights granted to Company with respect to the **** Licensed Patents shall

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

automatically become exclusive upon the expiration or termination of the existing license under the **** License without further action by either party. Penn will promptly notify Company of any change with respect to the rights licensed pursuant to the **** License.

(ii) The parties acknowledge that with respect to the patents and patent applications listed on Exhibit B-2 only (the “GSK Licensed Patents”), Penn has already granted a license to SmithKline Beecham Corporation dba GlaxoSmithKline (“GSK”) pursuant to the agreement identified in Exhibit B-2 (the “GSK License”). The parties agree that for long as GSK maintains a license to the GSK Licensed Patents in the Field of Use, the rights granted herein with respect to the GSK Licensed Patents only will be subject to the rights granted to GSK prior to the Effective Date. The parties acknowledge that Company is seeking a sublicense under the GSK Licensed Patents directly from GSK and understand that Penn may receive royalties (“Company Sublicense Royalty Revenues”) or other payments (“Company Sublicense Non-Royalty Revenues”) under the GSK License as a result of sublicenses granted to the Company. The parties agree that any Company Sublicense Royalty Revenues shall be offset against any amounts due to Penn hereunder. ****. Penn shall have no right to grant or authorize any third party to grant any further rights or licenses in the Field of Use with respect to the GSK Licensed Patents during the Term and to the extent any rights with respect to the GSK Licensed Patents in the Field of Use revert to Penn whether through expiration or termination of the GSK License, by contract or otherwise, such rights shall be automatically included within the scope of the license granted pursuant to Section 1.1(a) without further action by either party. Penn will promptly notify Company of any change with respect to the rights licensed pursuant to the GSK License.

(c) License to Background Know-How. Penn hereby grants to Company a non-exclusive, worldwide, license (the “Background Know-How License”) under the Background Know-How to make, have made, use, import, offer for sale and sell and otherwise exploit Licensed Products in the Field of Use and to practice the Licensed Processes in connection with the exercise of the foregoing rights in 1.1(a) and 1.1(b) (as such terms may be defined in Section 1.2). The Background Know-How License includes the right to sublicense as permitted by this Agreement. ****.

(d) No Other Licenses. Except as expressly provided in this Section 1.1, no other rights or licenses are granted by Penn. Any intellectual property created or conceived during the performance of the Sponsored Research Agreement between Penn and Company being entered into simultaneously with this Agreement (the “SRA”) will be governed by the terms of the SRA.

1.2 Related Definitions.

“Affiliate” means a legal entity that is controlling, controlled by or under common control with Company and that has executed either this Agreement or a written Joinder Agreement agreeing to be bound by all of the terms and conditions of this Agreement. For purposes of this

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Section 1.2, the word “control” means (x) the direct or indirect ownership of more than fifty percent (50%) of the outstanding voting securities of a legal entity, (y) the right to receive fifty percent (50%) or more of the profits or earnings of a legal entity, or (z) the right to determine the policy decisions of a legal entity.

“Background Know-How” means all Know-How that (a) was developed by Dr. Wilson , or other Penn researchers working under his direct supervision, at Penn, and (b) is related to the adeno associated virus technology platform discovered by Dr. Wilson at Penn prior to the date hereof, and (c) is owned by Penn, (d) is necessary or useful for the practice of the Patent Rights in connection with the manufacture, use, sale, importation and/or other exploitation of the Licensed Products or the practice of the Licensed Processes in the Territory in the Field of Use, including, without, limitation, any Know-How necessary for the Company to the manufacture or have manufactured the materials produced by the Penn Vector Core or Dr. Wilson’s lab at Penn.

“Field of Use” means any and all fields of use.

“Know-How” means any and all information, discoveries, software, methods, works of authorship, techniques, formulae, data, biological materials, processes, unpatentable inventions and other know-how, not including the Patent Rights, developed prior to the Effective Date.

“Licensed IP” means the Patent Rights and Background Know-How.

“Licensed Process” means any process or machine covered by the Licensed IP or any claim thereof, whether or not the claim is issued or pending.

“Licensed Products” means any products that are made, made for, used, imported, offered for sale or sold by or for Company or its Affiliates or sublicensees and that either (i) in the absence of this Agreement, would infringe or misappropriate the Licensed IP, or any claim thereof whether or not the claim is issued or pending, or (ii) use, or are manufactured using, a Licensed Process. Licensed Products include Licensed Pharmaceutical Products and Licensed Reagents.

“Licenses” means the Patent License and the Background Know-How License.

“Patent Rights” means (i) all of Penn’s patent rights represented by or issuing from the United States patents and patent applications (including provisional patent applications) listed in Exhibit A, as well as any continuations, continuations-in-part (to the extent the inventions claimed or disclosed in any such patent or patent applications are directed to subject matter specifically described in the patent or patent applications listed in Exhibit A), divisionals, reexaminations, renewals, re-issues, substitutions, extensions and foreign counterparts of any of the foregoing, and all other patents and patent applications that claim priority from or have common priority with any of the foregoing patents and patent applications, (to the extent the inventions claimed or disclosed in any such patent or patent applications are directed to subject matter specifically described in the patent or patent applications listed in Exhibit A) and including any patents issuing from any of the foregoing; and (ii) all patentable inventions (to the extent they are or become available for license) that (a) were discovered by Dr. Wilson, or other Penn researchers working under his direct supervision, at Penn prior to the Effective Date hereof, and (b) are related to the adeno associated virus technology platform discovered by Dr. Wilson at Penn prior to the date hereof, and (c) are owned by Penn.

CONFIDENTIAL TREATMENT REQUESTED

“SRA” means the Sponsored Research Agreement between the Company and Penn entered into simultaneously herewith, or thereafter.

“Territory” means worldwide.

1.3 Reservation of Rights by Penn. Penn reserves the fully-paid, royalty free right to use, and to permit other non-commercial entities to use, the Patent Rights, but not to authorize any commercial third party to use, the Patent Rights solely for educational and research purposes.

1.4 U.S. Government Rights. The parties acknowledge that the United States government retains rights in intellectual property funded under any grant or similar contract with a Federal agency. The License is expressly subject to all applicable United States government rights, including, but not limited to, any applicable requirement that products, which result from such intellectual property and are sold in the United States, must be substantially manufactured in the United States.

1.5 Sublicense Conditions. The Company’s right to sublicense granted by Penn under the License is subject to each of the following conditions:

(a) Within **** after Company enters into a sublicense agreement, Company will deliver to Penn a complete and accurate copy of the entire executed sublicense agreement written in the English language.

(b) In each sublicense agreement, Company will require the sublicensee, and any further sublicensees, to comply with the terms and conditions of this Agreement.

(c) Company’s execution of a sublicense agreement will not relieve Company of any of its obligations under this Agreement. ****.

2. DILIGENCE

2.1 Development Plan. Company will deliver to Penn, on or before the first anniversary of the Effective Date, a copy of the Company’s development plan for the Patent Rights (the “Development Plan”). The purpose of the Development Plan is (a) to present the Company’s strategy to bring the Patent Rights to commercialization, (b) to project the timeline for completing the necessary tasks to accomplish the goals of the strategy. Company will provide Penn with a written update to the Development Plan at least once every two years after the Effective Date.

2.2 Company’s Efforts. Company will use commercially reasonable efforts to develop, commercialize, market and sell a Licensed Product in any part of the Territory. Commercially reasonable efforts shall mean efforts consistent with those utilized by ****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

****. The Company will achieve each of the diligence events by the applicable completion date listed in the table below for the first Licensed Product:


DILIGENCE EVENT		COMPLETION DATE
****		****
****		****
****		****

****.

2.3 Satisfaction of Diligence. Upon the earlier of the satisfaction of each of the Diligence events specified or upon first Sale of a US government drug regulatory agency (or foreign equivalent) approved Licensed Product in the Territory, Company shall be deemed to have fully satisfied all of its obligations under this Section 2.

3. FEES AND ROYALTIES

3.1 Equity Issuance. In partial consideration for the Licenses, Company will issue to Penn on the Effective Date such number of shares of Common Stock of the Company as will cause Penn to own at least **** of the capital stock of Company (or ownership units of an LLC, as appropriate) on a fully diluted basis on the Effective Date, assuming the exercise, conversion and exchange of all outstanding securities of Company for or into shares of Common Stock (or ownership units, as appropriate). The issuance of equity or ownership units to Penn will be pursuant to a Stock Purchase Agreement and a Stockholders Agreement, or their LLC equivalents, between Company and Penn), the forms of which are attached as Exhibits C and D (the “Equity Documents”).

3.2 Earned Royalties. In partial consideration of the Licenses, on the terms and subject to the conditions set forth herein, during the Royalty Term, Company will pay to Penn the following royalty as set forth below:

(a) on Net Sales of Licensed Pharmaceutical Products sold by Company or its Affiliates:


Licensed Pharmaceutical Products	RoyaltyPercentage	ReGenX Annual Net Sales, Cumulative (Million)
1. Using Novel AAV	****	Up to $300;

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


	****	Greater than or equal to $300 and up to $600
	****	Greater than or equal to $600
2. Using Refinement or Modification to existing AAV	****	Up to $300;
	****	Greater than or equal to $300 and up to $600
	****	Greater than or equal to $600

(b) on Net Sales of Licensed Reagents sold by Company or its Affiliates or sublicensees:


Licensed Reagents	Royalty Percentage	ReGenX Annual Net Sales, Cumulative (Million)
1. Using Novel AAV	****	Up to $10;
	****	Greater than or equal to $10 and up to $20
	****	Greater than or equal to $20
2. Using Refinement or Modification to existing AAV	****	Up to $10;
	****	Greater than or equal to $10 and up to $20
	****	Greater than or equal to $20

(c) on royalties received by Company from third parties on Net Sales of Licensed Pharmaceutical Products by such third parties:


Licensed Pharmaceutical Products	Royalty Percentage	Third Party Annual Net Sales, Cumulative (Million)
1. Using Novel AAV	****	Up to $300;
	****	Greater than or equal to $300 and up to $600
	****	Greater than or equal to $600

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


2. Using Refinement or Modification to existing AAV	****	Up to $300;
	****	Greater than or equal to $300 and up to $600
	****	Greater than or equal to $600

To meet the requirements of the term “Novel AAV” (as used in Category 1), there must be neither any dominating third party patent nor any Penn-owned patent rights other than those licensed under this Agreement with respect to the vector per se (i.e., no third party patent or Penn-owned patent rights beyond those licensed under this Agreement is required in order to make, have made, use, import, offer for sale or sell the vector for the higher royalty level to apply). Licenses from Penn to ReGenX for genes used, promoters used other than those which are part of the vector as described in Penn Patent Rights and the like in the vector will not affect the royalty pursuant to this provision. If any dominating third party patent or any Penn-owned patent other than those licensed under this Agreement issues at any time during the term of this Agreement with respect to a vector licensed hereunder, then the royalty level will immediately drop to the “Refinement” level (Category 2 above) for any Licensed Product containing such vector.

Notwithstanding the foregoing (i) in no event shall the **** paid to Penn by the Company pursuant to (c) above, **** that would be payable to Penn by the Company on such Net Sales of Licensed Pharmaceutical Products sold by Company and (ii) in no event shall **** be payable in connection with any ****. No royalties other than the payments set forth herein shall be due in connection with the exercise of the rights granted herein. ****.

3.5 Sublicense Fees. In partial consideration of the Licenses, and subject to the terms and conditions set forth herein, Company will pay to Penn a sublicense fee equal to the following percentage of the sum of all fees and milestone payments received by Company from sublicensees from the grant of sublicenses (including options to obtain a sublicense) of the Licensed Intellectual Property during the Quarter (“Sublicensing Revenues”):


Date of Sublicense Grant	Sublicensing Fees
During the period commencing on the Effective Date and ending on the day prior to the fourth anniversary of the Effective Date		*	***
Any date on or after the fourth anniversary of the Effective Date		*	***

Sublicensing Revenues shall not include (a) royalties paid to Company by a sublicensee based upon Sales or Net Sales by the sublicensee; (b) equity investments in Company by a sublicensee and any other non-cash consideration; (c) loan proceeds paid to Company by a sublicensee in an arms length, full recourse debt financing to the extent that such loan is not forgiven; (d) sponsored research funding paid to Company by a sublicensee in a bona fide transaction for future research to be performed by Company.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

****.

3.8 Related Definitions.

“Fair Market Value” means the cash consideration that Company or its Affiliates or sublicensees would realize from an unrelated buyer in an arms length sale of an identical item sold in the same quantity and at the time and place of the transaction. The Fair Market Value shall be determined jointly by Penn and Company based on transactions of a similar type and standard industry practice, if any.

“Licensed Pharmaceutical Products” means all Licensed Products other than Licensed Reagents, including, without limitation, any Licensed Product that is intended for therapeutic use.

“Licensed Reagents” means a Licensed Product that is intended for research uses only, excluding any research uses in humans.

“Net Sales” means the total cash consideration received by Company or its Affiliates ****.

“Qualifying Costs” means: (a) ****.

“Quarter” means each three-month period beginning on January 1, April 1, July 1 and October 1.

“Royalty Term” means, on a product-by-product, country-by-country basis with respect to Licensed Products, the period commencing on the date of the first Sale of such Licensed Product and ending on the date the Licensed Product ceases to be covered by a valid claim (issued or pending) of the Patent Rights.

“Sale” means any bona fide transaction for which consideration is received by Company or its Affiliate or sublicensee for the sale, use, lease, transfer or other disposition of a Licensed

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Product to an unaffiliated third party. A Sale is deemed completed at the time that Company or its Affiliate or sublicensee receives payment for the Licensed Product.

3.9 Payment Reductions. In the event that during the Royalty Period, litigation between Company and a third party commences involving the Licensed IP, and the third party has launched a competitive product, until such litigation is finally settled or adjudicated pursuant to a nonappealable final order by a court of competent jurisdiction, or a final and binding order issued pursuant to an alternative dispute resolution procedure, ****. If following such verdict, Company is permitted to continue selling Licensed Products or the competitive product is prevented from entering or further sale in the applicable country in the territory by one or more valid claims of the Licensed Patent Rights, ****. If following such verdict, Company is prohibited from continuing to sell Licensed Products, ****.

4. REPORTS AND PAYMENTS

4.1 Royalty Reports. Within **** after the end of each Quarter following the first Sale, Company will deliver to Penn a report, certified by the chief financial officer of Company, detailing the calculation of all royalties, fees and other payments due to Penn for such Quarter. The report will include the following information for the Quarter, each listed by product, by country: (a) the number of units of Licensed Products constituting Sales; (b) the gross consideration received for Sales; (c) Qualifying Costs, listed by category of cost; (d) Net Sales; (e) the gross amount of any qualifying payments and other consideration received by Company from sublicensees; (f) amounts of any deductions permitted by Section 3.9; (g) the royalties, fees and other payments owed to Penn, listed by category; and (h) the computations for any applicable currency conversions. Each royalty report will be substantially in the form of the sample report attached as Exhibit E.

4.2 Payments. Company will pay all royalties, fees and other payments due to Penn under Sections 3.2, 3.3, 3.4, 3.5, 3.6 and 3.7 within **** after the end of the Quarter in which the royalties, fees or other payments accrued.

4.3 Records. Company will maintain, and will cause its Affiliates and sublicensees to maintain, complete and accurate books, records and related background information to verify Sales, Net Sales, and all of the royalties, fees, and other payments due or paid under this Agreement, as well as the various computations reported under Section 4.1. The records for each Quarter will be maintained for at least **** after submission of the applicable report required under Section 4.1.

4.4 Audit Rights. Upon reasonable prior written notice to Company, Company and its Affiliates and sublicensees will provide Penn and its accountants with access to all of the books, records and related background information required by Section 4.3 to conduct a review or audit of Sales, Net Sales, and all of the royalties, fees, and other payments payable or paid under this Agreement. Access will be made available: (a) during normal business hours; (b) in a manner reasonably designed to facilitate Penn’s review or audit without unreasonable disruption to Company’s business; and (c) no more than once each calendar year during the Term (as defined below) and for a period of **** thereafter. Company will promptly pay to Penn

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

the amount of any underpayment determined by the review or audit, plus accrued interest. If the review or audit determines that Company has underpaid any payment by **** or more, then Company will also promptly pay the costs and expenses of Penn and its accountants in connection with the review or audit.

4.5 Currency. All dollar amounts referred to in this Agreement are expressed in United States dollars. All payments will be made in United States dollars. If Company receives payment from a third party in a currency other than United States dollars for which a royalty or fee or other payment is owed under this Agreement, then (a) the payment will be converted into United States dollars at the conversion rate for the foreign currency as published in the eastern edition of the Wall Street Journal as of the last business day of the Quarter in which the payment was received by Company, and (b) the conversion computation will be documented by Company in the applicable report delivered to Penn under Section 4.1.

4.6 Place of Payment. All payments by Company are payable to “The Trustees of the University of Pennsylvania” and will be made to the following addresses:


By Electronic Transfer:		By Check:
Wachovia Bank, N.A.		The Trustees of the University of Pennsylvania
ABA #****		c/o Center for Technology Transfer
Account Number: ****		PO Box 785546
Center for Technology Transfer		Philadelphia, PA 19178-5546
Attention: ****

4.7 Interest. All amounts that are not paid by Company when due will accrue interest from the date due until paid at a rate equal to one and one-half percent (1.5%) per month (or the maximum allowed by law, if less).

5. CONFIDENTIALITY AND USE OF PENN’S NAME

5.1 Confidentiality Agreement. If Company and Penn entered into one or more Confidential Disclosure Agreements prior to the Effective Date, then such agreements will continue to govern the protection of confidential information under this Agreement, and each Affiliate and sublicensee of Company will be bound to Company’s obligations under such agreements. If, however, no Confidential Disclosure Agreement has been entered into between Company and Penn prior to the Effective Date, then in connection with the execution of this Agreement, the parties will enter into a Confidential Disclosure Agreement substantially similar to Penn’s standard form. The term “Confidentiality Agreement” means all Confidential Disclosure Agreements between the parties that remain in effect after the Effective Date.

5.2 Other Confidential Matters. Penn is not obligated to accept any confidential information from Company, except for the reports required by Sections 2.1, 4.1, 4.4 and 6.6. Penn, acting through its Center for Technology Transfer and finance offices, will use reasonable efforts not to disclose to any third party outside of Penn any confidential information of Company contained in those reports, for so long as such information remains confidential. Penn bears no institutional responsibility for maintaining the confidentiality of any other information of Company. Company may elect to enter into confidentiality agreements with individual investigators at Penn that comply with Penn’s internal policies.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

5.3 Use of Penn’s Name. Company and its Affiliates, sublicensees, employees, and agents may not use the name, logo, seal, trademark, or service mark (including any adaptation of them) of Penn or any Penn school, organization, employee, student or representative, without the prior written consent of Penn.

6. TERM AND TERMINATION

6.1 Term. This Agreement will commence on Effective Date and end upon the expiration of the Royalty Term (the “Term”). Earned royalties pursuant to Section 3.4 shall only be payable hereunder during the Royalty Term.

6.2 Early Termination by Company. Company may terminate this Agreement at any time effective upon completion of each of the following conditions: (a) providing at least sixty (60) days prior written notice to Penn of such intention to terminate; (b) ceasing to make, have made, use, import, offer for sale and sell all Licensed Products; (c) terminating all sublicenses and causing all Affiliates and sublicensees to cease making, having made, using, importing, offering for sale and selling all Licensed Products; and (d) paying all amounts owed to Penn under this Agreement and any Sponsored Research Agreement between Penn and Company related to the Patent Rights, through the effective date of termination.

6.3 Early Termination by Penn. Penn may terminate this Agreement if: (a) Company is more than thirty (30) days late in paying to Penn any amounts owed under this Agreement and does not pay Penn in full within thirty (30) days after receipt of written notice indicating such default and demanding payment, including accrued interest (a “Payment Default”); (b) other than a Payment Default, Company or its Affiliate or sublicensee fails to achieve a diligence event on or before the applicable completion date or otherwise breaches this Agreement and does not cure such failure or breach within sixty (60) days after written notice of the breach; or (c) Company or its Affiliate or sublicensee experiences a Trigger Event.

6.4 Trigger Event. The term “Trigger Event” means any of the following: (a) if Company or its Affiliate or sublicensee (i) becomes insolvent, bankrupt or generally fails to pay its debts as such debts become due, (ii) is adjudicated insolvent or bankrupt, (iii) admits in writing its inability to pay its debts, (iv) suffers the appointment of a custodian, receiver or trustee for it or its property and, if appointed without its consent, not discharged within thirty (30) days, (v) makes an assignment for the benefit of creditors, or (vi) suffers proceedings being instituted against it under any law related to bankruptcy, insolvency, liquidation or the reorganization, readjustment or release of debtors and, if contested by it, not dismissed or stayed within ten (10) days; (b) the institution or commencement by Company or its Affiliate or sublicensee of any proceeding under any law related to bankruptcy, insolvency, liquidation or the reorganization, readjustment or release of debtors; (c) the entering of any order for relief relating to any of the proceedings described in Section 6.4(a) or (b) above; (d) the calling by Company or its Affiliate or sublicensee of a meeting of its creditors with a view to arranging a composition or adjustment of its debts; (e) the act or failure to act by Company or its Affiliate or sublicensee indicating its consent to, approval of or acquiescence in any of the proceedings described in Section 6.4(b) — (d) above; or (f) the commencement by Company of any action against Penn, including an action for declaratory judgment, to declare or render invalid or unenforceable the Patent Rights, or any claim thereof.

CONFIDENTIAL TREATMENT REQUESTED

6.5 Effect of Termination. Upon the valid early termination of this Agreement pursuant to Section 6.2 or 6.3: (a) the Licenses shall terminate; (b) Company and all its Affiliates and sublicensees will cease all making, having made, using, importing, offering for sale and selling all Licensed Products and practicing the Licensed Processes; (c) Company will pay to Penn all amounts, including accrued interest, owed to Penn under this Agreement and any Sponsored Research Agreement related to the Patent Rights, through the date of termination, including royalties on Licensed Products invoiced or shipped through the date of termination when such payments are received, whether or not payment is received prior to termination; (d) each Party will, at the other Party’s request, return to such Party all confidential information of such Party; and (e) except as provided in Section 6.6, all rights and duties of Penn and the Company under this Agreement immediately terminate without further action required by either Penn or Company.

6.6 Survival. Company’s obligation to pay all amounts, including accrued interest, owed to Penn under this Agreement will survive the termination of this Agreement for any reason. Sections 13.9 and 13.10 and Articles 4, 5, 6, 9, 10, and 11 will survive the termination of this Agreement for any reason in accordance with their respective terms.

7. PATENT PROSECUTION AND MAINTENANCE

7.1 Patent Control. Except as otherwise provided in this Section 7.1, Penn shall control the preparation, prosecution and maintenance of the Patent Rights and the selection of patent counsel, with input from Company. If, however, Company desires a greater degree of control over the Patent Rights, then Company and Penn will use good faith efforts to promptly enter into a Client and Billing Agreement with patent counsel acceptable to the Company in substantially in the form attached as Exhibit F. During the term of the Client and Billing Agreement, Company will control and manage the preparation, prosecution and maintenance of the Patent Rights, with input from Penn. In the absence of or upon termination of a Client and Billing Agreement for any reason, control reverts to Penn with input from Company. For purposes of this Article 7, the word “maintenance” includes any interference negotiations, claims, or proceedings, in any forum, brought by Penn, Company, a third party, or the United States Patent and Trademark Office, and any requests by Penn or Company that the United States Patent and Trademark Office reexamine or reissue any patent in the Patent Rights.

7.2 Payment and Reimbursement. Company will reimburse Penn for (i) **** of all attorneys fees, expenses, official fees and all other charges accumulated **** to the Effective Date incident to the preparation, filing, prosecution and maintenance of the ****, and (ii) **** of all attorneys fees, expenses, official fees and all other charges accumulated prior to the Effective Date incident to the preparation, filing, prosecution and maintenance of the Patent Rights other than the ****, in the case of clause (ii) to the extent that such amounts have not already been reimbursed by third parties to Penn, provided that such reimbursement obligation with respect to clauses (i) and (ii) shall not exceed ****. The reimbursement obligation shall be paid in three equal installments, with the first installment payment becoming due thirty (30) days after the Effective Date and the two remaining payments becoming due on the first and second anniversary dates of the Effective Date. Thereafter, Company will either pay directly under a Client and Billing Agreement or reimburse Penn for **** documented attorneys fees, expenses, official fees and all other charges accumulated on or after the Effective Date incident to the preparation, filing, prosecution, and maintenance of the Patent Rights, within **** after Company’s receipt of invoices for such fees, expenses and

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

charges. Except during the term of a Client and Billing Agreement, Penn shall notify the Company promptly, and in advance to the extent reasonably practicable, of any upcoming expenditures in excess of **** in connection with the Patent Rights. Penn reserves the right to require the Company to provide a deposit in advance of incurring out of pocket patent expenses estimated by counsel to exceed ****. If Company fails to reimburse patent expenses under Paragraph 7.2, or provide a requested deposit with respect to a Patent Right, then Penn will be free at its discretion and expense to either abandon such applications or patents related to such Patent Right or to continue such preparation, prosecution and/or maintenance activities, and any patent rights associated with such patent action will be automatically excluded from the term “Patent Rights” hereunder, on a patent by patent or country by country basis, as applicable. Notwithstanding the foregoing, (i) Company shall have no obligation to pay any amounts pursuant to this Section 7.2 with respect to the GSK Licensed Patents; (ii) Company’s payment obligations pursuant to this Section 7.2 with respect to the **** shall be limited to **** of the amounts otherwise required to be paid; and (iii) **** payment obligations pursuant to this Section 7.2 shall be reduced**** by any patent expense reimbursement amounts received by Penn from **** of the Patent Rights.

8. INFRINGEMENT

8.1 Notice. Company and Penn will notify each other promptly of any infringement of the Patent Rights that may come to their attention. Company and Penn will consult each other in a timely manner concerning any appropriate response to the infringement.

8.2 Prosecution of Infringement. Company may prosecute any infringement of the Patent Rights at Company’s expense, including defending against any counterclaims or cross claims brought by any party against Company or Penn regarding the Patent Rights and defending against any claim that the Patent or Patent Rights are invalid in the course of any infringement action or in a declaratory judgment action. Penn reserves the right to intervene voluntarily and join Company in any such infringement litigation. If Penn chooses not to intervene voluntarily, but Penn is a necessary party to the action brought by Company, then Company may join Penn in the infringement litigation provided that Penn shall have the right to retain its own counsel, reasonably acceptable to Company, and Company will be responsible for **** of Penn’s reasonable litigation expenditures including any attorney’s fees, expenses, official fees and other charges incurred by Penn, even if there are no financial recoveries from the infringement action. Company will reimburse Penn within **** after receiving each invoice from Penn. If Company decides not to prosecute any infringement of the Patent Rights, then Penn may elect to prosecute such infringement independently of Company in Penn’s sole discretion, and at Penn’s expense.

8.3 Cooperation. In any litigation under this Article 8, either party, at the request and sole expense of the other party, will cooperate to the fullest extent reasonably possible. This Section 8.3 will not be construed to require either party to undertake any activities, including legal discovery, at the request of any third party, except as may be required by lawful process of a court of competent jurisdiction. If, however, either party is required to undertake any activity, including legal discovery, as a right of lawful process of a court of competent jurisdiction, then Company will pay all expenses incurred by Company and by Penn.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

8.4 Control of Litigation. Company controls any litigation or potential litigation involving the prosecution of infringement claims regarding the Patent Rights, including the selection of counsel, all with input from Penn. Notwithstanding the foregoing, Penn shall have the right to approve all decisions that would have a materially adverse affect on the validity, scope of patent claims, or enforceability of the Patent Rights. Company must not settle or compromise any such litigation in a manner that imposes any obligations or restrictions on Penn or grants any rights to the Patent Rights, other than any permitted sublicenses, without Penn’s prior written permission. In all instances in which Penn is a voluntary party, Penn reserves the right to select its own counsel, at its own expense. Penn shall have the right to control all litigation regarding the Patent Rights which is prosecuted by Penn independent of Company.

8.5 Recoveries from Litigation. Except as expressly provided in this Section 8.5, if Company prosecutes any infringement claims, Company will use the financial recoveries from such claims, if any, (a) first, to reimburse **** for its litigation expenditures; and (b) second, to retain any remainder but to treat the remainder as **** for the purpose of determining ****. If Company prosecutes any infringement claims with Penn joined as a voluntary party, then Company will use the financial recoveries from such claims, if any, (a) first, to reimburse **** and the **** for their respective litigation expenditures on a dollar-for-dollar basis; and (b) second, to retain any remainder but to ****. If Penn prosecutes any infringement claims independent of ****, then Penn will prosecute such infringement at **** expense and will retain any financial recoveries ****.

9. REPRESENTATIONS AND WARRANTIES; DISCLAIMER OF WARRANTIES

9.1 Mutual Representations and Warranties. Each party represents and warrants to the other party that Party that: (a) this Agreement is and shall be a legal and valid obligation binding upon such Party and enforceable in accordance with its terms; and (b) the execution, delivery and performance of this Agreement by such Party have been duly authorized by all necessary corporate and institutional action and do not and will not: (i) require any consent or approval of its stockholders or Trustees; or (ii) to such Party’s knowledge, violate any law, rule, regulation, order, writ, judgment, decree, determination or award of any court, governmental body or administrative or other agency having jurisdiction over such Party.

9.2 Representations and Warranties of Penn. Penn represents and warrants to Company that to the knowledge of the current staff of Penn’s Center for Technology (“CTT”):

(a) Penn has no commercial license agreements in effect as of the Effective Date, with third parties under the Patent Rights, other than to the US government;

(b) CTT has obtained from Dr. Wilson and the employees he has designated as being involved in the development of the Patent Rights an assignment of rights necessary to permit Penn to grant the Company the Licenses and make the representation set forth in Section 9.2(a) above;

(d) (i) there are no actual, pending actions, suits, claims, interferences, oppositions or governmental investigations involving Patent Rights; (ii) the Patent Rights are not subject

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

anywhere in the Territory to any pending re-examination, protest, opposition, interference or litigation proceeding.

9.3 Disclaimer. EXCEPT AS EXPRESSLY PROVIDED HEREIN, THE PENN PATENT RIGHTS, LICENSED PRODUCTS AND ANY OTHER TECHNOLOGY LICENSED UNDER THIS AGREEMENT ARE PROVIDED ON AN “AS IS” BASIS. PENN MAKES NO REPRESENTATIONS OR WARRANTIES, EXPRESS OR IMPLIED, INCLUDING BUT NOT LIMITED TO ANY WARRANTY OF ACCURACY, COMPLETENESS, PERFORMANCE, MERCHANTABILITY, FITNESS FOR A PARTICULAR PURPOSE, PROFITABILITY, COMMERCIAL UTILITY, NON-INFRINGEMENT OR TITLE.

10. LIMITATION OF LIABILITY

10.1 Limitation of Liability. PENN WILL NOT BE LIABLE TO COMPANY, ITS AFFILIATES, SUBLICENSEES, SUCCESSORS OR ASSIGNS, OR ANY THIRD PARTY WITH RESPECT TO ANY CLAIM: ARISING FROM COMPANY’S USE OF THE PENN PATENT RIGHTS, LICENSED PRODUCTS OR ANY OTHER TECHNOLOGY LICENSED UNDER THIS AGREEMENT; OR ARISING FROM THE DEVELOPMENT, TESTING, MANUFACTURE, USE OR SALE OF LICENSED PRODUCTS. PENN WILL NOT BE LIABLE TO COMPANY, ITS AFFILIATES, SUBLICENSEES, SUCCESSORS OR ASSIGNS, OR ANY THIRD PARTY FOR LOST PROFITS, BUSINESS INTERRUPTION, OR INDIRECT, SPECIAL, INCIDENTAL, OR CONSEQUENTIAL DAMAGES OF ANY KIND.

11. INDEMNIFICATION

11.1 Indemnification. Company will defend, indemnify, and hold harmless each Indemnified Party from and against any and all Liabilities with respect to an Indemnification Event. The term “Indemnified Party” means each of Penn and its trustees, officers, faculty, students, employees, contractors, and agents. The term “Liabilities” means all damages, awards, deficiencies, settlement amounts, defaults, assessments, fines, dues, penalties, costs, fees, liabilities, obligations, taxes, liens, losses, lost profits and expenses (including, but not limited to, court costs, interest and reasonable fees of attorneys, accountants and other experts) that are incurred by an Indemnified Party or awarded or otherwise required to be paid to third parties by an Indemnified Party. The term “Indemnification Event” means any Claim against one or more Indemnified Parties to the extent arising out of or resulting from: ****. The term “Claim” means any charges, complaints, actions, suits, proceedings, hearings, investigations, claims or demands.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

11.2 Reimbursement of Costs. Company will pay directly all Liabilities incurred for defense or negotiation of any Claim or will reimburse Penn for all documented Liabilities incident to the defense or negotiation of any Claim within **** after Company’s receipt of invoices for such fees, expenses and charges.

11.3 Control of Litigation. Company controls any litigation or potential litigation involving the defense of any Claim, including the selection of counsel, with input from Penn.

11.4 Other Provisions. Company will not settle or compromise any Claim giving rise to Liabilities in any manner that imposes any restrictions or obligations on Penn or grants any rights to the Licensed IP or the Licensed Products without Penn’s prior written consent. If Company fails or declines to assume the defense of any Claim within thirty (30) days after notice of the Claim, or fails to reimburse an Indemnified Party for any Liabilities pursuant to Sections 11.1 and 11.2 within the thirty (30) day time period set forth in Section 11.2, then Penn may assume the defense of such Claim for the account and at the risk of Company, and any Liabilities related to such Claim will be conclusively deemed a liability of Company. The indemnification rights of the Indemnified Parties under this Article 11 are in addition to all other rights that an Indemnified Party may have at law, in equity or otherwise.

12. INSURANCE

12.1 Coverages. Company will procure and maintain insurance policies for the following coverages with respect to personal injury, bodily injury and property damage arising out of Company’s performance under this Agreement: (a) during the Term, comprehensive general liability, including broad form and contractual liability, in a minimum amount of **** combined single limit per occurrence and in the aggregate; (b) prior to the commencement of clinical trials involving Licensed Products, clinical trials coverage in a minimum amount of **** combined single limit per occurrence and in the aggregate; and (c) prior to the Sale of the first Licensed Product, product liability coverage, in a minimum amount of **** combined single limit per occurrence and in the aggregate. Penn may review periodically the adequacy of the minimum amounts of insurance for each coverage required by this Section 12.1, ****. The required minimum amounts of insurance do not constitute a limitation on Company’s liability or indemnification obligations to Penn under this Agreement.

12.2 Other Requirements. The policies of insurance required by Section 12.1 will be issued by an insurance carrier with an A.M. Best rating of **** or better and will name Penn as an additional insured with respect to Company’s performance under this Agreement. Company will provide Penn with insurance certificates evidencing the required coverage within **** after the Effective Date and the commencement of each policy period and any renewal periods. Each certificate will provide that the insurance carrier will notify Penn in writing at least **** prior to the cancellation or material change in coverage.

13. ADDITIONAL PROVISIONS

13.1 Independent Contractors. The parties are independent contractors. Nothing contained in this Agreement is intended to create an agency, partnership or joint venture between the parties. At no time will either party make commitments or incur any charges or expenses for or on behalf of the other party.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

13.2 No Discrimination. Neither Penn nor Company will discriminate against any employee or applicant for employment because of race, color, sex, sexual or affectional preference, age, religion, national or ethnic origin, handicap, or veteran status.

13.3 Compliance with Laws. Company must comply with all prevailing laws, rules and regulations that apply to its activities or obligations under this Agreement. For example, Company will comply with applicable United States export laws and regulations. The transfer of certain technical data and commodities may require a license from the applicable agency of the United States government and/or written assurances by Company that Company will not export data or commodities to certain foreign countries without prior approval of the agency. Penn does not represent that no license is required, or that, if required, the license will issue.

13.4 Modification, Waiver & Remedies. This Agreement may only be modified by a written amendment that is executed by an authorized representative of each party. Any waiver must be express and in writing. No waiver by either party of a breach by the other party will constitute a waiver of any different or succeeding breach. Unless otherwise specified, all remedies are cumulative.

13.5 Assignment & Hypothecation. Neither Party may assign this Agreement or any part of it to any entity, other than an Affiliate, without the prior written consent of the other Party, such consent not to be unreasonably withheld or delayed. Notwithstanding the foregoing, Company shall be permitted to assign this Agreement, without the prior written consent of Penn, pursuant to a merger or sale of all or substantially all of the assets to which the Agreement relates to a company in the business of developing and commercializing pharmaceutical products that has, together with its affiliates, a market value or, in the case of a publicly traded company listed on a nationally recognized exchange, market capitalization, of at least $250,000,000. As part of any permitted assignment, the assigning party will require any assignee to agree in writing to be legally bound by this Agreement to the same extent as the assigning party. The non-assigning party will not unreasonably withhold or delay its consent, provided that: (a) at least thirty (30) days before the proposed transaction, the assigning party gives the non-assigning party written notice and such background information as may be reasonably necessary to enable the non-assigning party to give an informed consent; (b) the assignee agrees in writing to be legally bound by this Agreement; and (c) the assigning party provides the non-assigning party with a copy of assignee’s undertaking. Any permitted assignment will not relieve the assigning party of responsibility for performance of any obligation of the assigning party that has accrued at the time of the assignment. Further, in the event of assignment to an Affiliate, the assigning party will assume responsibility to ensure that Affiliate assignee complies fully with all of its obligations under the Agreement on an ongoing basis. Neither party will grant a security interest in the Licenses or this Agreement during the Term. Any prohibited assignment or security interest will be null and void.

13.6 Notices. Any notice or other required communication (each, a “Notice”) must be in writing, addressed to the party’s respective Notice Address listed on the signature page, and delivered: (a) personally; (b) by certified mail, postage prepaid, return receipt requested; (c) by recognized overnight courier service, charges prepaid; or (d) by facsimile. A Notice will be deemed received: if delivered personally, on the date of delivery; if mailed, five (5) days after deposit in the United States mail; if sent via courier, one (1) business day after deposit with the

CONFIDENTIAL TREATMENT REQUESTED

courier service; or if sent via facsimile, upon receipt of confirmation of transmission provided that a confirming copy of such Notice is sent by certified mail, postage prepaid, return receipt requested.

13.7 Severability & Reformation. If any provision of this Agreement is held to be invalid or unenforceable by a court of competent jurisdiction, then the remaining provisions of this Agreement will remain in full force and effect. Such invalid or unenforceable provision will be automatically revised to be a valid or enforceable provision that comes as close as permitted by law to the parties’ original intent.

13.8 Headings & Counterparts. The headings of the articles and sections included in this Agreement are inserted for convenience only and are not intended to affect the meaning or interpretation of this Agreement. This Agreement may be executed in several counterparts, all of which taken together will constitute the same instrument.

13.9 Governing Law. This Agreement will be governed in accordance with the laws of the Commonwealth of Pennsylvania, without giving effect to the conflict of law provisions of any jurisdiction.

13.10 Dispute Resolution. If a dispute arises between the parties concerning any right or duty under this Agreement, then the parties will confer, as soon as practicable, in an attempt to resolve the dispute. If the parties are unable to resolve the dispute amicably, then the parties will submit to the exclusive jurisdiction of, and venue in, the state and Federal courts located in the Eastern District of Pennsylvania with respect to all disputes arising under this Agreement.

13.11 Integration. This Agreement with its Exhibits and the Sponsored Research Agreement, the Equity Documents, and the Confidentiality Agreement, contain the entire agreement between the parties with respect to the Patent Rights and the License and supersede all other oral or written representations, statements, or agreements with respect to such subject matter, including but not limited to the Term Sheet.

CONFIDENTIAL TREATMENT REQUESTED

Each party has caused this Agreement to be executed by its duly authorized representative.


THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA		ReGenX, LLC

By:	/s/ Mike Cleare	By:	/s/ Kenneth T. Mills
Name: Mike Cleare		Name: Kenneth T. Mills
Title: Executive Director		Title: Chief Executive Officer
Center for Technology Transfer

Address:

Center for Technology Transfer

University of Pennsylvania

3160 Chestnut Street, Suite 200

Philadelphia, PA 19104-6283

Attention: Executive Director

Address: ReGenX, LLC

750 17^th Street, NW

Washington, DC 20006

Attention: Board of Managers

Required copy to:

University of Pennsylvania

Office of General Counsel

133 South 36th Street, Suite 300

Philadelphia, PA 19104-3246

Attention: General Counsel

CONFIDENTIAL TREATMENT REQUESTED

EXHIBIT INDEX


Exhibit A		Patents and Patent Applications in Patent Rights

Exhibit B		Patents and Patent Applications Subject to Certain Limitations
Exhibit B-1		****
Exhibit B-2		GSK Licensed Patents

Exhibit C		Form of Stock Purchase Agreement (or LLC unit purchase agreement)

Exhibit D		Form of Stockholders Agreement (or LLC unit-holders agreement)

Exhibit E		Format of Royalty Report

Exhibit F		Form of Patent Management Agreement

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Exhibit A

Patents and Patent Applications in Patent Rights


Penn #	Disclosure Title	US Patents	Foreign Patents
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


	****	****	****
****	****	****	****
****	****
****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Exhibit B-1

**** Licensed Patents


Penn #	Disclosure Title	US Patents	Foreign Patents
****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Exhibit B-2

GSK Licensed Patents


Penn #	Disclosure Title	US Patents	Foreign Patents
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

LOGO

CONFIDENTIAL TREATMENT REQUESTED

[NOTE THAT A PMA CAN BE USED ONLY DURING THE PERIOD WHERE THERE IS ONLY ONE LICENSEE TO THE PATENT RIGHTS IN ANY FIELD]

PATENT MANAGEMENT AGREEMENT

The Trustees of the University of Pennsylvania (“Penn”), a Pennsylvania non-profit corporation doing business at 3160 Chestnut Street, Suite 200, Philadelphia, PA 19104-6283; and ______ (“Company”), a corporation doing business at ____________________________________________, have entered into a License Agreement with respect to certain inventions which are the subject of the patent applications and patents listed in Appendix A hereto, including any continuations, divisions, extensions thereof, and any foreign counterpart patents, applications, or registrations (“Patent Rights”).

Penn has retained the services of _________________ (“Law Firm”), with offices at ____________, to prepare, file and prosecute the pending patent applications constituting the Patent Rights and to maintain the patents that issue thereon.

Penn, Company and Law Firm, intending to formalize their business relationships, agree as follows:

1.	Penn is the owner of the Patent Rights.

2.	Company is the licensee of Penn’s interest in the Patent Rights.

3.	Penn shall maintain an attorney-client relationship with Law Firm in furtherance of efforts to secure and maintain the Patent Rights.

Law Firm will interact directly with Company on all patent prosecution and patent maintenance matters related to the Patent Rights and will copy Penn on all correspondence related thereto. Company and Law Firm agree to use all reasonable efforts to notify Penn in writing at least thirty (30) days prior to the due date or deadline for any action which could adversely affect the pending status of any patent application within the Patent Rights, the maintenance of any granted patent within the Patent Rights, Penn’s right to file any continuing application or foreign counterpart application based on the Patent Rights, or the breadth of any claim within the Patent Rights. In any case, Company shall give Penn written notice of any final decision regarding the action to be taken or not to be taken on such matters prior to instructing Law Firm to implement the decision. Penn reserves the right to countermand any instruction given by Company to Law Firm.

5.	Law Firm’s legal services relating to the Patent Rights will be performed on behalf of Penn. Law Firm will invoice Penn for all such services. Company will reimburse Penn for all such services within thirty (30) days of Company’s receipt of Penn’s invoice for such services.

To clarify each party’s position with regard to prosecution and maintenance of the Patent Rights, Company will notify Law Firm in writing of all decisions to authorize the performance of any desired service(s), which shall be subject to Penn’s right to countermand, as provided in paragraph 4, above. In the event Penn countermands any decision or instruction of Company, such countermand shall be promptly communicated in writing to Law Firm.

7.	Penn may terminate this agreement at any time upon notice to Law Firm and Company.

This agreement represents the complete understanding of each of the undersigned parties as to the arrangements defined herein. Additions or deletions of dockets identified in Appendix A will become effective only by written addendum to Appendix A. All such additions or deletions of individual patents or applications filed in the US, or as foreign counterparts thereof are considered to be within the terms of this Patent Management Agreement.

CONFIDENTIAL TREATMENT REQUESTED

9.	Notices and copies of all correspondence relating to the Patent Rights should be sent to the following:


To PENN:						To COMPANY:

Center for Technology Transfer University of Pennsylvania 3160 Chestnut Street, Suite 200 Philadelphia, PA 19104-6283 Attn: Director, Intellectual Property To Law Firm:


ACCEPTED AND AGREED TO:

THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA						COMPANY

By:						By:

Name:						Name:

Title:						Title:

Date:						Date:

LAW FIRM

By:

Name:

Title:

Date:

CONFIDENTIAL TREATMENT REQUESTED

Appendix A

COMPANY LICENSED TECHNOLOGIES


PENN Docket Number		Title		Patent Numbers

EX-10.11

Exhibit 10.11

CONFIDENTIAL TREATMENT REQUESTED

UNIVERSITY of PENNSYLVANIA

Second Amendment to License Agreement

This Second Amendment to License Agreement (this “Second Amendment”) effective as of September 9, 2014 (this “Second Amendment Effective Date”), is made by and between The Trustees of the University of Pennsylvania (“Penn”) and ReGenX Biosciences, LLC (“Company”) (collectively, the “Parties”) and amends the License Agreement between the Parties, which was effective as of February 24, 2009, as subsequently amended by a First Amendment dated March 6, 2009 (the “License Agreement”). All capitalized terms used but not defined herein shall have the meaning set forth in the License Agreement.

BACKGROUND

The License Agreement relates to certain intellectual property developed by Dr. James M. Wilson of Penn’s Perelman School of Medicine, which intellectual property is the subject of patents or patent applications (the “Penn Dockets”).

WHEREAS, the Company has elected to exercise certain of its option rights pursuant to the sponsored research agreement by and between the Parties effective as of February 24, 2009 and as subsequently amended with respect to certain Penn patentable inventions and patent rights. Penn and Company have reached agreement on terms to extend such additional Penn patent rights to Company as set forth herein;

WHEREAS, The Parties wish to amend the License Agreement to reflect these changes.

Now, therefore, in consideration of the promises and covenants contained in this Second Amendment and for other good and valuable consideration the receipt and sufficiency of which is hereby acknowledged and intending to be legally bound, the Parties agree as follows:

1) Exhibit A of the License Agreement is hereby amended and restated in its entirety by Exhibit A to this Second Amendment to add the additional Patent Rights and Know-How designated therein as “Second Amendment Patent Rights and Know-How”. On the Second Amendment Effective Date, Company will reimburse Penn for all patent prosecution and maintenance expenses associated with the Second Amendment Patent Rights that have not previously been reimbursed by Company.

2) The following definitions in Section 1.2 of the License Agreement shall be amended and restated in their entirety as follows:

“Background Know-How” means all Know-How that (a) was developed by Dr. Wilson, or other Penn researchers working under his direct supervision, at Penn, and (b) is related to the adeno associated virus technology platform discovered by Dr. Wilson at Penn prior to February 24, 2009, or is related to the adeno associated virus technology platform discovered by Dr. Wilson at Penn after February 24, 2009 pursuant to the SRA, arid (c) is owned by Penn and available for licensing,

CONFIDENTIAL TREATMENT REQUESTED

and (d) is necessary or useful for the practice of the Patent Rights in connection with the manufacture, use, sale, importation and/or other exploitation of the Licensed Products or the practice of the Licensed Processes in the Territory in the Field of Use, including, without, limitation, any Know-How necessary for the Company to manufacture or have manufactured the materials produced by the Penn Vector Core or Dr. Wilson’s lab at Penn.

“Field of Use” means any and all fields of use, except with respect to the Patent Rights listed in Exhibit A, Part 2 and all related Know-How and data, for which the Field of Use is limited to viral vector mediated gene therapy.

“Know-How” means any and all information, discoveries, software, methods, works of authorship, techniques, formulae, data, biological materials, processes, unpatentable inventions and other know-how, not including the Patent Rights, developed prior to the Effective Date or under the SRA.

“Patent Rights” means (i) all of Penn’s patent rights represented by or issuing from the United States patents and patent applications (including provisional patent applications) listed in Exhibit A, as well as any continuations, continuations-in-part (to the extent the inventions claimed or disclosed in any such patent or patent applications are directed to subject matter specifically described in the patent or patent applications listed in Exhibit A), divisionals, reexaminations, renewals, re-issues, substitutions, extensions and foreign counterparts of any of the foregoing, and all other patents and patent applications that claim priority from or have common priority with any of the foregoing patents and patent applications, (to the extent the inventions claimed or disclosed in any such patent or patent applications are directed to subject matter specifically described in the patent or patent applications listed in Exhibit A) and including any patents issuing from any of the foregoing; and (ii) all patentable inventions (to the extent they are or become available for license) that (a) were discovered by Dr. Wilson, or other Penn researchers working under his direct supervision, at Penn prior to the Second Amendment Effective Date, and (b) are related to the adeno associated virus technology platform discovered by Dr. Wilson at Penn prior to the Effective Date or under the SRA, and (c) are owned by Penn and available for licensing.

“SRA” means the each of: 1) the Sponsored Research Agreement between the Company and Penn effective as of February 24, 2009, as subsequently amended and 2) the Sponsored Research Agreement between the Company and Penn effective as of November 1, 2013 and any future amendments thereof

3) Section 1.5 (a) of the License Agreement shall be amended and restated in its entirety as follows:

“1.5(a) Within **** after Company enters into a sublicense agreement, Company will deliver to Penn a complete and accurate copy of the entire executed sublicense agreement written in the English language.”

****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

4) Section 2.3 of the License Agreement shall be amended and restated in its entirety as follows:

“Company is deemed to have fully satisfied all of its obligations under this Section 2.”

5) Section 3.2 is hereby amended to add the following language at the end:

“Notwithstanding anything herein to the contrary, in no event shall Penn receive a royalty of less than **** (****) of Net Sales of Licensed Pharmaceutical Products sold by ****, where such Licensed Pharmaceuticals Products are not also covered by GSK Licensed Patents. For clarity, **** include ****.”

6) Section 6.1 of the License Agreement shall be amended and restated in its entirety as follows:

“6.1 Term. This Agreement will commence on Effective Date and end upon the expiration of the Royalty Term (the “Term”). Earned royalties pursuant to Section 3.4 shall only be payable hereunder during the Royalty Term. Upon expiration of the Agreement, Company’s license under Section 1.1(c) will become perpetual, irrevocable, royalty-free, transferable, sublicensable, and fully paid-up.”

7) Section 6.3 of the License Agreement shall be amended and restated in its entirety as follows:

“6.3 Early Termination by Penn. Penn may terminate this Agreement if: (a) Company is more than thirty (30) days late in paying to Penn any amounts owed under this Agreement and does not pay Penn in full within thirty (30) days after receipt of written notice indicating such default and demanding payment, including accrued interest (a “Payment Default”); (b) other than a Payment Default, Company or its Affiliate fails to achieve a diligence event on or before the applicable completion date or otherwise breaches this Agreement and does not cure such failure or breach within sixty (60) days after written notice of the breach; or (c) Company or its Affiliate experiences a Trigger Event.”

8) Section 6.4 of the License Agreement shall be amended and restated in its entirety as follows:

“6.4 Trigger Event. The term “Trigger Event” means any of the following: (a) if Company or its Affiliate (i) becomes insolvent, bankrupt or generally fails to pay its debts as such debts become due, (ii) is adjudicated insolvent or bankrupt, (iii) admits in writing its inability to pay its debts, (iv) suffers the appointment of a custodian, receiver or trustee for it or its property and, if

****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

appointed without its consent, not discharged within ****, (v) makes an assignment for the benefit of creditors, or (vi) suffers proceedings being instituted against it under any law related to bankruptcy, insolvency, liquidation or the reorganization, readjustment or release of debtors and, if contested by it, not dismissed or stayed within ****; (b) the institution or commencement by Company or its Affiliate of any proceeding under any law related to bankruptcy, insolvency, liquidation or the reorganization, readjustment or release of debtors; (c) the entering of any order for relief relating to any of the proceedings described in Section 6.4(a) or (b) above; (d) the calling by Company or its Affiliate of a meeting of its creditors with a view to arranging a composition or adjustment of its debts; (e) the act or failure to act by Company or its Affiliate indicating its consent to, approval of or acquiescence in any of the proceedings described in Section 6.4(b) (d) above; or (f) the commencement by Company of any action against Penn, including an action for declaratory judgment, to declare or render invalid or unenforceable the Patent Rights, or any claim thereof.”

9) Section 6.5 of the License Agreement shall be amended and restated in its entirety as follows:

“6.5 Effect of Termination. Upon the early termination of this Agreement pursuant to Section 6.2 or 6.3: (a) the Licenses shall terminate; (b) Company and all its Affiliates will cease all making, having made, using, importing, offering for sale and selling all Licensed Products and practicing the Licensed Processes; (c) Company shall assign and Penn will accept the assignment of all sublicenses granted to sublicensees to the extent related solely to the Patent Rights or the Licensed Products; provided, however, that any such sublicensee (i) is not in breach of any provision of this Agreement or the applicable sublicense agreement as of the effective date of such termination and (ii) upon request by Penn, agrees in writing that such sublicensee will perform all obligations of Company under this Agreement that are applicable to the sublicensed rights; (d) Company will pay to Penn all amounts, including accrued interest, owed to Penn under this Agreement and any Sponsored Research Agreement related to the Patent Rights, through the date of termination, including royalties on Licensed Products invoiced or shipped through the date of termination when such payments are received, whether or not payment is received prior to termination; (e) each Party will, at the other Party’s request, return to such Party all confidential information of such Party; and (0 except as provided in Section 6.6, all rights and duties of Penn and the Company under this Agreement immediately terminate without further action required by either Penn or Company.”

10) The second sentence of Section 13.5 is amended and restated as follows:

****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Notwithstanding the foregoing, provided that Company is not in breach of any provisions of this Agreement, Company shalt be permitted to assign this Agreement, without the prior written consent of Penn, pursuant to a merger or sale of all or substantially all of the assets to which the Agreement relates to a company in the business of developing and commercializing pharmaceutical products that has, together with its affiliates, a market value or, in the case of a publicly traded company listed on a nationally recognized exchange, market capitalization, of at least $250,000,000.

11. For clarity, Exhibit A now includes, in Part 2, all available intellectual property and technology owned and controlled by Penn and created at Penn in the laboratory of Dr. James M. Wilson, MA., Ph.D. through the Second Amendment Effective Date in the disease indications of familial hypercholesterolemia (FH) and ornithine transcarbarnylase deficiency (OTC), including any related data and know-how.

12. This Second Amendment, together with the License Agreement and First Amendment, constitute the entire agreement between the Parties. All other terms and provisions of the License Agreement, except as expressly amended by this Second Amendment, remain in full force and effect.

13. This Second Amendment may be executed in two or more counterparts, each of which shall be deemed an original and together shall be deemed one and the same instrument.

IN WITNESS WHEREOF, the Parties, intending to be legally bound, have caused this Second Amendment to be executed by their duly authorized representatives.


THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

By:		/s/ John S. Swartley, Ph.D.
Name:		John S. Swartley, Ph.D.
Title:		Associate Vice President for Research
		Executive Director, PCI
Date:		September 9, 2014

REGENX BIOSCIENCES, LLC

By:		/s/ Kenneth T. Mills
Name:		Kenneth T. Mills
Title:		President and CEO
Date:		September 9, 2014

CONFIDENTIAL TREATMENT REQUESTED

Exhibit A

Patents and Patent Applications in the Patent Rights

Part 1; No Field of Use Limitation


Penn #	Disclosure Title	US Patents	Foreign Patents
****	****	********	********
****	****	********	********
****	****	********	********
****	****	********	********

****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


	****	********	********
****	****	********	********
****	****	********	********
****	****	****	****
****	****	****	****
****	****	****	****

****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****
****	****	****	****

****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


Docket V5085	Combination method to generate novel and/or hybrid coding sequences within the defined domains
Serial No	Patent No	App Type	File Date	Country	Issue Date
****		****	****	****
****		****	****	****

Docket V5131	A domain of the adeno-associated virus stereotype 6 capsid that confers transduction of conducting airway epithelium
Serial No	Patent No	App Type	File Date	Country	Issue Date
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****

Docket X5836	The receptor for AAV9 and its modification in vivo to improve lung gene therapy
Serial No	Patent No	App Type	File Date	Country	Issue Date
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****
****		****	****	****

****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


Docket Z6622	Treatment of MPS1
Serial No	Patent No	App Type	File Date	Country	Issue Date
****		****	****	****
****		****	****	****

Exhibit A, Part 2

Field of Use limited to viral vector mediated delivery of gene therapy product


Docket 14-7025	Improved AAV-LDLR for treating human disease
Serial No	Patent No	App Type	File Date	Country	Issue Date
****		****	****	****
****		****	****	****

Docket 14-7037	AAV OTC for treating human disease
Serial No	Patent No	App Type	File Date	Country	Issue Date
****		****	****	****

Penn / Wilson Lab Know-How for the Familial Hypercholesterolemia and Onithine Transcarbamylase Deficiency (OTC) Programs

FH Know-How (associated with ****)

****

****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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OTC Know-How (associated with ****)

****

****	CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

EX-10.12

Exhibit 10.12

CONFIDENTIAL TREATMENT REQUESTED

Execution Version

Confidential

LICENSE AGREEMENT

This LICENSE AGREEMENT (“Agreement”) is entered into as of March 6, 2009 (“Effective Date”) by and between ReGenX, a limited liability company organized under the laws of the State of Delaware, with offices at 750 17th Street, NW, Suite 1100, Washington, DC 20006 (“ReGenX”) and SmithKline Beecham Corporation, a Pennsylvania corporation doing business as GlaxoSmithKline, with offices at One Franklin Plaza, 200 North 16th Street, Philadelphia, Pennsylvania, 19102 (“GSK”). ReGenX and GSK are hereinafter referred to individually as a “Party” and collectively as the “Parties.”

WHEREAS, pursuant to a license agreement by and between GSK and the University of Pennsylvania (“Penn”), dated as of May 31, 2002, as amended from time to time (the “Penn License Agreement”) GSK has exclusive rights under certain Penn Patent Rights (as defined herein) pertaining to various recombinant adeno-associated virus vectors; and

WHEREAS, ReGenX desires to obtain an exclusive sublicense from GSK under the Penn Patent Rights (as defined herein).

NOW, THEREFORE, in consideration of the promises and covenants contained in this Agreement, and intending to be legally bound, the Parties hereby agree as follows:

ARTICLE 1: DEFINITIONS

1.1 “Affiliate” means any legal entity directly or indirectly controlling, controlled by or under common control with a Party or sublicensee. For purposes of this Agreement, “control” means the direct or indirect ownership of more than fifty percent (50%) of the outstanding voting securities of a legal entity, or the right to receive more than fifty percent (50%) of the profits or earnings of a legal entity, or the right to control the policy decisions of a legal entity.

1.2 “Calendar Quarter” means each three (3) month period or any portion thereof, beginning on January 1, April 1, July 1 and October 1.

1.3 “Confidential Information” means and includes all technical information, inventions, developments, discoveries, software, know-how, methods, techniques, formulae, animate and inanimate materials, data, processes and other proprietary ideas, whether or not patentable or copyrightable, of either Party (a) that is identified as confidential or proprietary at the time of disclosure; or (b) whose confidential or proprietary status would be reasonably apparent under the circumstances. Notwithstanding the foregoing, Confidential Information shall not include the following:

1.3.1

information that is lawfully known to the receiving Party prior to the time of disclosure or independently developed by the receiving Party without use of the disclosing Party’s Confidential Information, in each case, to the extent evidenced by written records;

CONFIDENTIAL TREATMENT REQUESTED

1.3.2

information disclosed to a Party by a Third Party that has a right to make such disclosure;

1.3.3

information that becomes patented, published or otherwise part of the public domain as a result of acts by the Party owning such information or a Third Party obtaining such information as a matter of right; or

1.3.4

information that is required to be disclosed by order of a governmental authority or a court of competent jurisdiction; provided that the Receiving Party (as defined below) must use reasonable efforts to obtain confidential treatment of such information by the agency or court and notifies the Disclosing Party (as defined below) in the event that such information is required to be disclosed pursuant to Section 5.2.

1.4 “Domain Antibody” ****.

1.5 “GSK Collaborators” means entities (a) with which GSK has an active drug research and development agreement; and (b) from which GSK has retained substantial commercial rights to products derived from the development of drug candidates in the RNAi or antisense field.

1.6 “Licensed Product” means (a) products which are made, made for, used, sold or imported by ReGenX, its Affiliates and any of its or their sublicensees, the manufacture, use, sale or import of which, in the absence of the license granted pursuant to this Agreement, would infringe at least one Valid Claim in the country of manufacture, use, sale or import, including products manufactured by a process which would infringe at least one Valid Claim in the country of manufacture, use, sale or import; or (b) services sold by ReGenX, its Affiliates or its or their sublicensees which, in the absence of the licenses granted pursuant to this Agreement, would infringe at least one Valid Claim of the Penn Patent Rights in the country of sale.

1.7 “Muscular Dystrophy” ****.

1.8 “Net Sales” means the gross receipts from sales of a Licensed Product by ReGenX and/or its Affiliates and/or its or their sublicensees to Third Parties under this Agreement less deductions for ****.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

1.9 “Penn Patent Rights” means all United States patents and patent applications, re-examination certificates thereof, and their foreign counterparts and extensions, continuations, divisionals, and re-issue applications listed in Exhibit 1 which cover adeno-associated vectors.

1.10 “ReGenX Materials” means those vector materials that are (a) the subject of a claim within the Penn Patent Rights; or (b) made using a process that is the subject of a claim within the Penn Patent Rights, in each case, that ReGenX or its Affiliates offer for commercial sale from time-to-time to Third Parties and that are intended and licensed for research uses only, but excluding any research uses in humans.

1.11 “Retained Rights” shall have the meaning set forth in Section 2.1

1.12 “Third Party” means any person or entity other than a Party to this Agreement or Affiliates of a Party to this Agreement.

1.13 “Valid Claim” means a claim of an issued and unexpired patent included within the Penn Patent Rights, which has not lapsed, been abandoned, been held revoked or deemed unenforceable or invalid by a non-appealable decision or an appealable decision from which no appeal was taken within the time allowed for such appeal of a court or other governmental agency of competent jurisdiction.

ARTICLE 2: LICENSE GRANT

2.1 License Grant. GSK hereby grants to ReGenX an exclusive (except as provided in Section 2.2 and in this Section 2.1), royalty-bearing, worldwide right and license, with the right to grant sublicenses, under the Penn Patent Rights to make, have made, use, import, sell and offer for sale Licensed Products anywhere in the world, including, for the avoidance of doubt, the right to conduct research and development under the Penn Patent Rights. No other rights are granted. Notwithstanding the foregoing, GSK retains the following rights (individually and collectively, the “Retained Rights”) with respect to the Penn Patent Rights:

2.1.1

An exclusive (even as to ReGenX), fully sublicensable right under the Penn Patent Rights to make, have made, use, sell, offer to sell and import Domain Antibodies which are expressed by an adeno-associated vector that is the subject of at least one Valid Claim or a claim in a pending patent application within the Penn Patent Rights.

2.1.2

A non-exclusive right, sublicensable only to GSK’s Affiliates and GSK Collaborators, under the Penn Patent Rights to make, have made, use, sell, offer to sell and import products that deliver RNA interference and antisense drugs using an adeno-associated vector that is the subject of at least one Valid Claim or a claim in a pending patent application within the Penn Patent Rights. GSK will provide to ReGenX reasonable written notice of any sublicense it grants to any GSK Collaborator pursuant to the Retained Rights under this Section 2.1.2, and such notice shall include the field for which such sublicense has been granted.

CONFIDENTIAL TREATMENT REQUESTED

2.1.3

A non-exclusive right to use the Penn Patent Rights solely for internal (except as provided for in this Section 2.1.3), non-commercial research purposes and for GSK’s discovery research efforts with non-profit organizations. Such right shall be sublicensable only to (a) GSK’s Affiliates; and (b) GSK Collaborators; provided, however, that for GSK Collaborators, GSK shall provide written notice to ReGenX of such sublicense and such sublicense shall be limited only to the field of research identified for such collaboration; provided that ReGenX acknowledges that fee-for-service work with Third Parties will not be considered a sublicense. In addition, ReGenX acknowledges that GSK has granted a sublicense to the Penn Patent Rights solely for non-commercial research purposes to one (1) Third Party sublicensee who is not an Affiliate of GSK or a GSK Collaborator. Pursuant to the sublicense agreement between GSK and such Third Party sublicensee, such sublicensee is permitted to sublicense the rights granted pursuant to such sublicense to its Affiliates and contract research organizations who are performing specific research on behalf of such sublicensee. Upon termination or expiration of the sublicense agreement with such Third Party sublicensee, GSK shall have no right to grant any further sublicenses under this Section 2.1.3 except to GSK Affiliates and GSK Collaborators, as set forth herein.

2.1.4

In order to comply with potential and existing Third Party commitments existing as of the Effective Date, an exclusive, worldwide, sublicensable right under (a) the Penn Patent Rights which cover the rAAV serotype 8, to make, have made, use, sell, offer for sale and import Licensed Products for the treatment of all forms of hemophilia B; or (b) the Penn Patent Rights which cover rAAV serotype 9, to make, have made, use, sell, offer for sale and import Licensed Products for the treatment of (i) all forms of Muscular Dystrophy; (ii) congestive heart failure suffered by Muscular Dystrophy patients; (iii) all forms of cardiovascular disease by delivery of genes encoding I-lc and Serca2a and creatine kinase and (iv) amyotrophic lateral sclerosis, acid maltase deficiency and spinal muscular atrophies; provided, however, that with respect to category (iv), GSK agrees to use its commercially reasonable efforts to provide these rights to ReGenX within forty-five (45) days of the Effective Date; provided further, that GSK shall be under no obligation to make payments or otherwise incur financial obligations in order to provide such rights to ReGenX. In the event that GSK’s existing commitments to any such Third Party cease, GSK’s Retained Rights under this Section 2.1.4 shall be exclusively licensed to ReGenX.

2.1.5

In order to comply with Third Party commitments existing as of the Effective Date, a non-exclusive, sublicensable right to make, have made, and use all of the various serotypes of any adeno-associated vector that is the subject of at least one claim in the Penn Patent Rights solely for non-commercial research in the areas of Muscular Dystrophy, hemophilia B, congestive heart failure suffered by Muscular Dystrophy patients, and other cardiovascular disease. In no event shall the Retained Rights granted under this Section 2.1.5 be used for any commercial purposes. In the event that GSK’s existing commitments to any such Third Party cease, GSK’s Retained Rights under this Section 2.1.5 shall be exclusively

CONFIDENTIAL TREATMENT REQUESTED

licensed to ReGenX, subject to GSK’s Retained Rights under Section 2.1.1, 2.1.2 and 2.1.3.

2.2 Penn Retained Rights. Notwithstanding anything to the contrary in Section 2.1, Penn may use and permit other non-profit organizations to use the Penn Patent Rights for educational and non-commercial research purposes only.

2.3 Government Rights. ReGenX acknowledges that, pursuant to Public Laws 96-517, 97-256 and 98-620, codified at 35 U.S.C. §§ 200-212, the United States government retains certain rights in intellectual property funded in whole or part under any contract, grant or similar agreement with a federal agency. Pursuant to these laws, the government may impose certain requirements regarding such intellectual property, including but not limited to the requirement that products resulting from such intellectual property sold in the United States must be substantially manufactured in the United States. The license grant hereunder is expressly subject to all applicable United States government rights as provided in the above-mentioned laws and any regulations issued under those laws, as those laws or regulations may be amended from time to time.

2.4 Sublicensing. The right to sublicense granted to ReGenX under this Agreement is subject to the following conditions:

2.4.1

In each such sublicense, ReGenX must require that the sublicensee be subject to the material terms and conditions of the licenses granted to ReGenX under this Agreement.

2.4.2

Within **** after ReGenX enters into any sublicense, ReGenX must send to GSK a complete copy of the sublicense written in the English language in order for GSK to send such sublicense to Penn, pursuant to the terms of the Penn License Agreement.

2.4.3

In the event ReGenX enters into sublicenses, ****

2.5 License to GSK. ReGenX hereby grants to GSK a non-exclusive, worldwide, royalty-free license to use any patentable modifications or improvements developed by ReGenX to any vector which is the subject of a claim within the Penn Patent Rights consummate in scope to the Retained Rights set forth in Sections 2.1.1 through 2.1.3 above (“Licensed Back Improvements”). Such license shall be sublicensable solely as described and within the scope of the Retained Rights under Sections 2.1.1 through 2.1.3. ReGenX shall provide reasonable notice to GSK upon the filing of any patent application covering such Licensed Back Improvements.

2.6 Ownership. Subject to any licenses expressly set forth herein, ReGenX shall own and retain all right, title and interest (including all intellectual property rights) in all technical information, inventions (whether or not patentable), developments, discoveries, software, know-

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

how, methods, techniques, formulae, animate and inanimate materials, data, processes and other proprietary information, including without limitation the Licensed Products, that are created, discovered, conceived or reduced to practice by ReGenX, its Affiliates and Third Party collaborators using the rights granted to ReGenX hereunder.

ARTICLE 3: CONSIDERATION

3.1 ReGenX Equity. In consideration for the licenses granted to ReGenX hereunder, upon the Effective Date, ReGenX will issue to GSK units of limited liability company interest in ReGenX equal to 19.9% of the outstanding units of limited liability company interest in ReGenX.

3.2 Milestone Fees. ReGenX will pay to GSK the following milestone payments the first time a Licensed Product that fits within Category la or lb, as listed in Section 3.3, achieves such milestone event:


Milestone		Exclusive License
Phase I Entry		****
Phase III Entry		****
Product Approval		****

****

3.3 Royalties. In further consideration of the license granted to ReGenX, ReGenX shall pay to GSK the following royalty based upon Net Sales of Licensed Products, subject always to the reductions in royalty rates set forth in Sections 3.3.4 and 3.3.5:

LICENSED

PRODUCTS

Royalty Percentage

Cumulative Annual
NET SALES
(Million $)

1a. Novel Vector

****

Up to ****;

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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	****	In excess of ** up to ; and In excess of **.
1b. i) Refinement of an existing vector or ii) a Novel Vector made by ReGenX through the material use of technology recited in a Valid Claim	****	Up to **; In excess of up to ; and In excess of **.
2. Licensed Products that would otherwise infringe a Valid Claim covering a therapeutic use of a vector or compound.	****	Up to **; In excess of up to ; and In excess of **.
3. Manufacturing technology that would otherwise infringe a Valid Claim covering a method or process of manufacture	****	Up to **; In excess of up to ; and In excess of **.

3.3.1

Description of Categories:

3.3.1.1

To meet the requirements of the term “Novel Vector” (as used in Categories la and lb(ii)), there must be neither any dominating Third Party patent, including any Penn-owned patent rights other than those licensed under this Agreement with respect to the vector per se (i.e., no Third Party patent or Penn-owned patent rights beyond those licensed under this Agreement is required in order to make, have made, use, import, offer for sale or sell the vector for the higher royalty level to apply). Licenses from Penn or GSK to ReGenX for genes used, promoters used other than those which are part of the vector as described in Penn Patent Rights and the like in the vector will not affect the royalty pursuant to this provision. If any dominating Third Party patent or any Penn-owned patent other than those licensed under this Agreement issues at any time during the term of this Agreement with respect to a vector licensed hereunder, then the royalty level will immediately drop to the “Refinement” level (Category lb above) for any Licensed Product containing such vector.

3.3.1.2

As used in Category 2 of the Table in Section 3.3, a “therapeutic use” shall mean use in the prevention and/or treatment of a disease in a patient. For the

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CONFIDENTIAL TREATMENT REQUESTED

avoidance of doubt, ReGenX shall have no obligation under Section 3.3 or under any other provision of this Agreement to pay any royalties on any “reach-through” basis for any reason whatsoever, including without limitation, on the basis of the use by ReGenX of any subject matter recited in a Valid Claim in the development, selection or advancement of any Licensed Product, except only as may be expressly provided under Category lb(ii) in Section 3.3 above.

3.3.1.3

In the event that any Licensed Product does not fall into any of the Categories 1-3 above, ReGenX shall pay to GSK a royalty of **** based upon Net Sales of such Licensed Product, subject always to the reductions in royalty rates set forth in Sections 3.3.4 and 3.3.5, and subject always to the terms and conditions of this Section 3.3.

3.3.2

One Royalty: For any single Licensed Product, ****. In addition, the **** royalty shall be subject to the **** provided below.

3.3.4

Valid Claims: Except as set forth below, royalties will only apply to Net Sales of Licensed Products that are the subject of at least one Valid Claim of any issued patent in the Penn Patent Rights. ****.

3.3.5

Third Party Royalties Stacking Provision: If, in connection with the manufacture, use or commercialization of a given Licensed Product, ReGenX is obligated to pay royalties to GSK and any Third Parties that, ****, then the royalty owed to GSK for that Licensed Product will be reduced by an amount calculated as follows:

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CONFIDENTIAL TREATMENT REQUESTED

STACKING ROYALTY CALCULATIONS

R = (C*(A/(A+B)))

Where

R = Reduction of GSK royalty.

A = Unreduced GSK royalty,

B = sum of all Third Party royalties,

C = increment of projected total royalty above ****.

Example Calculation:


assume:		i) all Third Party royalties = ****%
		ii) unreduced GSK royalty = ****%
		iii) projected total royalty = ****%


		R = (**-*)(**/( + **))
		R = (*********)
		R = ****
		GSK Stacked Royalty = **- = **%

Notwithstanding the foregoing, ReGenX will pay to GSK no less than **** of the royalties that ReGenX would otherwise pay to GSK if there were no royalties due to Third Parties.

3.3.6

Termination of Obligation to Pay Royalty: Subject to Section 3.3.4, ReGenX’s obligation hereunder for payment of a royalty on the Net Sales of Licensed Products in a given country will end on a country by country basis when there is no Valid Claim in that country claiming the Licensed Product.

3.4 Sublicense Fees. ReGenX shall pay GSK a percentage of any sublicense fees (****) received by ReGenX for the Penn Patent Rights from any sublicensee; ****. The applicable percentage due to GSK for each sublicense shall be:

**** - if sublicensed on or before the **** of the Effective Date;

**** - if sublicensed on or before the **** of the Effective Date, but after the **** of the Effective Date; and

**** - if sublicensed after the **** of the Effective Date.

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CONFIDENTIAL TREATMENT REQUESTED

Notwithstanding anything to the contrary herein, in the event ReGenX receives non-cash consideration for any sublicense granted to a Third Party hereunder, and such non-cash consideration is liquidated ****, then, with respect to the proceeds resulting from the liquidation of such non-cash consideration, ReGenX shall pay to GSK the percentage of sublicense fees that **** if such payment had been received in cash.

3.5 Reports and Records.

3.5.1

ReGenX must deliver to GSK within **** after the end of each Calendar Quarter after the first commercial sale of a Licensed Product a report setting forth the calculation of the royalties due to GSK for such Calendar Quarter, including, without limitation:

3.5.1.1

Number of Licensed Products included within Net Sales, listed by country;

3.5.1.2

Gross consideration for Net Sales of Licensed Product, including all amounts invoiced, billed, or received;

3.5.1.3

Qualifying costs to be excluded from the gross consideration, as defined in Section 1.7, listed by category of cost;

3.5.1.4

Net Sales of Licensed Products listed by country; and

3.5.1.5

Royalties owed to GSK, listed by category, including without limitation earned and sublicensee-derived categories.

3.5.2

ReGenX shall pay the royalties due under Section 3.3 and other payments due under Section 3.4 within **** following the last day of the Calendar Quarter in which the royalties accrue or the other consideration is received. ReGenX shall send the royalty payments along with the report described in Section 3.5.1.

3.5.3

ReGenX shall maintain and require its Affiliates and its or their sublicensees to maintain, complete and accurate books and records which enable the royalties, fees, and payments payable under this Agreement to be verified. The records for each Calendar Quarter must be maintained for **** after the submission of each report under Article 3. If any such verification determines that ReGenX has underpaid royalties by **** or more of the money actually owed, ReGenX shall pay the costs and expenses of GSK and its accountants in connection with their review or audit. Such audit or review shall be made by an independent certified public accountant, and only upon reasonable prior written notice to ReGenX (not less than **** notice) and during ReGenX’s normal working hours; and shall not be made more than once in any given year.

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CONFIDENTIAL TREATMENT REQUESTED

3.6 Currency, Place of Payment, Interest.

3.6.1

All dollar amounts referred to in this Agreement are expressed in United States dollars. All payments to GSK under this Agreement must be made in United States dollars.

3.6.2

If ReGenX receives revenues from the sale of Licensed Product in currency other than United States dollars, revenues shall be converted into United States dollars at the conversion rate used by GSK in producing its quarterly and annual accounts, as confirmed by GSK’s auditors. If government regulations prevent remittances from a foreign country with respect to sale made in that country, the obligation to pay royalties on the sale in that country shall be suspended until such remittances are possible.

3.6.3

Notwithstanding anything herein to the contrary, in the event that any amounts due under the terms of this Agreement may be passed on by GSK to Penn or otherwise are ultimately payable to Penn under the terms of this Agreement, the Penn License Agreement or any step-through or other agreement entered into by ReGenX and Penn, ReGenX shall have the right to make such payment directly to Penn and deduct any such amounts from fees due to GSK (as applicable) hereunder.

3.7 Favored Nation Pricing to GSK. During the term of this Agreement, ReGenX agrees to provide ReGenX Materials to GSK at a price that is equal to or lower than the price **** for the same (in substance and amount and on substantially the same terms) products at the time of such GSK purchase. ****.

ARTICLE 4: DILIGENCE

4.1 ReGenX shall use commercially reasonable efforts to develop, market, promote and sell a Licensed Product ****. Without limiting the foregoing, ReGenX shall be deemed to have satisfactorily discharged its diligence obligations hereunder as long as ReGenX and/or its Affiliates and/or sublicensees are diligently pursuing the development and/or commercialization of ****.

4.2 Within **** after the Effective Date and within **** of each December 1 thereafter, ReGenX shall provide GSK with written progress reports, setting forth in such detail as GSK may reasonably request, the progress of the development, evaluation, testing and commercialization of each Licensed Product. ReGenX acknowledges that GSK is required to notify Penn within **** of the first commercial sale by ReGenX, its Affiliates, or its or their sublicensees of each Licensed Product. Such a report (“Development Progress

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CONFIDENTIAL TREATMENT REQUESTED

Report”), setting forth the current stage of development of Licensed Products, shall include, without limitation:

4.2.1

Date of Development Progress Report and time covered by such report;

4.2.2

Major activities and accomplishments completed by ReGenX, its Affiliates, and its or their sublicensees relating directly to the Licensed

4.2.3

Product since the last Development Progress Report;

4.2.4

Significant research and development projects relating directly to the Licensed Product currently being performed by ReGenX, its Affiliates, and its or their sublicensees and projected dates of completion.

4.2.5

Future development activities to be undertaken by ReGenX, its Affiliates or its or their sublicensees during the next reporting period relating directly to the Licensed Product;

4.2.6

Projected total development remaining before product launch of each Licensed Product; and

4.2.7

Summary of significant development efforts using the Penn Patent Rights being performed by Third Parties including the nature of the relationship between ReGenX and such Third Parties.

4.3 In the event that Penn and GSK modify the Penn License Agreement or any of the terms or provisions thereof (whether by formal amendment, letter agreement or other binding or applicable arrangement) where such modification has a material effect on ReGenX’s obligations under this Agreement (a “PLA Modification”), GSK shall provide notice of such PLA Modification in writing to ReGenX. The Parties shall then enter into discussions to determine whether this Agreement should be amended to reflect the PLA Modification in the Penn License Agreement so that ReGenX may benefit from all applicable terms of the PLA Modification to the extent that the PLA Modification lessens GSK’s obligations or requirements thereunder or otherwise benefits GSK. ****.

ARTICLE 5: CONFIDENTIALITY

5.1 Treatment of Confidential Information. Each Party, as a receiving party (a “Receiving Party”), agrees that it will (a) treat Confidential Information of the other Party (the “Disclosing Party”) as strictly confidential; (b) not disclose such Confidential Information to Third Parties without the prior written consent of the Disclosing Party, except as may be permitted by the

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CONFIDENTIAL TREATMENT REQUESTED

license grants contained herein; provided that such disclosure be under confidentiality agreements with provisions comparable to those contained in this Agreement; and (c) not use such Confidential Information for purposes other than those authorized expressly. A Receiving Party agrees to ensure that its employees who have access to Confidential Information are obligated in writing to abide by confidentiality obligations at least as stringent as those under this Agreement.

5.2 No Public Announcement. No public announcement or other disclosure to Third Parties concerning the existence of or terms of this Agreement shall be made, either directly or indirectly, by any Party to this Agreement, except as set forth in this Section 5.2 or to the extent legally required; provided that either Party may make such a disclosure of the existence of and/or terms of this Agreement to ****, who are obligated to keep such information confidential on terms no less stringent than those set forth herein. Furthermore, GSK shall be permitted to provide a copy of this Agreement to Penn, pursuant to GSK’s obligations under the Penn License Agreement. In the event that the Receiving Party becomes obligated by law to disclose the existence of or terms of this Agreement to any governmental authority, the Receiving Party shall promptly notify the Disclosing Party, so that the Disclosing Party may seek an appropriate protective order or other remedy with respect to narrowing the scope of such requirement and/or waive compliance by the Receiving Party with the provisions of this Agreement. If, in the absence of such protective order or other remedy, the Receiving Party is nonetheless required by law to disclose the existence of or terms of this Agreement, the Receiving Party may disclose such Confidential Information without liability hereunder; provided, that the Receiving Party shall furnish only such portion of the Confidential Information which is legally required to be disclosed and only to the extent required by law.

5.3 Treatment of Development Progress Reports. All Development Progress Reports provided by ReGenX to GSK shall be considered the Confidential Information of ReGenX. GSK shall be permitted to provide such Development Progress Reports to Penn, subject to GSK’s obligations of confidentiality hereunder. Notwithstanding anything to the contrary in this Article 5., GSK shall be obligated to keep such Development Progress Reports confidential for a period of **** following the expiration or termination of this Agreement.

ARTICLE 6: TERM AND TERMINATION

6.1 Term of Agreement. This Agreement, unless sooner terminated as provided in this Agreement, terminates upon the expiration, lapse, abandonment or invalidation of the last Valid Claim to expire, lapse, become abandoned or unenforceable in all the countries of the world where Penn Patent Rights existed; provided, however, that if not one patent ever issues from the Penn Patent Rights, then this Agreement will terminate ten (10) years after the first commercial sale of the first Licensed Product in any country. Expiration of this Agreement or expiration of ReGenX’s obligation to pay royalties to GSK in any country hereunder shall not preclude ReGenX from continuing to market, have marketed, sell and have sold Licensed Product in such country without further payment or obligation to GSK.

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CONFIDENTIAL TREATMENT REQUESTED

6.2 ReGenX’s Right to Terminate. ReGenX may, upon **** written notice to GSK, terminate this Agreement for any reason, with or without cause.

6.3 Termination for Breach. GSK may terminate this Agreement if:

6.3.1

ReGenX is more than forty-five (45) days late in paying to GSK royalties, expenses, or any other monies due under this Agreement and ReGenX does not pay GSK in full within thirty (30) days upon written demand from GSK; or

6.3.2

ReGenX materially breaches this Agreement and does not cure such material breach within forty-five (45) days after written notice of the breach.

6.4 Termination for Insolvency. In the event that ReGenX files for protection under bankruptcy laws, makes a general assignment for the benefit of creditors, appoints or suffers appointment of a receiver or trustee over its business, files a petition under any bankruptcy or insolvency act or has any such petition filed against it which is not dismissed or stayed within ninety (90) days of the filing thereof, GSK may terminate this Agreement effective immediately upon written notice to ReGenX.

6.5 Effects of Termination. The effect of termination by ReGenX pursuant to Section 6.2, or by GSK pursuant to Section 6.3 or 6.4 shall be as follows:

6.5.1

ReGenX shall cease to make, have made, use, import, sell and offer for sale all Licensed Products;

6.5.2

ReGenX shall assign to GSK all sublicenses granted to Third Parties related solely to the Penn Patent Rights or the Licensed Products;

6.5.3

ReGenX shall grant, and hereby grants to GSK a non-exclusive, perpetual, irrevocable, worldwide, royalty-free license under any patentable modifications or improvements developed by ReGenX to any vector that is the subject of a claim in any of the Penn Patent Rights, for use by GSK for the research, development and commercialization of products in any therapeutic indication. Such license shall be (a) fully sublicensable to GSK’s Affiliates, GSK Collaborators, and Third Parties with respect to the research, development and commercialization of Domain Antibodies, and (b) only sublicensable to GSK’s Affiliates and GSK Collaborators for all other purposes;

6.5.4

ReGenX shall transfer to GSK all remaining ReGenX Materials that have been requested by and made for GSK under Section 3.7 prior to the effective date of termination at GSK’s cost and expense;

6.5.5

ReGenX shall pay all monies then-owed to GSK under this Agreement; and

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CONFIDENTIAL TREATMENT REQUESTED

6.5.6

Each Party shall, at the other Party’s request, return all Confidential Information of the Disclosing Party. Notwithstanding the foregoing, one copy may be kept by either Party for a record of that Party’s obligations.

6.6 Survival. ReGenX’s obligation to pay all monies due and owed to GSK under this Agreement which have matured as of the effective date of termination shall survive the termination of this Agreement. In addition, the provisions of Articles 5—Confidentiality, Article 6—Term and Termination, Article 8—Warranties; Indemnification, Article 9—Use of ReGenX’s and GSK’s Name; and Article 10—Additional Provisions shall survive such termination or expiration of this Agreement in accordance with their respective terms.

ARTICLE 7: PATENT MAINTENANCE; PATENT INFRINGEMENT

7.1 Prosecution of Penn Licensed Patents. Pursuant to the Penn License Agreement, Penn controls the preparation, prosecution and maintenance of the Penn Patent Rights. As of the Effective Date, Penn and GSK have agreed on the selection of appropriate patent counsel to carry out Penn’s prosecution responsibilities. If, after the Effective Date, Penn seeks to appoint different patent counsel, GSK will not agree to such patent counsel without consultation with ReGenX. GSK shall provide to ReGenX a copy of any paper in a reasonable time prior to submission to the Patent and Trademark Office for review and comment, and GSK shall reasonably consider any comments thereon by ReGenX and relay such comments to Penn. Further, GSK shall keep ReGenX informed of the progress of any patent filings. ReGenX shall also have the right to consult directly with Penn patent counsel. The Parties shall keep each other informed with respect to any material communications with Penn regarding the Penn Patent Rights licensed hereunder. **** upon receipt of an invoice for all documented and reasonable Third Party expenses incurred in connection with the filing, prosecution and the maintenance of the Penn Patent Rights. If, in ReGenX’s opinion, the costs of preparation, prosecution and maintenance are inappropriate, then ReGenX and GSK shall discuss and agree on a resolution. If ReGenX elects not to pay for the filing, prosecution or maintenance of any patent or patent application within the Penn Patent Rights, ReGenX shall provide GSK with reasonable advance notice in order to enable GSK to assume responsibility for the filing, prosecution or maintenance of any such patent or patent application, and then such patent or patent application shall no longer be a part of the Penn Patent Rights licensed hereunder.

7.2 Infringement Actions Against Third Parties.

7.2.1

ReGenX and GSK are responsible for notifying each other promptly of any infringement of Penn Patent Rights (other than Retained Rights) which may come to their attention. ReGenX and GSK shall consult one another in a timely manner concerning any appropriate response to the infringement.

7.2.2

To the extent permitted under the Penn License Agreement, ReGenX may prosecute such infringement at its own expense. ReGenX shall not settle or compromise any such suit in a manner that imposes any material obligations or restrictions on GSK or Penn or grants any rights to the Penn Patent Rights other

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CONFIDENTIAL TREATMENT REQUESTED

than rights which ReGenX has the right to grant under this Agreement, without GSK’s prior written permission. All monies recovered upon the final judgment or settlement of any such action shall be used (a) first, to reimburse the costs and expenses (including reasonable attorneys’ fees and costs) of ****, **** and ****; (b) second, to **** to account for lost sales or lost profits (to the extent that damages are awarded for lost sales or lost profits from the sale ****); (c) third, to **** to the extent necessary to account for the royalties that would have been payable to **** but for the lost sales or lost profits; and (d) the remainder to the account of the **** that undertake such actions to the relative extent of their financial participation therein.

7.2.3

ReGenX’s rights under Section 7.2 are subject to the continuing right of Penn and GSK to intervene at Penn’s or GSK’s own expense and join ReGenX in any claim or suit for infringement of the Penn Patent Rights. Any consideration received by GSK or Penn in the settlement or award for any claim or suit shall be shared between ReGenX, GSK and Penn as set forth in Section 7.2.2 above.

7.2.4

If ReGenX elects to pursue an infringer under Section 7.2.2 above and ReGenX fails to prosecute such infringement, then GSK may prosecute such infringement at its own expense. In such event, financial recoveries will be entirely retained by GSK.

7.2.5

In any action to enforce any of the Penn Patent Rights, either Party, at the request and expense of the other Party shall cooperate to the fullest extent reasonably possible, including in the event that if either Party is unable to initiate or prosecute such action solely in its own name, the other Party shall join such action voluntarily and shall execute all documents necessary to initiate litigation to prosecute and maintain such action. This provision shall not be construed to require either Party to undertake any activities, including legal discovery, at the request of any Third Party except as may be required by lawful process of a court of competent jurisdiction.

7.3 Defense of Infringement Claims.

7.3.1

In the event ReGenX or GSK becomes aware that ReGenX’s or any of its Affiliates’ or sublicensees’ practice of the Penn Patent Rights is the subject of a claim for patent infringement by a Third Party, that Party shall promptly notify the other and the Parties shall consider the claim and the most appropriate action to take. ReGenX shall cause each of its Affiliates and sublicensees to notify ReGenX promptly in the event such entity becomes aware that its practice of the Penn Patent Rights is the subject of a claim of patent infringements by another. ReGenX shall have the right to control the defense of any such suit brought against ReGenX or any of its Affiliates or sublicensees and shall do so at its own expense. ReGenX shall have the right to require GSK’s and Penn’s reasonable cooperation in any such suit, upon written notice to GSK and Penn, and GSK and Penn shall have the obligation to participate and ReGenX shall bear the cost of

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CONFIDENTIAL TREATMENT REQUESTED

GSK’s and Penn’s participation. ReGenX must not settle or compromise any such suit in a manner that imposes any material obligations or restrictions on GSK or Penn or grants any rights to the Penn Patent Rights other than rights which ReGenX has the right to grant under this Agreement, without GSK’s prior written permission.

ARTICLE 8: WARRANTIES; INDEMNIFICATION

8.1 Warranty. GSK represents, warrants and covenants to ReGenX that it has sufficient rights in the Penn Patent Rights to grant to ReGenX the rights specified in this Agreement.

8.2 Disclaimer of Warranties.

8.2.1

Disclaimer by GSK. EXCEPT AS SET FORTH IN SECTION 8.1, THE PENN PATENT RIGHTS, LICENSED PRODUCTS AND ALL OTHER TECHNOLOGY LICENSED UNDER THIS AGREEMENT ARE PROVIDED ON AN “AS IS” BASIS AND GSK MAKES NO REPRESENTATIONS OR WARRANTIES, EXPRESS OR IMPLIED, WITH RESPECT THERETO. BY WAY OF EXAMPLE BUT NOT OF LIMITATION, GSK MAKES NO REPRESENTATIONS OR WARRANTIES (i) OF COMMERCIAL UTILITY; (ii) OF MERCHANTABILITY OR FITNESS FOR A PARTICULAR PURPOSE; OR (iii) THAT THE USE OF THE PENN PATENT RIGHTS, LICENSED PRODUCTS AND ALL TECHNOLOGY LICENSED UNDER THIS AGREEMENT WILL NOT INFRINGE ANY PATENT, COPYRIGHT OR TRADEMARK OR OTHER PROPRIETARY RIGHTS OF THIRD PARTIES. EXCEPT AS SET FORTH HEREIN, GSK SHALL NOT BE LIABLE TO REGENX, REGENX’S SUCCESSORS OR ASSIGNS OR ANY THIRD PARTY WITH RESPECT TO: ANY CLAIM ARISING FROM REGENX’S USE OF THE PENN PATENT RIGHTS, LICENSED PRODUCTS AND ALL RIGHTS LICENSED UNDER THIS AGREEMENT OR FROM THE MANUFACTURE, USE OR SALE OF LICENSED PRODUCTS; OR ANY CLAIM FOR LOSS OF PROFITS, LOSS OR INTERRUPTION OF BUSINESS, OR FOR INDIRECT, SPECIAL OR CONSEQUENTIAL DAMAGES OF ANY KIND.

8.2.2

Disclaimer by ReGenX. THE REGENX MATERIALS AND ALL LICENSED BACK IMPROVEMENTS UNDER SECTION 2.5 PROVIDED OR LICENSED UNDER THIS AGREEMENT ARE PROVIDED ON AN “AS IS” BASIS AND REGENX MAKES NO REPRESENTATIONS OR WARRANTIES, EXPRESS OR IMPLIED, WITH RESPECT THERETO. BY WAY OF EXAMPLE BUT NOT OF LIMITATION, REGENX MAKES NO REPRESENTATIONS OR WARRANTIES (i) OF COMMERCIAL UTILITY; (ii) OF MERCHANTABILITY OR FITNESS FOR A PARTICULAR PURPOSE; OR (iii) THAT THE USE OF THE REGENX MATERIALS THE LICENSED BACK IMPROVEMENTS AND ALL TECHNOLOGY LICENSED UNDER THIS AGREEMENT WILL NOT INFRINGE ANY PATENT, COPYRIGHT OR TRADEMARK OR OTHER PROPRIETARY RIGHTS OF THIRD PARTIES. EXCEPT AS SET FORTH HEREIN, REGENX SHALL NOT BE LIABLE TO GSK, GSK’S SUCCESSORS OR ASSIGNS OR ANY THIRD PARTY WITH RESPECT TO: ANY CLAIM ARISING FROM GSK’S USE OF THE REGENX MATERIALS, THE

CONFIDENTIAL TREATMENT REQUESTED

LICENSED BACK MATERIALS AND ALL RIGHTS LICENSED UNDER THIS AGREEMENT OR FROM THE MANUFACTURE, USE OR SALE OF PRODUCTS BASED THEREON; OR ANY CLAIM FOR LOSS OF PROFITS, LOSS OR INTERRUPTION OF BUSINESS, OR FOR INDIRECT, SPECIAL OR CONSEQUENTIAL DAMAGES OF ANY KIND.

8.3 Indemnification.

8.3.1

By ReGenX. ReGenX shall defend, indemnify and hold harmless GSK, its officers, agents and employees (individually, a “GSK Indemnified Party”, and collectively, the “GSK Indemnified Parties”), from and against any and all liability, loss, damage, action, claim or expense (including attorneys’ fees) (individually, a “Liability”, and collectively, the “Liabilities”) suffered or incurred by the GSK Indemnified Parties from Third Parties that results from or arises out of: ****; provided, however, that ReGenX shall not be liable for claims based on the gross negligence or intentional misconduct of any of the GSK Indemnified Parties. Without limiting the foregoing, ReGenX must defend, indemnify and hold harmless the GSK Indemnified Parties from and against any Liabilities resulting from:

(a)

any **** or other claim of any kind related to the **** by a Third Party of a Licensed Product that was **** by ReGenX, its Affiliates, assignees, sublicensees, or vendors; and

(b)

**** conducted by or on behalf of ReGenX or its Affiliates or sublicensees relating to the Penn Patent Rights or Licensed Products, including, without limitation, any claim by or on behalf of ****.

8.3.2

By GSK. GSK shall defend, indemnify and hold harmless ReGenX, its officers, agents and employees (individually, a “ReGenX Indemnified Party”, and collectively, the “ReGenX Indemnified Parties”), from and against any and all Liabilities suffered or incurred by the ReGenX Indemnified Parties from Third Parties that results from or arises out of: ****; provided, however, that GSK shall not be liable for claims based on the gross negligence or intentional misconduct of any of the ReGenX Indemnified Parties. Without limiting the foregoing, GSK must defend, indemnify and hold harmless the ReGenX Indemnified Parties from and against any Liabilities resulting from:

(a)

any **** or other claim of any kind related to the **** by a Third Party of a product developed from or based on the ReGenX Materials or Licensed Back Improvements

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CONFIDENTIAL TREATMENT REQUESTED

that was **** **** by GSK, its Affiliates, assignees, sublicensees (other than ReGenX), or vendors; and

(b)

**** conducted by or on behalf of GSK or its Affiliates or sublicensees (other than ReGenX) relating to a product developed from or based on the ReGenX Materials or Licensed Back Improvements, including, without limitation, any claim by or on behalf of ****.

8.4 Indemnification Procedure. Each Party, as an indemnifying party (a “Indemnifying Party”), shall not be permitted to settle or compromise any claim or action giving rise to Liabilities in a manner that imposes any restrictions or obligations on the other Party (the “Indemnified Party”) (or Penn as applicable) or grant any rights to the Penn Patent Rights, Licensed Products, ReGenX Materials or Licensed Back Improvements other than those ReGenX has the right to grant under this Agreement without GSK’s prior written consent. If an Indemnifying Party fails or declines to assume the defense of any such claim or action within twenty (20) days after notice thereof, the Indemnified Party may assume the defense of such claim or action at the cost and risk of the Indemnifying Party, and any Liabilities related thereto shall be conclusively deemed a Liability of the Indemnifying Party. The indemnification rights of a Indemnified Party contained herein are in addition to all other rights which such Indemnified Party may have at law or in equity or otherwise.

8.5 Insurance. Prior to the first administration of a Licensed Product to a human, ReGenX shall obtain and/or maintain, at its sole cost and expense, **** insurance in amounts, which are reasonable and customary in the U.S. pharmaceutical industry, subject always to a minimum limit of **** per occurrence (or claim) and in the aggregate annually. Such **** insurance shall insure against all liability, including ****.

ARTICLE 9: USE OF A PARTY’S NAME

9.1 ReGenX and its employees and agents must not use and ReGenX must not permit its Affiliates or sublicensees to use GSK’s or Penn’s name or any adaptation thereof, or any GSK or Penn seal, logotype, trademark, or service mark, or the name, mark, or logotype of any GSK or Penn representative or organization in any way without the prior written consent of GSK or Penn; provided, however that ReGenX may acknowledge the existence and general nature of this Agreement.

9.2 GSK and its employees and agents must not use ReGenX’s name or any adaptation thereof, or any ReGenX seal, logotype, trademark, or service mark, or the name, mark, or logotype of any ReGenX representative or organization in any way without the prior written consent of ReGenX; provided, however, that GSK may acknowledge the existence and general nature of this Agreement.

ARTICLE 10: ADDITIONAL PROVISIONS

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CONFIDENTIAL TREATMENT REQUESTED

10.1 Relationship. Nothing in this Agreement shall be deemed to establish a relationship of principal and agent between ReGenX and GSK, nor any of their agents or employees for any purpose whatsoever, nor shall this Agreement be construed as creating any other form of legal association or arrangement which would impose liability upon one Party for the act or failure to act of the other Party.

10.2 Assignment. The rights and obligations of ReGenX and GSK hereunder shall inure to the benefit of, and shall be binding upon, their respective successors and assigns. Neither Party may assign its rights under this Agreement without the prior written consent of the other Party, such consent not to be unreasonably withheld; provided however, a Party may assign this Agreement to (a) any Affiliate of such Party or (b) any corporation or other entity to which such Party may transfer all or substantially all of its assets to which this Agreement relates (“Sale of Assets”). ReGenX shall notify GSK in writing at least thirty (30) days prior to the anticipated closing of any bona fide Sale of Assets it proposes to effect or any merger or consolidation pursuant to which the holders of the voting power of ReGenX immediately prior to such merger or consolidation hold, immediately after such merger or consolidation, less than 50% of the voting power of ReGenX (“Sale by Merger”). Upon ReGenX’s closing of a Sale of Assets or Sale by Merger, GSK’s obligations under Section 2.1.2 and Section 2.1.3 to provide written notice to ReGenX of any sublicense it grants to the Retained Rights shall cease. No assignment shall relieve such Party of responsibility for the performance of any accrued obligations which it has prior to such assignment.

10.3 Waiver. A waiver by either Party of a breach of any provision of this Agreement will not constitute a waiver of any subsequent breach of that provision or a waiver of any breach of any other provision of this Agreement.

10.4 Notices. Notices, payments, statements, reports and other communications under this Agreement shall be in writing and shall be deemed to have been received as of the date sent if sent by public courier (e.g. Federal Express) or by Express Mail, receipt requested, and addressed as follows:


If for ReGenX:		with a copy to:

ReGenX, LLC		ReGenX, LLC
750 17th Street, NW		750 17th Street, NW
Suite 1100		Suite 1100
Washington, DC 20006		Washington, DC 20006
USA		USA
Attn: Chief Executive Officer		Attn: General Counsel

If for GSK:		with a copy to:

GlaxoSmithKline		GlaxoSmithKline
Gunnels Wood Road		2301 Renaissance Blvd.
Mail Code 3T123		Mail Code RN0220

CONFIDENTIAL TREATMENT REQUESTED


Stevenage		King of Prussia, PA 19406
Hertfordshire SG12NY		Attn: Associate General
United Kingdom		Counsel
Attn: Vice President,
Business Development,
BioPharm Management and Administration

Either Party may change its official address upon written notice to the other Party.

10.5 Applicable Law. This Agreement shall be construed and governed in accordance with the laws of the Commonwealth of Pennsylvania, without giving effect to conflict of law provisions. In the event that a Party to this Agreement perceives the existence of a dispute with the other Party concerning any right or duty provided for herein, the Parties will, as soon as practicable, confer in an attempt to resolve the dispute. If the Parties are unable to resolve such dispute amicably, then the Parties hereby submit to the exclusive jurisdiction of and venue in the courts located in the Eastern District of the Commonwealth of Pennsylvania with respect to any and all disputes concerning the subject of this Agreement.

10.6 No Discrimination. ReGenX and GSK, in their activities under this Agreement, shall not discriminate against any employee or applicant for employment because of race, color, sex, sexual or affectional preference, age, religion, national or ethnic origin, handicap, or because he or she is a disabled veteran or a veteran of the Vietnam Era.

10.7 Compliance with Law. GSK and ReGenX must comply with all prevailing laws, rules and regulations that apply to its activities or obligations under this Agreement. Without limiting the foregoing, it is understood that this Agreement may be subject to United States laws and regulations controlling the export of technical data, computer software, laboratory prototypes and other commodities, articles and information, including the Arms Export Control Act as amended in the Export Administration Act of 1979, and that the Parties’ obligations are contingent upon compliance with applicable United States export laws and regulations. The transfer of certain technical data and commodities may require a license from the cognizant agency of the United States Government and/or written assurances by GSK or ReGenX that GSK or ReGenX shall not export data or commodities to certain foreign countries without prior approval of such agency. ReGenX neither represents that a license is not required nor that, if required, it will issue.

10.8 Entire Agreement. This Agreement embodies the entire understanding between the Parties relating to the subject matter hereof and supersedes all prior understandings and agreements, whether written or oral. This Agreement may not be varied except by a written document signed by duly authorized representatives of both Parties.

[REMAINDER OF PAGE INTENTIONALLY LEFT BLANK]

CONFIDENTIAL TREATMENT REQUESTED

IN WITNESS WHEREOF, the Parties, intending to be legally bound, have caused this LICENSE AGREEMENT to be executed by their duly authorized representatives.


REGENX		SMITHKLINE BEECHAM CORPORATION d/b/a GLAXOSMITHKLINE

By:	/s/ Kenneth T. Mills	By:	/s/ William J. Mosher


Name:	Kenneth T. Mills	Name:	William J. Mosher
Title:	CEO	Title:	Vice President & Secretary

CONFIDENTIAL TREATMENT REQUESTED

Exhibit 1

Penn Patent Rights


Penn #	Disclosure Title	Inventors	Applicant(s)	US Patents	Foreign Patents
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****
	****
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****
****	****	****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Exhibit 2

Muscular Dystrophies

****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

EX-10.14

Exhibit 10.14

CONFIDENTIAL TREATMENT REQUESTED

LICENSE AGREEMENT

This LICENSE AGREEMENT (“Agreement”) is entered into as of April 10, 2014 (“Effective Date”) by and between ReGenX Biosciences, LLC, a limited liability company organized under the laws of the State of Delaware, with offices at 750 17th Street, NW, Suite 1100, Washington, DC 20006, USA (“Licensor”), and AAVLife, a French simplified joint stock company (Société par actions simplifiée) whose registered office is 183/189 avenue de Choisy – 75013 Paris, France (“Licensee”). Licensor and Licensee are hereinafter referred to individually as a “Party” and collectively as the “Parties.”

WHEREAS, Licensor has rights under certain Licensed Patents (as defined herein) pertaining to certain recombinant adeno-associated virus vectors; and

WHEREAS, Licensee desires to obtain from Licensor certain licenses under the Licensed Patents under the terms set forth herein;

NOW, THEREFORE, in consideration of the promises and covenants contained in this Agreement, and intending to be legally bound, the Parties hereby agree as follows:

ARTICLE 1: DEFINITIONS

1.1 “AAV7” means (a) the recombinant adeno-associated virus serotype 7 vector with the specified sequence set forth in GenBank **** and (b) any recombinant adeno-associated virus derivatives of such serotype 7 vector that are covered by the claims of the Licensed Patents set forth on Exhibit A-1 (or other Licensed Patents relating thereto described in Section 1.19(b) or 1.22(b), as applicable).

1.2 “AAV8” means (a) the recombinant adeno-associated virus serotype 8 vector with the specified sequence set forth in GenBank **** and (b) any recombinant adeno-associated virus derivatives of such serotype 8 vector that are covered by the claims of the Licensed Patents set forth on Exhibit A-2 (or other Licensed Patents relating thereto described in Section 1.19(b) or 1.22(b), as applicable).

1.3 “AAV9” means (a) the recombinant adeno-associated virus serotype 9 vector with the specified sequence set forth in GenBank **** and (b) any recombinant adeno-associated virus derivatives of such serotype 9 vector that are covered by the claims of the Licensed Patents set forth on Exhibit A-3 (or other Licensed Patents relating thereto described in Section 1.19(b) or 1.22(b), as applicable).

1.4 “AAVrhl0” means (a) the recombinant adeno-associated virus serotype rh10 vector with the specified sequence set forth in GenBank **** and (b) any recombinant adeno-associated virus derivatives of such serotype rh10 vector that are covered by the claims of the Licensed Patents set forth on Exhibit A-4 (or other Licensed Patents relating thereto described in Section 1.19(b) or 1.22(b), as applicable).

1.5 “AAV Materials” means AAV Vectors, and any materials that are made or used for the sole purpose of making AAV Vectors, in each case, which, in the absence of the license granted

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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pursuant to Section 2.2, would infringe or is covered by at least one Valid Claim of the applicable Licensed Research Patents in the country of manufacture or use.

1.6 “AAV Vectors” means, collectively, AAV7, AAV8, AAV9, and AAVrh10.

1.7 “Affiliate” means any legal entity directly or indirectly, during the Term, controlling, controlled by, or under common control with another entity. For purposes of this Agreement, “control” means the direct or indirect ownership of more than 50% of the outstanding voting securities of a legal entity, or the right to receive more than 50% of the profits or earnings of a legal entity, or the right to control the policy decisions of a legal entity. An entity may be or become an Affiliate of an entity and may cease to be an Affiliate of an entity, in each case, during the Term.

1.8 “Calendar Quarter” means each three-month period or any portion thereof, beginning on January 1, April 1, July 1, and October 1.

1.9 “Commercial Field” means (a) the treatment of Friedreich’s Ataxia (Systemic) in human beings by in vivo gene therapy with AAVrh10; and (b) if and when a Commercial Option is exercised for a Disease Indication by Licensee under Section 2.3, the treatment of such Disease Indication in human beings by in vivo gene therapy with the Specified Vector selected for such Disease Indication.

1.10 “Commercial Option” has the meaning set forth in Section 2.3.

1.11 “Confidential Information” means and includes all technical information, inventions, developments, discoveries, software, know-how, methods, techniques, formulae, animate and inanimate materials, data, processes, finances, business operations or affairs, and other proprietary ideas, whether or not patentable or copyrightable, of either Party that are (a) marked or otherwise identified as confidential or proprietary at the time of disclosure in writing; or (b) if disclosed orally, visually, or in another non-written form, identified as confidential at the time of disclosure and summarized in reasonable detail in writing as to its general content within 30 days after original disclosure. The Parties acknowledge that (i) the terms and conditions of this Agreement and (ii) the records and reports referred to in Section 3.7 will be deemed the Confidential Information of both Parties, regardless of whether such information is marked or identified as confidential. Notwithstanding the foregoing, Confidential Information will not include the following, in each case, to the extent evidenced by competent written proof of the Receiving Party:

1.11.1 information that was already known to the Receiving Party, other than under an obligation of confidentiality, at the time of disclosure by the Disclosing Party;

1.11.2 information that was generally available to the public or otherwise part of the public domain at the time of its disclosure to the Receiving Party;

1.11.3 information that became generally available to the public or otherwise part of the public domain after its disclosure, other than through any act or omission of the Receiving Party in breach of this Agreement;

CONFIDENTIAL TREATMENT REQUESTED

1.11.4 information that is independently discovered or developed by the Receiving Party without the use of Confidential Information of the Disclosing Party; or

1.11.5 information that was disclosed to the Receiving Party, other than under an obligation of confidentiality, by a Third Party who had no obligation to the Disclosing Party not to disclose such information to others.

1.12 “Disclosing Party” has the meaning set forth in Section 5.1.

1.13 “Disease Indication(s)” means Friedreich’s Ataxia (CNS) and Friedreich’s Ataxia (Systemic).

1.14 “Domain Antibody” ****.

1.15 “FDA” means the United States Food and Drug Administration, or a successor agency in the United States with responsibilities comparable to those of the United States Food and Drug Administration.

1.16 “Friedreich’s Ataxia (CNS)” means Friedreich’s Ataxia that is treated by administration of the applicable AAV Vector directly to the central nervous system (brain and spinal cord).

1.17 “Friedreich’s Ataxia (Systemic)” means Friedreich’s Ataxia that is treated by administration of the applicable AAV Vector by any route except administration directly to the central nervous system (brain and spinal cord).

1.18 “GSK Agreement” means that certain License Agreement entered into between Licensor and SmithKline Beecham Corporation, effective on March 6, 2009, as amended by that certain Amendment to License Agreement dated April 15, 2009, and as amended from time to time.

1.19 “Licensed Commercial Patents” means, on a Specified Vector-by-Specified Vector basis, to the extent they cover such Specified Vector, (a) all United States patents and patent applications listed in Exhibit A-1 (if the Specified Vector is AAV7), Exhibit A-2 (if the Specified Vector is AAV8), Exhibit A-3 (if the Specified Vector is AAV9), or Exhibit A-4 (if the Specified Vector is AAVrh10), including patents arising from such patent applications; and (b) any re-examination certificates thereof, and their foreign counterparts and extensions, continuations, divisionals, and re-issue applications; provided that “Licensed Commercial Patents” will not include any claim of a patent or patent application covering any Manufacturing Technology.

1.20 “Licensed Patents” means the Licensed Commercial Patents or Licensed Research Patents, as applicable.

1.21 “Licensed Product” means (a) any product using the applicable Specified Vector that is made, made for, used, sold, offered for sale, or imported by Licensee, its Affiliates, and any of its or their Sublicensees, the manufacture, use, sale, offer for sale, or import of which product, in the absence of the license granted pursuant to this Agreement, would infringe or is covered by at

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

least one Valid Claim of the Licensed Commercial Patents in the country of manufacture, use, sale, offer for sale, or import, including products manufactured by a process that would infringe or is covered by at least one Valid Claim of the Licensed Commercial Patents in the country of manufacture, use, sale, offer for sale, or import; or (b) any service sold by Licensee, its Affiliates, and any of its or their Sublicensees with respect to the administration of any product using the applicable Specified Vector to patients that, in the absence of the license granted pursuant to this Agreement, would infringe or is covered by at least one Valid Claim of the Licensed Commercial Patents in the country of sale.

1.22 “Licensed Research Patents” means (a) all United States patents and patent applications listed in Exhibit A-1 (in the case of AAV7), Exhibit A-2 (in the case of AAV8), Exhibit A-3 (in the case of AAV9), and Exhibit A-4 (in the case of AAVrh10), in each case, including patents arising from such patent applications; and (b) any re-examination certificates thereof, and their foreign counterparts and extensions, continuations, divisionals, and re-issue applications; provided that “Licensed Research Patents” will not include any claim of a patent or patent application covering any Manufacturing Technology.

1.23 “Manufacturing Technology” means any and all patents, patent applications, know-how, and all intellectual property rights associated therewith that are owned or controlled by Licensor, and including all tangible embodiments thereof, that are necessary or useful for the manufacture of adeno-associated viruses, adeno-associated virus vectors, research or commercial reagents related thereto, Licensed Products, or other products, including manufacturing processes, technical information relating to the methods of manufacture, protocols, standard operating procedures, batch records, assays, formulations, quality control data, specifications, scale up, any and all improvements, modifications, and changes thereto, and any and all activities associated with such manufacture. Any and all chemistry, manufacturing, and controls (CMC), drug master files (DMFs), or similar materials provided to regulatory authorities and the information contained therein are deemed Manufacturing Technology.

1.24 “NDA” means a New Drug Application filed with the FDA as described in 21 C.F.R. § 314, a Biological License Application (BLA) pursuant to 21 C.F.R. § 601.2, or any equivalent or any corresponding application for regulatory approval in any country or regulatory jurisdiction other than the United States.

1.25 “Net Sales” means the gross receipts from sales or other disposition of a Licensed Product (including fees for services within the definition of “Licensed Product”) by Licensee and/or its Affiliates and/or any Sublicensees to Third Parties less the following deductions that are directly attributable to a sale, specifically and separately identified on an invoice or other documentation and actually borne by Licensee, its Affiliates, or any Sublicensees: ****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

****. In the event consideration other than cash is paid to Licensee, its Affiliates, or any Sublicensees, for purposes of determining Net Sales, the Parties shall use the cash consideration that Licensee, its Affiliates, or any Sublicensees would realize from an unrelated buyer in an arm’s length sale of an identical item sold in the same quantity and at the time and place of the transaction, as determined jointly by Licensor and Licensee based on transactions of a similar type and standard industry practice, if any.

1.26 “Penn Agreement” means that certain License Agreement entered into between Licensor and The Trustees of the University of Pennsylvania, effective on February 24, 2009, as amended by that letter agreement dated March 6, 2009, and as amended from time to time.

1.27 “Phase 3 Clinical Trial” means a pivotal clinical trial in humans performed to gain evidence with statistical significance of the efficacy of a product in a target population, and to obtain expanded evidence of safety for such product that is needed to evaluate the overall benefit-risk relationship of such product, to form the basis for approval of an NDA and to provide an adequate basis for physician labeling, as described in 21 C.F.R. § 312.21(c) or the corresponding regulation in jurisdictions other than the United States.

1.28 “Prosecute” means preparation, filing, and prosecuting patent applications and maintaining patents, including any reexaminations, reissues, oppositions, inter partes review, and interferences.

1.29 “Receiving Party” has the meaning set forth in Section 5.1.

1.30 “ReGenX Licensors” means SmithKline Beecham Corporation (or any successor thereto under the GSK Agreement) and The Trustees of the University of Pennsylvania (or any successor thereto under the Penn Agreement).

1.31 “Research Field” means Licensee’s internal research and pre-clinical development for the treatment of either Disease Indication in humans by in vivo gene therapy using AAV Materials (excluding AAVrh10 for Friedreich’s Ataxia (Systemic)). “Research Field” specifically excludes (without limitation) (a) all human clinical trial use, diagnostic use, therapeutic use, and prophylactic use, and (b) any commercial uses.

1.32 “Research Term” means the following:

(a)

with respect to Friedreich’s Ataxia (Systemic), a period beginning with the Effective Date and ending on the earlier of (i) the Grant Date, if any, for such Disease Indication and (ii) the first anniversary of the Effective Date; and

(b)

with respect to Friedreich’s Ataxia (CNS), a period beginning with the Effective Date and ending on the earlier of (i) the Grant Date, if any, for such Disease Indication and (ii) the second anniversary of the Effective Date.

1.33 “Retained Rights” has the meaning set forth in Section 2.4.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

1.34 “Specified Vector” means the following:

(a)

with respect to Friedreich’s Ataxia (Systemic), (i) AAVrhl0 and (ii) if a Commercial Option is exercised with respect to Friedreich’s Ataxia (Systemic), the AAV Vector that is selected by Licensee pursuant to Section 2.3, and

(b)

with respect to Friedreich’s Ataxia (CNS), if a Commercial Option is exercised with respect to Friedreich’s Ataxia (CNS), the AAV Vector that is selected by Licensee pursuant to Section 2.3.

The Specified Vectors and applicable Disease Indication will be set forth on Exhibit B (to be amended as of the applicable Grant Date as provided in Section 2.3).

1.35 “Sublicensee” means (i) any Third Party or Affiliate to whom Licensee grants a sublicense of some or all of the rights granted to Licensee under this Agreement as permitted by this Agreement; and (ii) any other Third Party or Affiliate to whom a sublicensee described in clause (i) has granted a further sublicense as permitted by this Agreement.

1.36 “Third Party” means any person or entity other than a Party to this Agreement or Affiliates of a Party to this Agreement.

1.37 “Valid Claim” means a claim of an issued and unexpired patent (including any patent claim the term of which is extended by any extension, supplementary protection certificate, patent term restoration, or the like) included within the Licensed Patents or a claim of a pending patent application included within the Licensed Patents, which has not lapsed, been abandoned, been held revoked, or been deemed unenforceable or invalid by a non-appealable decision or an appealable decision from which no appeal was taken within the time allowed for such appeal of a court or other governmental agency of competent jurisdiction.

ARTICLE 2: LICENSE GRANTS

2.1 Exclusive License Grant. Subject to the terms and conditions of this Agreement, including the Retained Rights, Licensor hereby grants to Licensee an exclusive, sublicensable (as provided in Section 2.6 only), non-transferable (except as provided in Section 10.2), royalty-bearing, worldwide license under the Licensed Commercial Patents to make, have made, use, import, sell, and offer for sale Licensed Products using AAVrh10 solely in the Commercial Field of Friedreich’s Ataxia (Systemic), including, for the avoidance of doubt, the right to conduct research and development.

2.2 Research License Grant. Subject to the terms and conditions of this Agreement, including the Retained Rights, during the Research Term, Licensor hereby grants to Licensee a non-exclusive, sublicensable (as provided in Section 2.6 only), non-transferable (except as provided in Section 10.2), worldwide license under the Licensed Research Patents to make, have made, and use AAV Materials in the Research Field (including, for the avoidance of doubt, the right to conduct research and pre-clinical development) solely for purposes of selecting Specified Vector(s) for use in the Commercial Field upon exercise of a Commercial Option. For the

CONFIDENTIAL TREATMENT REQUESTED

avoidance of doubt, the foregoing license in this Section 2.2 does not include the right to sell, offer for sale, or import any AAV Materials.

2.3 Commercial License Option. Subject to the terms and conditions of this Agreement, Licensor hereby grants to Licensee the option, exercisable at Licensee’s sole discretion, to obtain a non-exclusive worldwide license with respect to each of the Disease Indications and a single Specified Vector for such Disease Indication (each such right with respect to a particular Disease Indication, a “Commercial Option”) in accordance with the following provisions:

2.3.1 Method of Exercise. To exercise the Commercial Option for a particular Disease Indication, Licensee must provide written notice to Licensor prior to the end of the applicable Research Term, which written notice must specify the Disease Indication and Specified Vector (as further described in Section 2.3.2) with respect to which Licensee desires to exercise its Commercial Option. For Friedreich’s Ataxia (CNS), such written notice must be accompanied by a wire transfer of the commercial option fee set forth in Section 3.2.

2.3.2 Specified Vector. For purposes of selecting a Specified Vector for use with a Disease Indication, the Specified Vector must be ****. Upon Licensor’s receipt of the notice and, if applicable, fee described in Section 2.3.1, Exhibit B will be amended to set forth the Specified Vector for each Disease Indication with respect to which a Commercial Option is exercised.

2.3.3 License Grant Upon Exercise. If Licensee exercises the Commercial Option for a particular Disease Indication, effective upon Licensor’s receipt of the notice and, if applicable, fee described in Section 2.3.1 (the “Grant Date” for such Disease Indication with respect to the applicable Specified Vector), subject to the terms and conditions of this Agreement, including the Retained Rights, Licensor shall be deemed to have granted to Licensee a non-exclusive, sublicensable (as provided in Section 2.6 only), non-transferable (except as provided in Section 10.2), royalty-bearing, worldwide license under the applicable Licensed Commercial Patents to make, have made, use, import, sell, and offer for sale Licensed Products using the Specified Vector solely in the Commercial Field of such Disease Indication, including, for the avoidance of doubt, the right to conduct research and development.

2.3.4 Disease Indications. For the avoidance of doubt, the foregoing license granted pursuant to Section 2.3.3 will be deemed granted on the Grant Date on a Disease Indication-by-Disease Indication basis, solely with respect to the Commercial Field associated with the Disease Indication for which the Commercial Option was exercised under this Section 2.3 and solely with respect to Licensed Products using the Specified Vector selected for the particular Disease Indication. The Parties acknowledge that there may be different Grant Dates for each Disease Indication, depending on when and if Licensee exercises the Commercial Option for a particular Disease Indication. As set forth above, Licensee, at its sole discretion, may exercise the Commercial Option with respect to either or both of the two Disease Indications. If Licensee exercises the Commercial Option with respect to only one of the Disease Indications but not both, the Commercial Option will terminate with respect to the unexercised Disease Indication at the end of the applicable Research Term (together with the license granted under Section 2.2), and Licensee will have no further rights under this Agreement with respect to Friedreich’s Ataxia

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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(CNS) if it is the unexercised Disease Indication or with respect to Friedreich’s Ataxia (Systemic) with respect to any Specified Vector (other than AAVrhl0) if it is the unexercised Disease Indication; provided that the termination of a Commercial Option with respect to Friedreich’s Ataxia (Systemic) will not affect Licensee’s rights under this Agreement with respect to the license granted under Section 2.1.

2.4 Retained Rights. Except for the rights and licenses specified in Sections 2.1, 2.2, and, if applicable, 2.3.3, no license or other rights are granted to Licensee under any intellectual property of Licensor, whether by implication, estoppel, or otherwise and whether such intellectual property is subordinate, dominant, or otherwise useful for the practice of the Licensed Patents. Notwithstanding anything to the contrary in this Agreement, Licensor may use and permit others to use the Licensed Patents for any research, development, commercial, or other purposes inside or outside of the Commercial Field (other than to the extent of the exclusive license under Section 2.1) or the Research Field. Without limiting the foregoing, and notwithstanding anything in this Agreement to the contrary, Licensee acknowledges and agrees to the following rights retained by Licensor and the ReGenX Licensors (individually and collectively, the “Retained Rights”), whether inside or outside the Commercial Field or Research Field:

2.4.1 The rights and licenses granted in Sections 2.1, 2.2, and, if applicable, 2.3.3 shall not include any right (and Licensor and the ReGenX Licensors retain the exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents to make, have made, use, sell, offer to sell, and import Domain Antibodies that are expressed by an adeno-associated vector, including any Specified Vector.

2.4.2 Licensor and the ReGenX Licensors retain the following rights with respect to the Licensed Patents:

(a)

A non-exclusive, sublicensable right under the Licensed Patents to make, have made, use, sell, offer to sell, and import products that deliver RNA interference and antisense drugs using an adeno-associated vector, including any Specified Vector; and

(b)

A non-exclusive right for the ReGenX Licensors (which right is sublicensable by such licensors) to use the Licensed Patents for non-commercial research purposes and to use the Licensed Patents for such licensors’ discovery research efforts with non-profit organizations and collaborators.

2.4.3 The rights and licenses granted in Sections 2.1, 2.2 and, if applicable, 2.3.3 shall not include any right (and Licensor retains the exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents:

(a)

to conduct commercial reagent and services businesses, which includes the right to make, have made, use, sell, offer to sell, and import research reagents, including any viral vector construct; provided that, for clarity, the foregoing retained right does not give Licensor (i) the right to conduct

CONFIDENTIAL TREATMENT REQUESTED

clinical trials in humans in the Commercial Field for Friedreich’s Ataxia (Systemic) using AAVrh10 or (ii) the exclusive right to conduct clinical trials in humans in any other Commercial Field with respect to which a Commercial Option has been exercised, though Licensor retains the non-exclusive right to do so; or

(b)

to use the Licensed Patents to provide services to any Third Parties; provided that Licensee’s license under Sections 2.1 and, if applicable, 2.3.3 does include the right to provide the service of the administration of Licensed Products to patients.

2.4.4 Licensor retains the fully sublicensable right under the Licensed Patents to grant non-exclusive research and development licenses to Affiliates and Third Parties; provided that such development rights granted by Licensor shall not include the right to conduct clinical trials in humans in the Commercial Field for Friedreich’s Ataxia (Systemic) using AAVrhl0 or any rights to sell products using AAVrhl0 in the Commercial Field for Friedreich’s Ataxia (Systemic).

2.4.5 The Trustees of the University of Pennsylvania may use and permit other non-profit organizations or other non-commercial entities to use the Licensed Patents for educational and research purposes.

2.5 Government Rights. Licensee acknowledges that the United States government retains certain rights in intellectual property funded in whole or part under any contract, grant, or similar agreement with a federal agency. The license grants hereunder are expressly subject to all applicable United States government rights, including any applicable requirement that products that result from such intellectual property and are sold in the United States must be substantially manufactured in the United States.

2.6 Sublicensing.

2.6.1 The research license granted pursuant to Section 2.2 is not sublicensable by Licensee, except to its Affiliates; provided that any such sublicense to an Affiliate must comply with the provisions of this Section 2.6 (including Section 2.6.2). The license granted pursuant to Sections 2.1 and, if applicable, 2.3.3 is sublicensable by Licensee to any Affiliates or Third Parties; provided that any such sublicense must comply with the provisions of this Section 2.6 (including Section 2.6.2).

2.6.2 The right to sublicense granted to Licensee under this Agreement is subject to the following conditions:

(a)

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(b)

In each sublicense agreement, the Sublicensee must be required to comply with the terms and conditions of this Agreement to the same extent as Licensee has agreed and must acknowledge that Licensor is an express third party beneficiary of such terms and conditions under such sublicense agreement.

(c)

The official language of any sublicense agreement shall be English.

(d)

Within **** after entering into a sublicense, Licensor must receive an unredacted copy of the sublicense written in the English language for Licensor’s records and to share with the ReGenX Licensors.

(e)

Licensee’s execution of a sublicense agreement will not relieve Licensee of any of its obligations under this Agreement. Licensee is and shall remain **** to Licensor for all of Licensee’s duties and obligations contained in this Agreement and for any act or omission of an Affiliate or Sublicensee that would be a breach of this Agreement if performed or omitted by Licensee, and Licensee will be deemed to be in breach of this Agreement as a result of such act or omission.

2.7 Improvements.

2.7.1 Licensee hereby grants to Licensor a non-exclusive, worldwide, royalty-free, transferable, sublicensable, irrevocable, perpetual license:

(a)

to use any Licensed Back Improvements (and any intellectual property rights with respect thereto) consummate in scope to the Retained Rights, and

(b)

to practice the Licensed Back Improvements (and any intellectual property rights with respect thereto) for any and all purposes, including the right to research, develop, make, have made, use, offer for sale, and sell products and services; provided that, during the term of this Agreement, Licensor shall have no right, under the license in this Section 2.7.1(b), to practice the Licensed Back Improvements with respect to AAVrh10 in the Commercial Field of Friedreich’s Ataxia (Systemic).

2.7.2 For purposes of this Agreement, “Licensed Back Improvements” means any patentable modifications or improvements developed by Licensee, any of its Affiliates, or any Sublicensees to any vector that is the subject of a claim within the Licensed Patents, which modification or improvement is developed by Licensee or any of its Affiliate during the term of this Agreement or by any Sublicensee during the term of any sublicense agreement with such Sublicensee.

2.7.3 Licensee agrees to provide prompt notice to Licensor upon the filing of any patent application covering any Licensed Back Improvement, together with a reasonably detailed description of or access to such Licensed Back Improvement to permit the practice of any such invention or improvement.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

2.8 Covenants Regarding In-Licenses. During the term of this Agreement, without the prior written consent of Licensee, which consent shall not be unreasonably withheld, Licensor agrees not to exercise its right to terminate and will not amend either the GSK Agreement or Penn Agreement if such termination or amendment would materially, adversely affect Licensee’s rights under this Agreement with respect to the Licensed Patents.

2.9 Section 365(n) of the Bankruptcy Code. All rights and licenses granted to Licensee or Licensor under or pursuant to this Agreement are and will otherwise be deemed to be, for purposes of Section 365(n) of the United States Bankruptcy Code (Title 11, U.S. Code), as amended (the “Bankruptcy Code”) or any comparable law outside the United States, licenses of rights to “intellectual property” as defined in Section 101(35A) of the Bankruptcy Code. The Parties will retain and may fully exercise all of their respective rights and elections under the Bankruptcy Code and any comparable law outside the United States.

ARTICLE 3: CONSIDERATION

3.1 Initial Fee. In consideration of the rights and licenses granted to Licensee under this Agreement, Licensee shall pay Licensor an initial fee of $600,000, of which ****will be paid upon the Effective Date and **** will be paid upon the earlier of (a) December 31, 2014 and (b) the closing of a transaction (or series of transactions) involving the issuance or sale of equity securities of Licensee pursuant to which Licensee receives proceeds of not less than US ****; provided that such **** portion of the initial fee will be immediately payable upon any termination of this Agreement prior to the earlier of those events.

3.2 Commercial Option Fee. If Licensee exercises the Commercial Option granted to Licensee under Section 2.3 with respect to Friedreich’s Ataxia (CNS), Licensee shall pay Licensor a fee of $300,000. For clarity, no such fee will be required with respect to exercising the Commercial Option with respect to Friedreich’s Ataxia (Systemic).

3.3 Annual Maintenance Fee. In consideration of the rights and licenses granted to Licensee under this Agreement, Licensee shall pay Licensor on-going annual maintenance fees no later than **** after each anniversary of the Effective Date. The annual maintenance fees will be as follows:

(a)

**** for Friedreich’s Ataxia (Systemic) and

(b)

if the Commercial Option with respect to Friedreich’s Ataxia (CNS) is exercised, then, following such exercise, **** for Friedreich’s Ataxia (CNS).

3.4 Milestone Fees. In consideration of the rights and licenses granted to Licensee under this Agreement, Licensee shall pay Licensor the following milestone payments on a per-Disease Indication basis for the first Licensed Product to achieve such milestone event:

3.4.1 Friedreich’s Ataxia (Systemic) Milestones.


Friedreich’s Ataxia (Systemic) Milestone		Milestone Payment

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


Friedreich’s Ataxia (Systemic) Milestone	Milestone Payment
1. First treatment of human subject in a clinical trial (i.e., first patient, first dose)		****
2. First treatment in Phase 3 Clinical Trial (i.e., first patient, first dose)		****
3. NDA submission in the United States		****
4. NDA submission in the European Union or the rest of the world (excluding the United States)		****
5. NDA approval in the United States		****
6. NDA approval in the European Union or the rest of the world (excluding the United States)		****

Total (per such Disease Indication):	$	8,850,000

3.4.2 Friedreich’s Ataxia (CNS).


Friedreich’s Ataxia (CNS) Milestone	Milestone Payment
1. First treatment of human subject in a clinical trial (i.e., first patient, first dose)		****
2. First treatment in Phase 3 Clinical Trial (i.e., first patient, first dose)		****
3. NDA submission in the United States		****
4. NDA submission in the European Union or the rest of the world (excluding the United States)		****
5. NDA approval in the United States		****
6. NDA approval in the European Union or the rest of the world (excluding the United States)		****

Total (per such Disease Indication):		$5,000,000

3.4.3 For clarity, the milestone payments set forth in Section 3.4.1 are payable **** with respect to Friedreich’s Ataxia (Systemic), and the milestone payments set forth in Section 3.4.2 are payable **** with respect to Friedreich’s Ataxia (CNS), in each case, with respect to the **** Licensed Product for such Disease Indication that achieves the milestone event, ****. To the extent that either of the two development milestones in Section 3.4.1 or 3.4.2 (i.e., first treatment of human subject in a clinical trial or first treatment in Phase 3 Clinical Trial in the applicable Disease Indication) has not been paid at the time of achievement of either NDA submission milestone within the same Disease Indication, then, upon the achievement of either of such NDA submission milestones, the preceding unpaid development milestone payments within such Disease Indication shall be made in addition to the payment corresponding to the NDA submission milestone that has been achieved.

3.5 Royalties.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

3.5.1 In consideration of the rights and licenses granted to Licensee under this Agreement, Licensee shall pay to Licensor the following royalties based upon the annual Net Sales worldwide of all Licensed Products in a given calendar year, subject to the reductions in royalty rates set forth in Section 3.5.2:


Cumulative Annual Net Sales of all Licensed Products Worldwide	Royalty Percentage
Portion of Net Sales less than $300,000,000		*	***
Portion of Net Sales between (and including) $300,000,000 through (and including) $600,000,0000		*	***
Portion of Net Sales greater than $600,000,000		*	***

3.5.2 Third Party Royalties Stacking Provision. If Licensee must obtain a license from a Third Party to avoid infringement of such Third Party’s rights in order to manufacture, use, or commercialize a given Licensed Product and if the royalties required to be paid to such Third Party for such license, together with those royalties payable to Licensor, in the aggregate, exceed **** of Net Sales for any Licensed Product, then the royalty owed to Licensor for that Licensed Product will be reduced by an amount calculated as follows:

STACKING ROYALTY CALCULATIONS

R = (C * (A / (A+B)))

Where

R = reduction of Licensor royalty,

A = unreduced Licensor royalty,

B = sum of all Third Party royalties,

C = increment of projected total royalty above ****

Example Calculation:


Assume:		i) all Third Party royalties = ****
		ii) unreduced Licensor royalty = ****
		iii) projected total royalty = ****


		R= (** – *) (** / ( + **))
		R = (**** * ****)
		R = ****
		Licensor Stacked Royalty = ** – = **%

Notwithstanding the foregoing, Licensee will pay to Licensor no less than **** of the royalties that Licensee would otherwise pay to Licensor with respect to Net Sales of Licensee if there were no royalties due to Third Parties.

3.5.3 Royalty Payment Period. Licensee’s obligation hereunder for payment of a royalty under this Section 3.5 on the Net Sales of Licensed Products in a given country will end on a Licensed Product-by-Licensed Product and country-by-country basis when the Licensed

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Product ceases to infringe or be covered by a Valid Claim within the Licensed Commercial Patents in that country. For clarity, only one royalty, determined in accordance with this Section 3.5, is payable on the Net Sales of any unit of a Licensed Product.

3.6 Sublicense Fees.

3.6.1 In further consideration of the rights and licenses granted to Licensee under this Agreement, Licensee will pay Licensor a percentage of any sublicense fees (****) received by Licensee or its Affiliates for the Licensed Commercial Patents from any Third Party Sublicensee or from any Third Party granted any option to obtain a sublicense. The applicable percentage due to Licensor for each sublicense (or option) in the Commercial Field of Friedreich’s Ataxia (CNS) shall be **** The applicable percentage due to Licensor for each sublicense (or option) in the Commercial Field of Friedreich’s Ataxia (Systemic) shall be as follows:


Friedreich’s Ataxia (Systemic)

Event	Sublicense Fee Rate

If sublicensed (or optioned) on or before ****		*	***

If sublicensed (or optioned) on or before ****		*	***

If sublicensed (or optioned) on or before ****		*	***

If sublicensed (or optioned) after ****		*	***

For the avoidance of doubt, with respect to an option to obtain a sublicense in the Commercial Field of Friedreich’s Ataxia (Systemic), if a sublicense is later granted as a result of the exercise of such option, the sublicense fees applicable to such sublicense will be determined by reference to ****.

3.6.2 With respect to the obligations under this Section 3.6, Licensee shall not be required to submit any amounts received from a Third Party for the following:

(a)

Reimbursement or payment, in either case, of Licensee’s actual costs for research, development, and/or manufacturing activities performed by Licensee or its Affiliates corresponding directly to the research, development and/or manufacturing of Licensed Products pursuant to a specific agreement;

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

(b)

Consideration received for the purchase of an equity interest in Licensee or its Affiliates at fair market value or in the form of loans at commercially reasonable rates of interest; and

(c)

Any and all amounts paid to Licensee or its Affiliates by a Third Party Sublicensee as royalties on sales of Licensed Product sold by such Sublicensee under a sublicense agreement.

3.6.3 If Licensee or its Affiliate receives sublicense fees from Third Party Sublicensees or from any Third Party granted any option to obtain a sublicense under this Agreement in the form of non-cash consideration, then, at Licensor’s option, Licensee shall pay Licensor payments as required by this Section 3.6 (a) in the form of the non-cash consideration received by Licensee or its Affiliates or (b) a cash payment determined based on the fair market value of such non-cash consideration. If Licensee or its Affiliate enters into any sublicense that is not an arm’s length transaction, fees due under this Section 3.6 will be calculated based on the fair market value of such transaction, at the time of the transaction, assuming an arm’s length transaction made in the ordinary course of business, as determined jointly and in good faith by Licensor and Licensee based on transactions of a similar type and standard industry practice, if any.

3.6.4 To the extent Licensee or its Affiliates receives payment from a Third Party relating to one or more of the milestone events set forth in the tables in Section 3.4, then the amount of the payment made to Licensor under such Section 3.4 with respect to such milestone event shall not be deemed sublicense fees under this Section 3.6; instead, the amounts due under this Section 3.6 shall be calculated by applying the applicable sublicense fee rate set forth in Section 3.6.1 above to the sublicense fees received by Licensee or its Affiliates from such Third Party after deducting the amount of the payment under Section 3.4.

3.7 Reports and Records.

3.7.1 Licensee must deliver to Licensor within **** after the end of each Calendar Quarter after the first commercial sale of a Licensed Product a report setting forth the calculation of the royalties due to Licensor for such Calendar Quarter, including:

(a)

Number of Licensed Products included within Net Sales, listed by country;

(b)

Gross consideration for Net Sales of Licensed Product, including all amounts invoiced, billed, or received;

(c)

Qualifying costs to be excluded from the gross consideration, as described in Section 1.25, listed by category of cost;

(d)

Net Sales of Licensed Products listed by country;

(e)

A detailed accounting of any royalty reductions applied pursuant to Section 3.5.2;

(f)

Royalties owed to Licensor, listed by category; and

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

(g)

The computations for any applicable currency conversions.

3.7.2 Licensee shall pay the royalties due under Section 3.5 within **** following the last day of the Calendar Quarter in which the royalties accrue. Licensee shall send the royalty payments along with the report described in Section 3.7.1.

3.7.3 Within **** after the occurrence of a milestone event described in Section 3.4, Licensee must deliver to Licensor a report describing the milestone event that occurred, together with a payment of the applicable amount due to Licensor pursuant to Section 3.4.

3.7.4 Within **** after the receipt of any fees from any Third Party as described in Section 3.6, Licensee must deliver to Licensor a report describing the fees received, together with a payment of the applicable amount due to Licensor pursuant to Section 3.6.

3.7.5 All financial reports under this Section 3.7 will be certified by the chief financial officer of Licensee.

3.7.6 Licensee shall maintain and require its Affiliates and all Sublicensees to maintain, complete and accurate books and records which enable the royalties, fees, and payments payable under this Agreement to be verified. The records must be maintained for **** after the submission of each report under Article 3. Upon reasonable prior written notice to Licensee, Licensee and its Affiliates and all Sublicensees will provide Licensor and/or the ReGenX Licensors (and their respective accountants) with access to all of the relevant books, records, and related background information required to conduct a review or audit of the royalties, fees, and payments payable to Licensor under this Agreement to be verified. Access will be made available: (a) during normal business hours; (b) in a manner reasonably designed to facilitate the auditing party’s review or audit without unreasonable disruption to Licensee’s business; and (c) no more than once each calendar year during the term of this Agreement and for a period of five years thereafter. Licensee will promptly pay to Licensor the amount of any underpayment determined by the review or audit, plus accrued interest. If the review or audit determines that Licensee has underpaid any payment by **** or more, then Licensee will also promptly pay the costs and expenses of Licensor and the ReGenX Licensors and their respective accountants in connection with the review or audit.

3.8 Currency, Interest.

3.8.1 All dollar amounts referred to in this Agreement are expressed in United States dollars. All payments to Licensor under this Agreement must be made in United States dollars.

3.8.2 If Licensee receives payment in a currency other than United States dollars for which a royalty or fee or other payment is owed under this Agreement, then (a) the payment will be converted into United States dollars at the conversion rate for the foreign currency as published in the eastern edition of the Wall Street Journal, N.Y. edition, as of the last business day of the Calendar Quarter in which the payment was received by Licensee; and (b) the conversion computation will be documented by Licensee in the applicable report delivered to Licensor under Section 3.7.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

3.8.3 All amounts that are not paid by Licensee when due will accrue interest from the date due until paid at a rate equal to 1.5% per month (or the maximum allowed by law, if less).

3.9 Taxes and Withholding.

3.9.1 All payments hereunder will be made free and clear of, and without deduction or deferment in respect of, and Licensee shall pay and be responsible for, and shall hold Licensor harmless from and against, any taxes, duties, levies, fees, or charges, including sales, use, transfer, excise, import, and value added taxes (including any interest, penalties, or additional amounts imposed with respect thereto) but excluding withholding taxes to the extent provided in Section 3.9.2. At the request of Licensee, Licensor will give Licensee such reasonable assistance, which will include the provision of documentation as may be required by the relevant tax authority, to enable Licensee to pay and report and, as applicable, claim exemption from or reduction of, such tax, duty, levy, fee, or charge.

3.9.2 If any payment made by Licensee hereunder becomes subject to withholding taxes with respect to Licensor’s gross or net income under the laws of any jurisdiction, Licensee will deduct and withhold the amount of such taxes for the account of Licensor to the extent required by law and will pay the amounts of such taxes to the proper governmental authority in a timely manner and promptly transmit to Licensor appropriate proof of payment of such withholding taxes. At the request of Licensor, Licensee will give Licensor such reasonable assistance, which will include the provision of appropriate certificates of such deductions made together with other supporting documentation as may be required by the relevant tax authority, to enable Licensor to claim exemption from or reduction of, or otherwise obtain repayment of, such withholding taxes, and will upon request provide such additional documentation from time to time as is reasonably required to confirm the payment of withholding tax.

ARTICLE 4: DILIGENCE

4.1 Diligence Obligations. Licensee will use commercially reasonable efforts to develop, commercialize, market, promote, and sell Licensed Products for Friedreich’s Ataxia (Systemic) in the Commercial Field. Furthermore, if Licensee exercises the Commercial Option granted to Licensee under Section 2.3 with respect to Friedreich’s Ataxia (CNS), Licensee will use commercially reasonable efforts to develop, commercialize, market, promote, and sell Licensed Products for Friedreich’s Ataxia (CNS) in the Commercial Field. Commercially reasonable efforts means efforts equivalent to those utilized by ****. Without limiting the foregoing, Licensee will meet the following:

(a)

acceptance by the FDA of an Investigational New Drug application, or acceptance by the European Medicines Agency (or any successor entity thereto) of an equivalent application, for a Licensed Product using AAVrh10 for Friedreich’s Ataxia (Systemic) by no later than **** after the Effective Date; and

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

(b)

if Licensee exercises the Commercial Option granted to Licensee under Section 2.3 with respect to Friedreich’s Ataxia (Systemic), acceptance by the FDA of an Investigational New Drug application, or acceptance by the European Medicines Agency (or any successor entity thereto) of an equivalent application, for a Licensed Product using the Specified Vector selected in the exercise of such Commercial Option for Friedreich’s Ataxia (Systemic) by no later than **** after the Grant Date;

provided, however, that, if Licensee expects not to achieve one of the milestones set forth in clause (a) or (b) on or before the specified deadline in such clause (a) or (b), Licensee may pay Licensor an extension fee of **** on or before such deadline and the relevant deadline in clause (a) or (b), as applicable, shall then be extended by an additional ****. Licensee will only be entitled to **** for **** of the milestones in clauses (a) and (b), ****.

4.2 Reporting. Within **** after the Effective Date and within **** of each December 1 thereafter, Licensee shall provide Licensor with written progress reports, setting forth in such detail as Licensor may reasonably request, the progress of the development, evaluation, testing, and commercialization of each Licensed Product. Licensee will also notify Licensor within **** of the first commercial sale by Licensee, its Affiliates, or any Sublicensees of each Licensed Product. Such a report (“Development Progress Report”), setting forth the current stage of development of Licensed Products, shall include:

4.2.1 Date of Development Progress Report and time covered by such report;

4.2.2 Major activities and accomplishments completed by Licensee, its Affiliates, and any Sublicensees relating directly to the Licensed Product since the last Development Progress Report;

4.2.3 Significant research and development projects relating directly to the Licensed Product currently being performed by Licensee, its Affiliates, and any Sublicensees and good faith, but non-binding, projected dates of completion;

4.2.4 A development plan covering the next two years at least, which will include future development activities to be undertaken by Licensee, its Affiliates, or any Sublicensees during the next reporting period relating directly to the Licensed Product, Licensee’s strategy to bring the Licensed Product to commercialization, and projected timeline for completing the necessary tasks to accomplish the goals of the strategy;

4.2.5 Projected total development remaining before product launch of each Licensed Product; and

4.2.6 Summary of significant development efforts using the Licensed Patents being performed by Third Parties, including the nature of the relationship between Licensee and such Third Parties.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

4.3 Confidential Information. The Parties agree that Development Progress Reports shall be deemed Licensee’s Confidential Information; provided that Licensor may share a copy of such reports with the ReGenX Licensors.

4.4 Improvements. Simultaneously with the Development Progress Report, Licensee shall deliver a detailed description of any Licensed Back Improvements, if not previously provided pursuant to Section 2.7.3.

ARTICLE 5: CONFIDENTIALITY

5.1 Treatment of Confidential Information. Each Party, as a receiving party (a “Receiving Party”), agrees that it will (a) treat Confidential Information of the other Party (the “Disclosing Party”) as strictly confidential; (b) not disclose such Confidential Information to Third Parties without the prior written consent of the Disclosing Party, except as may be permitted in this Agreement; provided that any disclosure permitted hereunder be under confidentiality agreements with provisions at least as stringent as those contained in this Agreement; and (c) not use such Confidential Information for purposes other than those authorized expressly in this Agreement. The Receiving Party agrees to ensure that its employees who have access to Confidential Information are obligated in writing to abide by confidentiality obligations at least as stringent as those contained under this Agreement.

5.2 Public Announcements.

5.2.1 The Parties agree they will release a joint press release in the form attached hereto as Exhibit C. Except as provided in Section 5.2.2, any other press releases by either Party with respect to the other Party or any other public disclosures concerning the existence of or terms of this Agreement shall be subject to review and approval by the other Party. Once the joint press release or any other written statement is approved for disclosure by both Parties, either Party may make subsequent public disclosure of the contents of such statement without the further approval of the other Party.

5.2.2 Notwithstanding Section 5.2.1, Licensor has the right to publish (through press releases, scientific journals, or otherwise) and refer to any clinical, regulatory, or research results related to Licensee’s Licensed Product or Specified Vector program that have been publicly disclosed by Licensee, including referring to Licensee by name as a licensee of Licensor, which publication or referral by Licensor shall not require the prior consent of Licensee, but Licensor will provide Licensee with a copy of any such publications or referrals two business days prior to release.

5.3 Authorized Disclosure. Notwithstanding the provisions of Section 5.1 or 5.2, either Party may disclose the other’s Confidential Information or make such a disclosure of the existence of and/or terms of this Agreement to any ****; provided that, in each case, such recipient of Confidential Information is obligated to keep such information confidential on terms no less stringent than those set forth in this Agreement. Furthermore, Licensee agrees that Licensor may share a copy of this Agreement, reports and

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

notices provided by Licensee to Licensor pursuant to the terms of this Agreement, and copies of sublicense agreements provided to Licensor hereunder, with the ReGenX Licensors to the extent required by the GSK Agreement and Penn Agreement. In the event that the Receiving Party receives service of legal process that purports to compel disclosure of the Disclosing Party’s Confidential Information or becomes obligated by law to disclose the Confidential Information of the Disclosing Party or the existence of or terms of this Agreement to any governmental authority, then, to the extent legally permitted, the Receiving Party shall promptly notify the Disclosing Party, so that the Disclosing Party may seek an appropriate protective order or other remedy with respect to narrowing the scope of such requirement and/or waive compliance by the Receiving Party with the provisions of this Agreement. The Receiving Party will, at the Disclosing Party’s request and expense, provide the Disclosing Party with reasonable assistance in obtaining such protective order or other remedy. If, in the absence of such protective order or other remedy, the Receiving Party is nonetheless required by law to disclose the existence of or terms of this Agreement or other Confidential Information of the Disclosing Party, the Receiving Party may disclose such Confidential Information without liability hereunder; provided that the Receiving Party shall furnish only such portion of the Confidential Information that is legally required to be disclosed and only to the extent required by law.

5.4 Term of Confidentiality. The obligations of this Article 5 shall continue for a period of **** following the expiration or termination of this Agreement.

ARTICLE 6: TERM AND TERMINATION

6.1 Term of Agreement. This Agreement, unless sooner terminated as provided in this Agreement, expires upon the expiration, lapse, abandonment, or invalidation of the last Valid Claim of the Licensed Commercial Patents to expire, lapse, or become abandoned or unenforceable in all the countries of the world.

6.2 Termination for Failure to Exercise Option. This Agreement will terminate automatically with respect to Friedreich’s Ataxia (CNS) at the end of the Research Term for Friedreich’s Ataxia (CNS) if Licensee does not exercise the Commercial Option for Friedreich’s Ataxia (CNS) in accordance with Section 2.3. This Agreement will terminate automatically with respect to Friedreich’s Ataxia (Systemic) with respect to any Specified Vector (other than AAVrh10) at the end of the Research Term for Friedreich’s Ataxia (Systemic) if Licensee does not exercise the Commercial Option for Friedreich’s Ataxia (Systemic) in accordance with Section 2.3; provided that such termination will not affect Licensee’s rights under this Agreement with respect to the license granted under Section 2.1.

6.3 Licensee’s Right to Terminate. Licensee may, upon six months’ prior written notice to Licensor, terminate this Agreement for any reason, with or without cause; provided that, if such termination notice is sent prior to the first anniversary of the Effective Date, such termination notice shall be accompanied by Licensee’s payment of $500,000 in satisfaction of the remainder of the initial fee under Section 3.1. In exercising such termination right, Licensee may terminate the Agreement in its entirety or, if desired, Licensee may specify in the written notice that this Agreement is terminating only with respect to one or more of the Disease Indications within the Research Field or Commercial Field, as applicable.

CONFIDENTIAL TREATMENT REQUESTED

6.4 Termination for Breach.

6.4.1 Licensor may terminate this Agreement, if Licensee is late in paying to Licensor royalties, fees, or any other monies due under this Agreement, and Licensee does not pay Licensor in full within 15 days upon written demand from Licensor, which termination shall be effective immediately upon the expiration of such 15-day cure period.

6.4.2 Either Party may terminate this Agreement, if the other Party materially breaches this Agreement and does not cure such material breach within 30 days after written notice of the breach, which termination shall be effective immediately upon the expiration of such 30-day cure period.

6.4.3 If the allegedly breaching Party disputes in good faith the allegation of breach or non-cure prior to the expiration of the applicable cure period, this Agreement shall not be terminated until such dispute is resolved in favor of the non-breaching Party in accordance with Section 10.6, and the breaching Party has not cured such material breach within an additional 15 days, or such payment breach within an additional 30 days, after such resolution; provided that Licensor shall be entitled to terminate this Agreement at the end of the original 30-day or 15-day, as applicable, cure period, without waiting for resolution of the dispute in accordance with Section 10.6 if the breach by Licensee of this Agreement would cause Licensor to be in breach of the GSK Agreement or the Penn Agreement.

6.5 Termination for Insolvency.

6.5.1 Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, if Licensee, any of its Affiliates, or any Sublicensees experiences any Trigger Event.

6.5.2 For purposes of this Section 6.5, “Trigger Event” means any of the following: (a) if Licensee, any Affiliate, or any Sublicensee, as applicable, (i) becomes insolvent, becomes bankrupt, or generally fails to pay its debts as such debts become due, (ii) is adjudicated insolvent or bankrupt, (iii) admits in writing its inability to pay its debts, (iv) suffers the appointment of a custodian, receiver, or trustee for it or its property and, if appointed without its consent, is not discharged within 30 days, (v) makes an assignment for the benefit of creditors, or (vi) suffers proceedings being instituted against it under any law related to bankruptcy, insolvency, liquidation, or the reorganization, readjustment, or release of debtors and, if contested by it, not dismissed or stayed within ten days; (b) the institution or commencement by Licensee, any Affiliate, or any Sublicensee, as applicable, of any proceeding under any law related to bankruptcy, insolvency, liquidation, or the reorganization, readjustment, or release of debtors; (c) the entering of any order for relief relating to any of the proceedings described in Section 6.5.2(a) or (b) above; (d) the calling by Licensee, any Affiliate, or any Sublicensee, as applicable, of a meeting of its creditors with a view to arranging a composition or adjustment of its debts; or (e) the act or failure to act by Licensee, any Affiliate, or any Sublicensee, as applicable, indicating its consent to, approval of, or acquiescence in any of the proceedings described in Section 6.5.2(b) through (d) above.

6.6 Patent Challenge.

CONFIDENTIAL TREATMENT REQUESTED

6.6.1 Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, upon the commencement by Licensee, any of its Affiliates, or any Sublicensee of a Patent Challenge.

6.6.2 For purposes of this Section 6.6, “Patent Challenge” means any action against Licensor, The Trustees of the University of Pennsylvania, or the ReGenX Licensors, including an action for declaratory judgment, to declare or render invalid or unenforceable the Licensed Patents, or any claim thereof.

6.7 Effects of Termination. The effect of termination pursuant to Section 6.2, by Licensee pursuant to Section 6.3, by either Party, as applicable, under Section 6.4, or by Licensor pursuant to Section 6.5 or 6.6 shall be as follows:

6.7.1 The licenses granted by Licensor hereunder shall terminate, and Licensee, its Affiliates, and (unless the sublicense agreement is assigned pursuant to Section 6.7.2) all Sublicensees shall cease to make, have made, use, import, sell, and offer for sale all AAV Materials or Licensed Products and shall cease to otherwise practice the Licensed Patents; provided that Licensee, its Affiliates, and Sublicensees shall have the right to continue to sell their existing inventories of Licensed Products for a period not to exceed **** after the effective date of such termination;

6.7.2 At Licensor’s request, Licensee shall assign to Licensor any or all sublicenses granted to Third Parties to the extent of the rights licensed to Licensee hereunder and sublicensed to the Sublicensee; provided that (i) prior to such assignment, Licensee shall advise Licensor whether such Sublicensee is then in full compliance with all terms and conditions of its sublicense and continues to perform thereunder, and, if such Sublicensee is not in full compliance or is not continuing to perform, Licensor may elect not to have such sublicense assigned; and (ii) following such assignment, Licensor shall not be liable to such Sublicensee with respect to any obligations of Licensee to the Sublicensee that are not consistent with, or not required by, Licensor’s obligations to Licensee under this Agreement; and all sublicenses not requested to be assigned to Licensor shall terminate;

6.7.3 If termination is by Licensee pursuant to Section 6.3 or by Licensor pursuant to Section 6.4, 6.5, or 6.6, Licensee shall grant, and hereby grants to Licensor a non-exclusive, perpetual, irrevocable, worldwide, royalty-free, transferable, sublicensable license under any patentable modifications or improvements (and any intellectual property rights with respect thereto) developed by Licensee or any Affiliates (during the term of this Agreement) or by any Sublicensees (during the term of any sublicense agreement with such Sublicensee) to any vector that is the subject of a claim within any of the Licensed Patents, for use by Licensor for the research, development, and commercialization of products in any therapeutic indication;

6.7.4 Licensee shall pay all monies then-owed to Licensor under this Agreement;

6.7.5 Each Receiving Party shall, at the Disclosing Party’s request, return all Confidential Information of the Disclosing Party. Notwithstanding the foregoing, one copy may be kept by either Party for a record of that Party’s obligations; and

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

6.7.6 If termination is only with respect to a particular Disease Indication within the Research Field or the Commercial Field, but not all Disease Indications, then the provisions of this Section 6.7 shall only apply with respect to the terminated Disease Indications, and this Agreement shall continue with respect to the non-terminated Disease Indications.

6.8 Survival. Licensee’s obligation to pay all monies due and owed to Licensor under this Agreement which have matured as of the effective date of termination or expiration shall survive the termination or expiration of this Agreement. In addition, the provisions of Section 2.4, (Retained Rights), Section 2.5 (Government Rights), Section 2.7 (Improvements), Article 3 (Consideration) (with respect to any final reports or to the extent any amounts are due but unpaid), Section 3.7 (Reports and Records), Section 4.3 (Confidential Information), Article 5 (Confidentiality), Article 6 (Term and Termination), Section 8.3 (Disclaimer of Warranties, Damages), Section 8.4 (Indemnification), Section 8.5 (Insurance), Article 9 (Use of Name), and Article 10 (Additional Provisions) shall survive such termination or expiration of this Agreement in accordance with their respective terms.

ARTICLE 7: PATENT MAINTENANCE; PATENT INFRINGEMENT

7.1 Prosecution of Licensed Patents. As between Licensor and Licensee, but subject to any obligations of Licensor to the ReGenX Licensors, the Parties agree as follows:

7.1.1 Licensor shall have the sole right, but not the obligation, to Prosecute patent applications and issued patents within Licensed Patents, in Licensor’s sole discretion.

7.1.2 Nothing in this Agreement obligates Licensor to continue to Prosecute any patent applications or issued patents, and Licensee acknowledges that Licensor shall have no obligation to undertake any inter-party proceedings, such as oppositions or interferences, or to undertake any re-examination or re-issue proceedings, in either case, with respect to the Licensed Patents.

7.2 Infringement Actions Against Third Parties.

7.2.1 Licensee is responsible for notifying Licensor promptly of any infringement of Licensed Patents (other than Retained Rights) that may come to Licensee’s attention.

7.2.2 As between Licensor and Licensee, but subject to any obligations of Licensor to the ReGenX Licensors, Licensor shall have the sole right, but not the obligation, to prosecute any such infringement at its **** recovered in connection therewith. In any action to enforce any of the Licensed Patents, Licensee, at the request and expense of Licensor, shall cooperate to the fullest extent reasonably possible, including in the event that, if Licensor is unable to initiate or prosecute such action solely in its own name, Licensee shall join such action voluntarily and shall execute all documents necessary to initiate litigation to prosecute, maintain, and settle such action. Nothing in this Agreement obligates Licensor to bring or prosecute lawsuits against Third Parties for infringement of any Licensed Patents.

7.2.3 Licensee shall have no right to undertake prosecution of any such infringement.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

7.3 Defense of Infringement Claims. In the event Licensee or Licensor becomes aware that Licensee’s or any of its Affiliates’ or any Sublicensees’ practice of the Licensed Patents is the subject of a claim for patent infringement by a Third Party, that Party shall promptly notify the other, and the Parties shall consider the claim and the most appropriate action to take. Licensee shall cause each of its Affiliates and each Sublicensee to notify Licensee promptly in the event such entity becomes aware that its practice of the Licensed Patents is the subject of a claim of patent infringement by another. To the extent Licensor takes any action, Licensor (or the ReGenX Licensors) shall have the right to require Licensee’s reasonable cooperation in any such suit, upon written notice to Licensee; and Licensee shall have the obligation to participate upon Licensor’s request, in which event, Licensor shall bear the cost of Licensee’s participation. Without Licensor’s prior written permission, Licensee must not settle or compromise any such suit in a manner that imposes any material obligations or restrictions on Licensor or the ReGenX Licensors or grants any rights to the Licensed Patents other than rights that Licensee has the right to grant under this Agreement.

ARTICLE 8: REPRESENTATIONS AND WARRANTIES; INDEMNIFICATION

8.1 Representations and Warranties by Licensor. Licensor represents and warrants to Licensee as of the Effective Date:

8.1.1 Licensor has the right, power, and authority to enter into this Agreement and to grant to Licensee the licenses specified in this Agreement;

8.1.2 This Agreement when executed shall become the legal, valid, and binding obligation of it, enforceable against it, in accordance with its terms;

8.1.3 There are no actions, suits, proceedings, or arbitrations pending or, to Licensor’s knowledge, threatened against Licensor relating to the Licensed Research Patents that would be inconsistent with the rights granted to Licensee under this Agreement;

8.1.4 To Licensor’s knowledge, (a) the Licensed Research Patents are solely owned by the Trustees of the University of Pennsylvania, and (b) no Third Party (other than the ReGenX Licensors) has any right, interest, or claim in or to such Licensed Research Patents with respect to the Disease Indications that are inconsistent with those granted to Licensee with respect to the Disease Indications;

8.1.5 To Licensor’s knowledge, no Third Party is infringing any of the Licensed Research Patents in a manner that is inconsistent with the scope of rights granted to Licensee with respect to the Disease Indications; and

8.1.6 Licensor has not received any written notice from any Third Party patentee alleging infringement of such Third Party’s patents by the practice of the Licensed Research Patents with respect to the Disease Indications.

8.2 Representations and Warranties by Licensee. Licensee represents and warrants to Licensor as of the Effective Date that:

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8.2.1 Licensee has the right, power, and authority to enter into this Agreement and to grant the licenses granted by it hereunder;

8.2.2 This Agreement when executed shall become the legal, valid, and binding obligation of it, enforceable against it, in accordance with its terms;

8.2.3 Licensee has the ability and the resources, including financial resources, necessary to carry out its obligations under this Agreement; and

8.2.4 There are no actions, suits, proceedings, or arbitrations pending or, to Licensee’s knowledge, threatened against Licensee that would impact activities under this Agreement.

8.3 Disclaimer of Warranties, Damages. EXCEPT AS SET FORTH IN SECTION 8.1, THE LICENSED PATENTS, AAV MATERIALS, LICENSED PRODUCTS, AND ALL RIGHTS LICENSED UNDER THIS AGREEMENT ARE PROVIDED ON AN “AS IS” BASIS, AND LICENSOR MAKES NO REPRESENTATIONS OR WARRANTIES, EXPRESS OR IMPLIED, WITH RESPECT THERETO. BY WAY OF EXAMPLE BUT NOT OF LIMITATION, LICENSOR MAKES NO REPRESENTATIONS OR WARRANTIES, AND HEREBY DISCLAIMS ALL EXPRESS AND IMPLIED REPRESENTATIONS AND WARRANTIES, (i) OF COMMERCIAL UTILITY, ACCURACY, COMPLETENESS, PERFORMANCE, MERCHANTABILITY, FITNESS FOR A PARTICULAR PURPOSE, VALIDITY OR ENFORCEABILITY OF THE LICENSED PATENTS, AND PROFITABILITY; OR (ii) THAT THE USE OF THE LICENSED PATENTS, AAV MATERIALS, OR LICENSED PRODUCTS WILL NOT INFRINGE ANY PATENT, COPYRIGHT, TRADEMARK, OR OTHER PROPRIETARY RIGHTS OF THIRD PARTIES. EXCEPT AS SET FORTH HEREIN, NONE OF LICENSOR OR EITHER OF THE REGENX LICENSORS SHALL BE LIABLE TO LICENSEE, LICENSEE’S SUCCESSORS OR ASSIGNS, ANY SUBLICENSEES, OR ANY THIRD PARTY WITH RESPECT TO: (a) ANY CLAIM ARISING FROM USE OF THE LICENSED PATENTS, AAV MATERIALS, LICENSED PRODUCTS, AND ANY OR ALL RIGHTS LICENSED UNDER THIS AGREEMENT OR FROM THE DEVELOPMENT, TESTING, MANUFACTURE, USE, OR SALE OF AAV MATERIALS OR LICENSED PRODUCTS; OR (b) ANY CLAIM FOR LOSS OF PROFITS, LOSS OR INTERRUPTION OF BUSINESS, OR FOR INDIRECT, SPECIAL, INCIDENTAL, PUNITIVE, OR CONSEQUENTIAL DAMAGES OF ANY KIND, INCLUDING ANY ARISING FROM OR RELATING TO ANY BREACH OF THIS AGREEMENT OR THE EXERCISE OF RIGHTS HEREUNDER, REGARDLESS OF ANY NOTICE OF SUCH DAMAGES. NOTHING IN THIS SECTION 8.3 IS INTENDED TO LIMIT OR RESTRICT THE INDEMNIFICATION RIGHTS OR OBLIGATIONS OF EITHER PARTY UNDER SECTION 8.4 OR TO LIMIT A PARTY’S LIABILITY FOR BREACHES OF ITS OBLIGATION REGARDING CONFIDENTIALITY UNDER ARTICLE 5.

8.4 Indemnification.

8.4.1 By Licensee. Licensee shall defend, indemnify, and hold harmless Licensor, the ReGenX Licensors, and their respective shareholders, members, officers, trustees, faculty, students, contractors, agents, and employees (individually, a “Licensor Indemnified Party” and, collectively, the “Licensor Indemnified Parties”) from and against any and all Third Party

CONFIDENTIAL TREATMENT REQUESTED

liability, loss, damage, action, claim, fee, cost, or expense (including attorneys’ fees) (individually, a “Third Party Liability” and, collectively, the “Third Party Liabilities”) suffered or incurred by the Licensor Indemnified Parties from claims of such Third Parties to the extent that such claims result from or arise out of: ****; provided, however, that Licensee shall not be liable for claims to the extent based on (1) any breach by Licensor of the representations, warranties, or obligations of Licensor under this Agreement or (2) the gross negligence or intentional misconduct of any of the Licensor Indemnified Parties. Without limiting the foregoing, but subject to clauses (1) and (2) above, Licensee must defend, indemnify, and hold harmless the Licensor Indemnified Parties from and against any Third Party Liabilities resulting from:

(a)

any **** or other claim of any kind related to the **** by a Third Party of a Licensed Product that was **** by Licensee, its Affiliates, any Sublicensees, their respective assignees, or vendors;

(b)

any claim by a Third Party that the ****; and

(c)

**** conducted by or on behalf of Licensee, its Affiliates, any Sublicensees, their respective assignees, or vendors relating to the Licensed Patents, AAV Materials, or Licensed Products, including any claim by or on behalf of a ****.

8.4.2 By Licensor. Licensor shall defend, indemnify, and hold harmless Licensee, its shareholders, members, officers, contractors, agents, and employees (individually, a “Licensee Indemnified Party” and, collectively, the “Licensee Indemnified Parties”) from and against any and all Third Party Liabilities suffered or incurred by the Licensee Indemnified Parties from claims of such Third Parties to the extent that such claims result from or arise out of: ****; provided, however, that Licensor shall not be liable for claims to the extent based on (1) any breach by Licensee of the representations, warranties, or obligations of Licensee under this Agreement or (2) the gross negligence or intentional misconduct of any of the Licensee Indemnified Parties.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

8.4.3 Indemnification Procedure. Each Party, as an indemnifying party (an “Indemnifying Party”), shall not be permitted to settle or compromise any claim or action giving rise to Third Party Liabilities in a manner that imposes any restrictions or obligations on any indemnified party (an “Indemnified Party”) without the Indemnified Party’s prior written consent or, if Licensee is the Indemnifying Party, that imposes any restrictions or obligations on Licensor’s direct or indirect licensors or grants any rights to the Licensed Patents or Licensed Products other than those Licensee has the right to grant under this Agreement without Licensor’s prior written consent. The Indemnifying Party shall be permitted to control any litigation or potential litigation involving the defense of any claim subject to indemnification pursuant to this Section 8.4, including the selection of counsel, with the reasonable approval of the Indemnified Party. Upon the Indemnifying Party’s reasonable request, the Indemnified Parties will reasonably cooperate with the Indemnifying Party in the defense and settlement of any such claim, at the Indemnifying Party’s cost and expense. If an Indemnifying Party fails or declines to assume the defense of any such claim or action within **** after notice thereof, the Indemnified Party may assume the defense of such claim or action at the cost and risk of the Indemnifying Party, and any Third Party Liabilities related thereto shall be conclusively deemed a Third Party Liability of the Indemnifying Party. The indemnification rights of a Indemnified Party contained in this Agreement are in addition to all other rights that such Indemnified Party may have at law or in equity or otherwise. The Indemnifying Party will pay directly all Third Party Liabilities incurred for defense or negotiation of any claim hereunder or will reimburse the Indemnified Party for all documented Third Party Liabilities incident to the defense or negotiation of any such claim within **** after the Indemnifying Party’s receipt of invoices for such fees, expenses, and charges.

8.5 Insurance. Licensee will procure and maintain insurance policies for the following coverages with respect to product liability, personal injury, bodily injury, and property damage arising out of Licensee’s (and its Affiliates’ and any Sublicensees’) performance under this Agreement: (a) during the term of this Agreement, comprehensive general liability, including broad form and contractual liability, in a minimum amount of **** combined single limit per occurrence (or claim) and in the aggregate annually; (b) prior to the commencement of clinical trials involving Licensed Products and thereafter for a period of not less than **** (or such longer period as Licensee is required by applicable law to continue to monitor the participants in the clinical trial), clinical trials coverage in amounts that are reasonable and customary in the U.S. pharmaceutical industry, subject always to a minimum limit of $3,000,000 combined single limit per occurrence (or claim) and in the aggregate annually; and (c) from prior to the first commercial sale of a Licensed Product until **** after the last sale of a Licensed Product, product liability coverage, in amounts that are reasonable and customary in the U.S. pharmaceutical industry, subject always to a minimum limit of **** combined single limit per occurrence (or claim) and in the aggregate annually. Licensor may review periodically the adequacy of the minimum amounts of insurance for each coverage required by this Section 8.5, and Licensor reserves the right to require Licensee to adjust the limits accordingly. The required minimum amounts of insurance do not constitute a limitation on Licensee’s liability or indemnification obligations to the Licensor Indemnified Parties under this Agreement. The policies of insurance required by this Section 8.5 will be issued by an insurance carrier with an A.M. best rating of **** or better and will name Licensor as an additional insured with respect to Licensee’s performance (and its Affiliates’ and any Sublicensees’) under this Agreement. Licensee will provide Licensor with insurance certificates evidencing the required coverage

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

within **** after the Effective Date and the commencement of each policy period and any renewal periods. Each certificate will provide that the insurance carrier will notify Licensor in writing at least **** prior to the cancellation or material change in coverage. Licensee will cause all Sublicensees to comply with the terms of this Section 8.5 to the same extent as Licensee.

ARTICLE 9: USE OF NAME

Licensee, its Affiliates, any Sublicensees, and all of its and their employees and agents must not use Licensor’s, the University of Pennsylvania’s, or SmithKline Beecham Corporation’s name, seal, logo, trademark, or service mark (or any adaptation thereof) or the name, seal, logo, trademark, or service mark (or any adaptation thereof) of any of such entities’ representative, school, organization, employee, or student in any way without the prior written consent of Licensor or such entity, as applicable; provided, however that Licensee may acknowledge the existence and general nature of this Agreement, subject to Section 5.3.

ARTICLE 10: ADDITIONAL PROVISIONS

10.1 Relationship. Nothing in this Agreement shall be deemed to establish a relationship of principal and agent between Licensee and Licensor, nor any of their agents or employees for any purpose whatsoever, nor shall this Agreement be construed as creating any other form of legal association or arrangement which would impose liability upon one Party for the act or failure to act of the other Party.

10.2 Assignment. The rights and obligations of Licensee and Licensor hereunder shall inure to the benefit of, and shall be binding upon, their respective permitted successors and assigns. Licensee may not assign this Agreement or any of its rights or obligations under this Agreement without the prior written consent of Licensor; provided, however, that Licensee may assign this Agreement, without Licensor’s prior written consent, pursuant to a merger or sale of all or substantially all of the assets of Licensee to which the Agreement relates; provided that, as part of any permitted assignment, (a) Licensee provides Licensor with written notice of such assignment at least five business days prior to the effectiveness of such assignment, and (b) Licensee requires any such assignee to agree in writing to be legally bound by this Agreement to the same extent as Licensee and provides Licensor with a copy of such assignee undertaking. In addition, Licensee will provide Licensor with written notice of any change of control (i.e., the acquisition by a person or group of “control” of Licensee, as defined in Section 1.7) of Licensee at least five business days prior to the effectiveness of such change of control. Licensor may assign this Agreement and its rights and obligations without the consent of Licensee. No assignment shall relieve the assigning Party of responsibility for the performance of any accrued obligations which it has prior to such assignment. Any attempted assignment by Licensee in violation of this Section 10.2 shall be null and void and of no legal effect.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

10.4 Notices. Notices, payments, statements, reports, and other communications under this Agreement shall be in writing and shall be deemed to have been received as of the date received if sent by public courier (e.g., Federal Express), by Express Mail, receipt requested, or by facsimile (with a copy of such facsimile also sent by one of the other methods of delivery) and addressed as follows:


If for Licensor:		with a copy to:

ReGenX Biosciences, LLC		ReGenX Biosciences, LLC
750 17th Street, NW		750 17th Street, NW
Suite 1100		Suite 1100
Washington, DC 20006		Washington, DC 20006
USA		USA
Attn: Chief Executive Officer		Attn: General Counsel
Telephone: 202-785-7438		Telephone: 202-785-7438
Facsimile: 202-785-7439		Facsimile: 202-785-7439

If for Licensee:		with a copy to:

AAVLife		WilmerHale
183/189 avenue de Choisy		60 State Street
75013 Paris		Boston, MA 02109
France		USA
Attn: Amber Salzman		Attn: Belinda M. Juran, Esq.
Telephone: 610-659-1098		Telephone: 617-526-6987
Facsimile: [ ]		Facsimile: 617-526-5000

Either Party may change its official address upon written notice to the other Party in accordance with this Section 10.4.

10.5 Applicable Law. This Agreement shall be construed and governed in accordance with the laws of the State of New York, without giving effect to conflict of law provisions that may require the application of the laws of another jurisdiction. Subject to Section 10.6, the Parties hereby submit to the exclusive jurisdiction of and venue in the courts located in the State of New York with respect to any and all disputes concerning the subject of this Agreement.

10.6 Dispute Resolution. In the event of any controversy or claim arising out of or relating to this Agreement, the Parties shall first attempt to resolve such controversy or claim through good faith negotiations for a period of not less than 30 days following notification of such controversy or claim to the other Party. If such controversy or claim cannot be resolved by means of such negotiations during such period, then such controversy or claim shall be resolved by binding arbitration administered by the American Arbitration Association (“AAA”) in accordance with the Commercial Arbitration Rules of the AAA in effect on the date of commencement of the arbitration, subject to the provisions of this Section 10.6. The arbitration shall be conducted as follows:

10.6.1 The arbitration shall be conducted by three arbitrators, each of whom by training, education, or experience has knowledge of the research, development, and commercialization of

CONFIDENTIAL TREATMENT REQUESTED

biological therapeutic products in the United States. The arbitration shall be conducted in English and held in New York, New York.

10.6.2 In its demand for arbitration, the Party initiating the arbitration shall provide a statement setting forth the nature of the dispute, the names and addresses of all other parties, an estimate of the amount involved (if any), the remedy sought, otherwise specifying the issue to be resolved, and appointing one neutral arbitrator. In an answering statement to be filed by the responding Party within **** after confirmation of the notice of filing of the demand is sent by the AAA, the responding Party shall appoint one neutral arbitrator. Within **** from the date on which the responding Party appoints its neutral arbitrator, the first two arbitrators shall appoint a chairperson.

10.6.3 If a Party fails to make the appointment of an arbitrator as provided in Section 10.6.2, the AAA shall make the appointment. If the appointed arbitrators fail to appoint a chairperson within the time specified in Section 10.6.2 and there is no agreed extension of time, the AAA shall appoint the chairperson.

10.6.4 The arbitrators will render their award in writing and, unless all Parties agree otherwise, will include an explanation in reasonable detail of the reasons for their award. Judgment upon the award rendered by the arbitrators may be entered in any court having jurisdiction thereof, including in the courts described in Section 10.5. The arbitrators will have the authority to grant injunctive relief and other specific performance; provided that the arbitrators will have no authority to award damages in contravention of this Agreement, and each Party irrevocably waives any claim to such damages in contravention of this Agreement. The arbitrators will, in rendering their decision, apply the substantive law of the State of New York, without giving effect to conflict of law provisions that may require the application of the laws of another jurisdiction. The decision and award rendered by the arbitrators will be final and non-appealable (except for an alleged act of corruption or fraud on the part of the arbitrator).

10.6.5 The Parties shall use their reasonable efforts to conduct all dispute resolution procedures under this Agreement as expeditiously, efficiently, and cost-effectively as possible.

10.6.6 All expenses and fees of the arbitrators and expenses for hearing facilities and other expenses of the arbitration will be borne equally by the Parties unless the Parties agree otherwise or unless the arbitrators in the award assess such expenses against one of the Parties or allocate such expenses other than equally between the Parties. Each of the Parties will bear its own counsel fees and the expenses of its witnesses except to the extent otherwise provided in this Agreement or by applicable law.

10.6.7 Compliance with this Section 10.6 is a condition precedent to seeking relief in any court or tribunal in respect of a dispute, but nothing in this Section 10.6 will prevent a Party from seeking equitable or other interlocutory relief in the courts of appropriate jurisdiction, pending the arbitrators’ determination of the merits of the controversy, if applicable to protect the confidential information, property, or other rights of that Party or to otherwise prevent irreparable harm that may be caused by the other Party’s actual or threatened breach of this Agreement.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

10.7 No Discrimination. Licensee and its Affiliates, and Licensee shall use reasonable efforts to require that any Sublicensees, in their respective activities under this Agreement, shall not discriminate against any employee or applicant for employment because of race, color, sex, sexual, or affectional preference, age, religion, national, or ethnic origin, handicap, or because he or she is a disabled veteran or a veteran (including a veteran of the Vietnam Era).

10.8 Compliance with Law. Licensee (and its Affiliates’ and any Sublicensees’) must comply with all prevailing laws, rules, and regulations that apply to its activities or obligations under this Agreement. Without limiting the foregoing, it is understood that this Agreement may be subject to United States laws and regulations controlling the export of technical data, computer software, laboratory prototypes, and other commodities, articles, and information, including the Arms Export Control Act as amended in the Export Administration Act of 1979 and that Licensee’s obligations are contingent upon compliance with applicable United States export laws and regulations. The transfer of certain technical data and commodities may require a license from the cognizant agency of the United States Government and/or written assurances by Licensee that Licensee shall not export data or commodities to certain foreign countries without prior approval of such agency. Licensor neither represents that a license is not required nor that, if required, it will issue.

10.9 Entire Agreement. This Agreement embodies the entire understanding between the Parties relating to the subject matter hereof and supersedes all prior understandings and agreements, whether written or oral, including that certain Mutual Non-Disclosure Agreement dated January 9, 2014 between the Parties. All “Confidential Information” (as defined in such Mutual Non-Disclosure Agreement) disclosed by one Party to the other Party pursuant to such Mutual Non-Disclosure Agreement shall be deemed “Confidential Information” of such disclosing Party under this Agreement (unless and until it falls within one of the exclusions set forth in Section 1.11). This Agreement may not be varied except by a written document signed by duly authorized representatives of both Parties.

10.10 Marking. Licensee, its Affiliates, and any Sublicensees shall mark any Licensed Product (or their containers or labels) made, sold, or otherwise distributed by it or them with any notice of patent rights necessary or desirable under applicable law to enable the Licensed Commercial Patents to be enforced to their full extent in any country where Licensed Products are made, used, sold, offered for sale, or imported.

10.11 Severability and Reformation. If any provision of this Agreement is held to be invalid or unenforceable by the arbitrators or a court of competent jurisdiction, then such invalid or unenforceable provision will be automatically revised to be a valid or enforceable provision that comes as close as permitted by law to the Parties’ original intent; provided that, if the Parties cannot agree upon such valid or enforceable provision, the remaining provisions of this Agreement will remain in full force and effect, unless the invalid or unenforceable provisions are of such essential importance to this Agreement that it is to be reasonably assumed that the Parties would not have entered into this Agreement without the invalid or unenforceable provisions.

10.12 Further Assurances. Each Party hereto agrees to execute, acknowledge, and deliver such further instruments, and to do all other reasonable acts, as may be necessary or appropriate in order to carry out the purposes and intent of this Agreement.

CONFIDENTIAL TREATMENT REQUESTED

10.13 Interpretation; Construction. The captions to the several Articles and Sections of this Agreement are included only for convenience of reference and shall not in any way affect the construction of, or be taken into consideration in interpreting, this Agreement. In this Agreement, unless the context requires otherwise, (a) the word “including” shall be deemed to be followed by the phrase “without limitation” or like expression; (b) references to the singular shall include the plural and vice versa; (c) references to masculine, feminine, and neuter pronouns and expressions shall be interchangeable; (d) the words “herein” or “hereunder” relate to this Agreement; (e) “or” is disjunctive but not necessarily exclusive; (f) the word “will” shall be construed to have the same meaning and effect as the word “shall”; (g) all references to “dollars” or “$” herein shall mean U.S. Dollars; (h) unless otherwise provided, all reference to Sections, Articles, and exhibits in this Agreement are to Sections, Articles, and exhibits of and in this Agreement; and (i) whenever this Agreement refers to a number of days, such number shall refer to calendar days unless business days are specified. Business days shall mean a day on which banking institutions in Washington, D.C. are open for business. Each Party represents that it has been represented by legal counsel in connection with this Agreement and acknowledges that it has participated in the drafting hereof. In interpreting and applying the terms and provisions of this Agreement, the Parties agree that no presumption will apply against the Party which drafted such terms and provisions.

10.14 Cumulative Rights and Remedies. The rights and remedies provided in this Agreement and all other rights and remedies available to either Party at law or in equity are, to the extent permitted by law, cumulative and not exclusive of any other right or remedy now or hereafter available at law or in equity. Neither asserting a right nor employing a remedy shall preclude the concurrent assertion of any other right or employment of any other remedy, nor shall the failure to assert any right or remedy constitute a waiver of that right or remedy.

10.15 Counterparts. This Agreement may be executed in one or more counterparts, each of which will be deemed an original, but all of which together will constitute one and the same instrument.

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IN WITNESS WHEREOF, the Parties, intending to be legally bound, have caused this License Agreement to be executed by their duly authorized representatives.


REGENX BIOSCIENCES, LLC		AAVLIFE

By: /s/ Kenneth Mills		By: /s/ Amber Salzman, PhD

Name: Kenneth Mills		Name: Amber Salzman, PhD

Title: President & CEO		Title: President

CONFIDENTIAL TREATMENT REQUESTED

Exhibit A-1

Licensed Research Patents (AAV7)


Application #	Patent #	Filing Date	Country	Status

	****	****	****	****

****		****	****	****

****		****	****	****

	****	****	****	****

****		****	****	****

	****	****	****	****

	****	****	****	****

	****	****	****	****

	****	****	****	****

****	****	****	****	****

	****	****	****	****

****		****	****	****

	****	****	****	****

****		****	****	****

	****	****	****	****

	****	****	****	****

	****	****	****	****

	****	****	****	****

	****	****	****	****

****		****	****	****

****		****	****	****

	****	****	****	****

	****	****	****	****

****		****	****	****

	****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Exhibit A-2

Licensed Research Patents (AAV8)


Application #	Patent #	Filing Date*	Country	Status

****		****	****	****

****		****	****	****

****		****	****	****

****	****	****	****	****

****		****	****	****

****		****	****	****

****	****	****	****	****

****	****	****	****	****

****	****	****	****	****

****		****	****	****

****		****	****	****

****		****	****	****

****		****	****	****

****	****	****	****	****

****		****	****	****

*	International Filing Date, where national stage application or foreign divisional thereof

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Exhibit A-3

Licensed Research Patents (AAV9)


Application #	Patent #	Filing Date	Country	Status

****	****	****	****	****

****	****	****	****	****

****	****	****	****	****

****	****	****	****	****

****	****	****	****	****

****	****	****	****	****

****	****	****	****	****

****	****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Exhibit A-4

Licensed Research Patents (AAVrh10)


Appin #	Title	Inventors	Nos	Docket
****	****	****	****	****

Docket

Country

Appln No

Filing Date

Patent
Number

Issue Date

Pubn
Number

Pub
Date

****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

Exhibit B

Specified Vectors


Specified Vector		Disease Indication

AAVrh10		Friedreich’s Ataxia (Systemic)

CONFIDENTIAL TREATMENT REQUESTED

Exhibit C

Press Release

CONFIDENTIAL TREATMENT REQUESTED

LOGO

REGENX BIOSCIENCES ENTERS INTO LICENSE AGREEMENT WITH AAVLIFE

FOR DEVELOPMENT OF TREATMENTS FOR FRIEDREICH’S ATAXIA USING NAV^® VECTORS

WASHINGTON, DC and Paris, France April 2014 — REGENX Biosciences, LLC announces that the company has entered into an agreement with AAVLife for the development and commercialization of products to treat Friedreich’s ataxia (FA) using NAV technology.

Under the terms of the Agreement, REGENX granted AAVLife an exclusive worldwide license, with rights to sublicense, to deliver REGENX’s NAV rAAVrh10 vector via non CNS routes to treat FA in humans. In addition, AAVLife was granted an option to obtain a non-exclusive worldwide license to additional NAV vectors for CNS delivery for the treatment of FA in humans. In return for these rights, REGENX receives payments in the form of up-front and on-going fees, certain milestone fees and royalties on net sales of products incorporating NAV vectors. REGENX would also receive a share of any sublicensing revenues.

“REGENX has been engaged with the team at AAVLife, including its stakeholders like the Friedreich’s Ataxia Research Alliance (FARA), since first becoming aware of their gene therapy research results and during the company’s process of formation. We are pleased to formally continue our collaboration with a team who has the leadership, expertise, resources, and commitment to patients that is required in order to develop innovative treatments for patients with FA through the application of NAV technology,” said Ken Mills, President and CEO of REGENX. “We believe this license agreement will be a key component to the successful development of treatments for patients suffering with FA.”

Amber Salzman, Ph.D., Chief Executive Officer and a co-founder of AAVLife, commented: “The right to the REGENX vector is a critical part of our program to advance into clinical trials a gene-therapy approach to treating Friedreich’s ataxia.”

Jennifer Farmer, Executive Director of FARA, added: “Heart disease accounts for most early deaths due to Friedreich’s ataxia. We believe that NAV technology will enable successful clinical studies that are urgently needed for patients with Friedreich’s ataxia.”

About Friedreich’s Ataxia (FA)

Friedreich’s ataxia is a rare, degenerative, life-shortening neuro-muscular disorder that affects children and adults, and involves the loss of strength and coordination usually leading to wheelchair use. Other symptoms may include diminished vision, hearing and speech; scoliosis (curvature of the spine); and increased risk of diabetes. Also associated with the disorder is a progressive decline in cardiac function which is the most common cause of death. There are no FDA-approved treatments.

CONFIDENTIAL TREATMENT REQUESTED

About REGENX Biosciences

REGENX Biosciences (www.regenxbio.com) is the leading AAV gene therapy company that is developing a new class of personalized therapies, based on its proprietary NAV vector technology platform, for a range of severe diseases with serious unmet needs. NAV vector technology includes novel AAV vectors such as rAAV7, rAAV8, rAAV9, and rAAVrh10. Our treatments in development include programs for hypercholesterolemia, mucopolysaccharidoses, and retinitis pigmentosa. REGENX’s leadership in AAV gene therapy and corresponding intellectual property has enabled it to establish collaborations with leading global partners including Chatham Therapeutics, Fondazione Telethon, Audentes Therapeutics, Lysogene, Esteve, and AveXis. In addition, together with Fidelity Biosciences, REGENX has formed Dimension Therapeutics, a company focused on the development and commercialization of AAV gene therapies for rare diseases.

For more information regarding REGENX, please visit www.regenxbio.com.

About AAVLife

AAVLife, registered in Paris, is a privately held company dedicated to advancing gene therapy for rare diseases. Further information is available at www.aavlife.com.

###

Contact:

REGENX Biosciences

Vit Vasista, 202-785-7438

vvasista @regenxbio.com

EX-10.15

Exhibit 10.15

CONFIDENTIAL TREATMENT REQUESTED

EXECUTION VERSION

LICENSE AGREEMENT

This LICENSE AGREEMENT (“Agreement”) is entered into as of July 9th, 2013 (“Effective Date”) by and between ReGenX Biosciences, LLC (formerly known as ReGenX, LLC), a limited liability company organized under the laws of the State of Delaware, with offices at 750 17th Street, NW, Suite 1100, Washington, DC 20006 (“Licensor”), and Audentes Therapeutics, Inc., a corporation organized under the laws of the State of Delaware, with offices at ****, San Francisco, California, 94115 (“Licensee”). Licensor and Licensee are hereinafter referred to individually as a “Party” and collectively as the “Parties.”

WHEREAS, Licensor has rights under certain Licensed Patents (as defined herein) pertaining to adeno-associated virus serotype 8 and 9; and

WHEREAS, Licensee desires to obtain an exclusive license under the Licensed Patents under the terms set forth herein;

NOW, THEREFORE, in consideration of the promises and covenants contained in this Agreement, and intending to be legally bound, the Parties hereby agree as follows:

ARTICLE 1: DEFINITIONS

1.1 “AAV8” means (a) the recombinant adeno-associated virus serotype 8 vector with the specified sequence set forth in GenBank **** and (b) any recombinant adeno-associated virus derivatives of such serotype 8 vector that are covered by the claims of the Licensed AAV8 Patents.

1.2 “AAV9” means (a) recombinant adeno-associated virus serotype 9 vector with the specified sequence set forth in GenBank **** and (b) any recombinant adeno-associated virus derivatives of such serotype 9 vector that are covered by the claims of the Licensed AAV9 Patents.

1.3 “Affiliate” means any legal entity directly or indirectly controlling, controlled by, or under common control with another entity. For purposes of this Agreement, “control” means the direct or indirect ownership of more than 50% of the outstanding voting securities of a legal entity, or the right to receive more than 50% of the profits or earnings of a legal entity, or the right to control the policy decisions of a legal entity.

1.4 “Calendar Quarter” means each three-month period or any portion thereof, beginning on January 1, April 1, July 1, and October 1.

1.5 “Confidential Information” means and includes all technical information, inventions, developments, discoveries, software, know-how, methods, techniques, formulae, animate and inanimate materials, data, processes, finances, business operations or affairs, and other proprietary ideas, whether or not patentable or copyrightable, of either Party that are (a) marked or otherwise identified as confidential or proprietary at the time of disclosure in writing; or (b) if disclosed orally, visually, or in another non-written form, identified as confidential at the time of disclosure and summarized in reasonable detail in writing as to its general content within 30 days after original disclosure. The Parties acknowledge that (i) the terms and conditions of this

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

EXECUTION VERSION

Agreement will be deemed the Confidential Information of both Parties and (ii) the records and reports referred to Section 3.6 of this Agreement will be deemed the Confidential Information of Licensee, regardless of whether such information is marked or identified as confidential. In addition, information provided to Licensee pursuant to the provisions of Section 7.1 will be deemed the Confidential Information of Licensor, regardless of whether such information is marked or identified as confidential. Notwithstanding the foregoing, Confidential Information will not include the following, in each case, to the extent evidenced by competent written proof of the Receiving Party:

1.5.1 information that was already known to the Receiving Party, other than under an obligation of confidentiality, at the time of disclosure by the Disclosing Party;

1.5.2 information that was generally available to the public or otherwise part of the public domain at the time of its disclosure to the Receiving Party;

1.5.3 information that became generally available to the public or otherwise part of the public domain after its disclosure, other than through any act or omission of the Receiving Party in breach of this Agreement;

1.5.4 information that is independently discovered or developed by the Receiving Party without the use of Confidential Information of the Disclosing Party; or

1.5.5 information that was disclosed to the Receiving Party, other than under an obligation of confidentiality, by a Third Party who had no obligation to the Disclosing Party not to disclose such information to others.

1.6 “Disclosing Party” has the meaning set forth in Section 5.1.

1.7 “Domain Antibody” ****.

1.8 “FDA” means the United States Food and Drug Administration, or a successor agency in the United States with responsibilities comparable to those of the United States Food and Drug Administration.

1.9 “Field” means, collectively, the XLMTM Field and the Pompe Field.

1.10 “GSK Agreement” means that certain License Agreement entered into between Licensor and SmithKline Beecham Corporation, effective on March 6, 2009, as amended by that certain Amendment to License Agreement dated April 15, 2009, and as amended from time to time.

1.11 “Licensed AAV8 Patents” means (a) all United States patents and patent applications listed in part 1 of Exhibit A and (b) any re-examination certificates thereof, and their foreign counterparts and extensions, continuations, divisionals, and re-issue applications.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

1.12 “Licensed AAV9 Patents” means (a) all United States patents and patent applications listed in part 2 of Exhibit A and (b) any re-examination certificates thereof, and their foreign counterparts and extensions, continuations, divisionals, and re-issue applications.

1.13 “Licensed Patents” means, collectively, (a) the Licensed AAV8 Patents and the Licensed AAV9 Patents and (b) any additional claims of patents and patent applications as required pursuant to Section 8.1.5.

1.14 “Licensed Product” means (a) any AAV8 or AAV9 product that is made, made for, used, sold, offered for sale, or imported by Licensee, its Affiliates and any of its or their Sublicensees, the manufacture, use, sale, offer for sale, or import of which product, in the absence of the license granted pursuant to this Agreement, would infringe or is covered by at least one Valid Claim in the country of manufacture, use, sale, offer for sale, or import, including products manufactured by a process that would infringe at least one Valid Claim in the country of manufacture, use, sale, offer for sale, or import; or (b) any service with respect to the administration of AAV8 or AAV9 to patients that, in the absence of the licenses granted pursuant to this Agreement, would infringe at least one Valid Claim of the Licensed Patents in the country of sale.

1.15 “NDA” means a New Drug Application filed with the FDA as described in 21 C.F.R. § 314, a Biological License Application (BLA) pursuant to 21 C.F.R. § 601.2, or any equivalent or any corresponding application for regulatory approval in any country or regulatory jurisdiction other than the United States.

1.16 “Net Sales” means the gross receipts from sales or other disposition of a Licensed Product (including fees for services within the definition of “Licensed Product”) by Licensee and/or its Affiliates and/or any Sublicensees to Third Parties less the following deductions that are directly attributable to a sale, specifically and separately identified on an invoice or other documentation and actually borne by Licensee, its Affiliates, or any Sublicensees: ****. In the event consideration other than cash is paid to Licensee, its Affiliates, or any Sublicensees, for purposes of determining Net Sales, the Parties shall use the cash consideration that Licensee, its Affiliates, or any Sublicensees would realize from an unrelated buyer in an arm’s length sale of an identical item sold in the same quantity and at the time and place of the transaction, as determined jointly by Licensor and Licensee based on transactions of a similar type and standard industry practice, if any.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

1.17 “Penn Agreement” means that certain License Agreement entered into between Licensor and The Trustees of the University of Pennsylvania, effective on February 24, 2009, as amended by that letter agreement dated March 6, 2009, and as amended from time to time.

1.18 “Phase 3 Clinical Trial” means a pivotal clinical trial in humans performed to gain evidence with statistical significance of the efficacy of a product in a target population, and to obtain expanded evidence of safety for such product that is needed to evaluate the overall benefit-risk relationship of such product, to form the basis for approval of an NDA and to provide an adequate basis for physician labeling, as described in 21 C.F.R. § 312.21(c) or the corresponding regulation in jurisdictions other than the United States.

1.19 “Pompe Field” means the treatment of Pompe Disease (GAA deficiency) in humans by in vivo gene therapy in humans using AAV8 or AAV9.

1.20 “Prosecute” means preparation, filing, and prosecuting patent applications and maintaining patents.

1.21 “Receiving Party” has the meaning set forth in Section 5.1.

1.22 “Retained Rights” has the meaning set forth in Section 2.2.

1.23 “Sublicensee” means any Third Party or Affiliate to whom Licensee grants a sublicense of some or all of the rights granted to Licensee under this Agreement as permitted by this Agreement.

1.24 “Third Party” means any person or entity other than a Party to this Agreement or Affiliates of a Party to this Agreement.

1.25 “Valid Claim” means a claim of an issued and unexpired patent (including any patent claim the term of which is extended by any extension, supplementary protection certificate, patent term restoration, or the like) or a claim of a pending patent application included within the Licensed Patents, which has not lapsed, been abandoned, been held revoked, or been deemed unenforceable or invalid by a non-appealable decision or an appealable decision from which no appeal was taken within the time allowed for such appeal of a court or other governmental agency of competent jurisdiction.

1.26 “XLMTM Field” means the treatment of X-linked myotubular myopathy (XLMTM) in humans by in vivo gene therapy in humans using AAV8 or AAV9.

ARTICLE 2: LICENSE GRANT

2.1 License Grant. Subject to the terms and conditions of this Agreement, Licensor hereby grants to Licensee an exclusive, sublicensable (as provided in Section 2.4 only), non-transferable (except as provided in Section 10.2), royalty-bearing, worldwide license, under the Licensed Patents to make, have made, use, import, sell, and offer for sale Licensed Products solely in the Field, including, for the avoidance of doubt, the right to conduct research and development (including by conducting clinical trials in humans and/or animal studies).

CONFIDENTIAL TREATMENT REQUESTED

2.2 Retained Rights. Except for the rights and licenses specified in Section 2.1 or as provided in Section 8.1.5, no license or other rights are granted to Licensee under any intellectual property of Licensor, whether by implication, estoppel, or otherwise, whether any such intellectual property dominates or is dominated by the Licensed Patents. Notwithstanding anything to the contrary this Agreement, Licensor may use and permit others to use the Licensed Patents for any research, development, commercial, or other purposes outside of the Field. Without limiting the foregoing, Licensee acknowledges and agrees to the following rights retained by Licensor and its direct and indirect licensors (individually and collectively, the “Retained Rights”), whether inside or outside the Field:

2.2.1 Notwithstanding anything in this Agreement to the contrary, the rights and licenses granted in Section 2.1 shall not include any right (and Licensor and its direct and indirect licensors retain the exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents to make, have made, use, sell, offer to sell, and import Domain Antibodies that are expressed by an adeno-associated vector, including AAV8 and/or AAV9.

2.2.2 Notwithstanding anything in this Agreement to the contrary, Licensor and its direct and indirect licensors retain the following rights with respect to the Licensed Patents:

(a)

(b)

A non-exclusive right for Licensor’s direct and indirect licensors (which right is sublicensable by such licensors) to use the Licensed Patents for non-commercial research purposes and to use the Licensed Patents for such licensors’ discovery research efforts with non-profit organizations and collaborators.

2.2.3 Notwithstanding anything in this Agreement to the contrary, the rights and licenses granted in Section 2.1 shall not include any right (and Licensor retains the exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents:

(a)

to conduct commercial reagent and services businesses, which includes the right to make, have made, use, sell, offer to sell, or import research reagents, including any viral vector construct (provided that, for clarity, such rights retained by Licensor shall not include the right to conduct clinical trials in humans in the Field); and

(b)

to use the Licensed Patents to provide services to any Third Parties; provided that Licensee’s license under Section 2.1 does include the right to provide the service of the administration of Licensed Products to patients.

2.2.4 Notwithstanding anything in this Agreement to the contrary, Licensor retains the fully sublicensable right under the Licensed Patents to grant non-exclusive research and development licenses to Affiliates and Third Parties; provided that such development rights

CONFIDENTIAL TREATMENT REQUESTED

granted by Licensor shall not include the right to conduct clinical trials in humans in the Field or any rights to sell products in the Field.

2.2.5 Notwithstanding anything to the contrary in this Agreement, the University of Pennsylvania may use and permit other non-profit organizations or other non-commercial entities to use the Licensed Patents for educational, research, and other non-commercial purposes.

2.3 Government Rights. Licensee acknowledges that the United States government retains certain rights in intellectual property funded in whole or part under any contract, grant, or similar agreement with a federal agency. The license grant hereunder is expressly subject to all applicable United States government rights, including any applicable requirement that products resulting from such intellectual property sold in the United States must be substantially manufactured in the United States.

2.4 Sublicensing.

2.4.1 The license granted pursuant to Section 2.1 is sublicensable by Licensee to any Affiliates or Third Parties; provided that any such sublicense must comply with the provisions of this Section 2.4 (including Section 2.4.2).

2.4.2 The right to sublicense granted to Licensee under this Agreement is subject to the following conditions:

(a)

Licensee may only grant sublicenses **** pursuant to a written sublicense agreement with the Sublicensee. Licensor must receive written notice as soon as practicable following execution of any such sublicenses.

(b)

(c)

The official language of any sublicense agreement shall be English.

(d)

Within **** after entering into a sublicense, Licensor must receive a copy of the sublicense written in the English language for Licensor’s records and to share with Licensor’s direct and indirect licensors. The copy of the sublicense may be redacted to exclude confidential information of the applicable Sublicensee, but such copy shall not be redacted to the extent that it impairs Licensor’s (or any of its direct or indirect licensors’) ability to ensure compliance with this Agreement; provided that, if any of Licensor’s direct or indirect licensors require a complete, unredacted copy of the sublicense, Licensee shall provide such complete, unredacted copy.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

(e)

2.5 Improvements.

2.5.1 Licensee hereby grants to Licensor a non-exclusive, worldwide, royalty-free, transferable, sublicensable, irrevocable, perpetual license (a) to use any Licensed Back Improvements (and any intellectual property rights with respect thereto) consummate in scope to the Retained Rights and (b) to practice the Licensed Back Improvements (and any intellectual property rights with respect thereto) in connection with AAV8 and AAV9 outside the Field, including the right to research, develop, make, have made, use, offer for sale, and sell products and services outside the Field. For purposes of this Agreement, “Licensed Back Improvements” means any **** by Licensee, any Affiliates ****, or any Sublicensees to any vector that is the subject of a claim within the Licensed Patents.

2.5.2 Licensee agrees to provide prompt notice to Licensor upon the filing of any patent application covering any Licensed Back Improvement, together with a reasonably detailed description of or access to such Licensed Back Improvement to permit the practice of any such invention or improvement by Licensor or its direct or indirect licensors or licensees.

ARTICLE 3: CONSIDERATION

3.1 Initial Fee. In consideration of the license granted to Licensee under Section 2.1, Licensee shall pay Licensor an initial fee of $600,000 upon the Effective Date. One-half of the amount paid by Licensee to Licensor under this Section 3.1 may be paid by Licensee in the form of shares of Licensee’s common stock, which will be issued in accordance with Section 3.8.

3.2 Annual Maintenance Fee. In consideration of the license granted to Licensee under Section 2.1, Licensee shall pay Licensor on-going annual maintenance fees of **** on each anniversary of the Effective Date.

3.3 Milestone Fees. In consideration of the license granted to Licensee under Section 2.1, Licensee shall pay Licensor the following milestone payments on a per-Licensed Product basis:


XLMTM Field Milestone		Milestone Payment
1. First treatment of human subject in a clinical trial (i.e., first patient, first dose)		****
2. First treatment in Phase 3 Clinical Trial (i.e., first patient, first dose)		****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


XLMTM Field Milestone	Milestone Payment
3. NDA submission in the United States		****
4. NDA submission in the European Union		****
5. NDA approval in the United States		****
6. NDA approval in the European Union		****

Total:		8.85 million

Pompe Field Milestone	Milestone Payment
1. First treatment of human subject in a clinical trial (i.e., first patient, first dose)		****
2. First treatment in Phase 3 Clinical Trial (i.e., first patient, first dose)		****
3. NDA submission in the United States		****
4. NDA submission in the European Union		****
5. NDA approval in the United States		****
6. NDA approval in the European Union		****

Total:	$	8.85 million

3.3.1 At Licensee’s option, up to **** of the amount paid by Licensee to Licensor under the first milestones (i.e., first treatment of human study) for each of the XLMTM Field and the Pompe Field may be paid by Licensee in the form of shares of Licensee’s common stock, which will be issued in accordance with Section 3.8.

3.3.2 For clarity, the milestone payments set forth in this Section 3.3 are payable **** in the XLMTM Field and once in the Pompe Field with respect to each Licensed Product that achieves the milestone event, regardless of whether the milestone is achieved by Licensee or any Sublicensee. To the extent that either of the two development milestones in this Section 3.3 with respect to a particular field (i.e., first treatment of human subject in a clinical trial or first treatment in Phase 3 Clinical Trial) has not been paid at the time of achievement of either NDA submission milestone for that field, then, upon the achievement of either of such NDA submission milestones, the preceding unpaid development milestone payments with respect to that field shall be made in addition to the payment corresponding to the NDA submission milestone that has been achieved.

3.4 Royalties. In further consideration of the license granted to Licensee under Section 2.1, Licensee shall pay to Licensor the following royalties based upon Net Sales of Licensed Products in the XLMTM Field or the Pompe Field, as applicable, subject to the reductions in royalty rates set forth in Section 3.4.1:

Cumulative Annual Net Sales of all Licensed Products

in the XLMTM Field Worldwide

Royalty Percentage for
XLMTM Field

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


Portion of Net Sales less than $300 million		*	***
Portion of Net Sales between (and including) $300 million through (and including) $600 million		*	***
Portion of Net Sales greater than $600 million		*	***

Cumulative Annual Net Sales of all Licensed Products in the Pompe Field Worldwide	Royalty Percentage for Pompe Field
Portion of Net Sales less than $300 million		*	***
Portion of Net Sales between (and including) $300 million through (and including) $600 Million		*	***
Portion of Net Sales greater than $600 million		*	***

3.4.1 Third Party Royalties Stacking Provision. If Licensee must obtain a license from a Third Party to avoid infringement of such Third Party’s rights in order to manufacture, use, or commercialize a given Licensed Product and if the royalties required to be paid to such Third Party for such license, together with those royalties payable to Licensor, in the aggregate, exceed **** of Net Sales for any Licensed Product, then the royalty owed to Licensor for that Licensed Product will be reduced by an amount calculated as follows:

STACKING ROYALTY CALCULATIONS

R = (C * (A / (A+B)))

Where

R = reduction of Licensor royalty,

A = unreduced Licensor royalty,

B = sum of all Third Party royalties,

C = increment of projected total royalty above ****.

Example Calculation:

assume: i) all Third Party royalties = ****

ii) unreduced Licensor royalty = ****

iii) projected total royalty = ****

R = (**** - ****)* (**** / (**** + ****))

R = (**** * ****)

R = ****

Licensor Stacked Royalty = **** — **** = ****%

Notwithstanding the foregoing, Licensee will pay to Licensor no less than ****% of the royalties that Licensee would otherwise pay to Licensor if there were no royalties due to Third Parties.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

3.4.2 Royalty Payment Period. Licensee’s obligation hereunder for payment of a royalty under this Section 3.4 on the Net Sales of Licensed Products in a given country will end on a country-by-country basis when all Valid Claims in that country claiming the Licensed Product have expired, lapsed, been abandoned, or been invalidated.

3.5 Sublicense Fees.

3.5.1 In further consideration of the license granted to Licensee under Section 2.1, Licensee will pay Licensor a percentage of any sublicense fees (including upfront payments and milestone payments) received by Licensee for the Licensed Patents from any Sublicensee or from any person or entity granted any option to obtain a sublicense. The applicable percentage due to Licensor for each sublicense (or option) shall be as follows:


Event	Sublicense Fee Rate
If sublicensed (or optioned) on or before the first anniversary of the Effective Date		****
If sublicensed (or optioned) on or before the third anniversary of the Effective Date but after the first anniversary of the Effective Date		****
If sublicensed (or optioned) on or before the fourth anniversary of the Effective Date but after the third anniversary of the Effective Date		****
If sublicensed (or optioned) after the fourth anniversary of the Effective Date		****

3.5.2 With respect to the obligations under this Section 3.5, Licensee shall not be required to submit any amounts received from a Third Party for the following:

(a)

Reimbursement for research, development, and/or manufacturing activities performed by Licensee corresponding directly to the development of Licensed Products pursuant to a specific agreement;

(b)

Consideration received for the purchase of an equity interest in Licensee at fair market value or in the form of loans at commercially reasonable rates of interest; and

(c)

Any and all amounts paid to Licensee by a Sublicensee as royalties on sales of Licensed Product sold by the Sublicensee under a sublicense agreement.

3.5.3 To the extent Licensee receives payment from a Third Party relating to one or more of the milestone events set forth in the table in Section 3.3, then the amount of the payment made to Licensor under such Section 3.3 with respect to such milestone event shall be not be deemed sublicense fees under this Section 3.5; instead, the amounts due under this Section 3.5 shall be calculated by applying the applicable sublicense fee rate set forth in Section 3.5.1 above

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

to the sublicense fees received by Licensee from such Third Party after deducting the amount of the payment under Section 3.3.

3.6 Reports and Records.

3.6.1 Licensee must deliver to Licensor within **** after the end of each Calendar Quarter after the first commercial sale of a Licensed Product a report setting forth the calculation of the royalties due to Licensor for such Calendar Quarter, including:

3.6.1.1

Number of Licensed Products included within Net Sales, listed by country;

3.6.1.2

Gross consideration for Net Sales of Licensed Product, including all amounts invoiced, billed, or received;

3.6.1.3

Qualifying costs to be excluded from the gross consideration, as described in Section 1.16, listed by category of cost;

3.6.1.4

Net Sales of Licensed Products listed by country;

3.6.1.5

A detailed accounting of any royalty reductions applied pursuant to Section 3.4.1;

3.6.1.6

Royalties owed to Licensor, listed by category; and

3.6.1.7

The computations for any applicable currency conversions.

3.6.2 Licensee shall pay the royalties due under Section 3.4 within **** following the last day of the Calendar Quarter in which the royalties accrue. Licensee shall send the royalty payments along with the report described in Section 3.6.1.

3.6.3 Within **** after the occurrence of a milestone event described in Section 3.3, Licensee must deliver to Licensor a report describing the milestone event that occurred, together with a payment of the applicable amount due to Licensor pursuant to Section 3.3. In addition, within **** after the receipt of sublicense fees from any Sublicensee as described in Section 3.5, Licensee must deliver to Licensor a report describing the fees received, together with a payment of the applicable amount due to Licensor pursuant to Section 3.5.

3.6.4 All financial reports under this Section 3.6 will be certified by the chief financial officer of Licensee.

3.6.5 Licensee shall maintain and require its Affiliates and all Sublicensees to maintain, complete and accurate books and records which enable the royalties, fees, and payments payable under this Agreement to be verified. The records must be maintained for **** after the submission of each report under Article 3. Upon reasonable prior written notice to Licensee, Licensee and its Affiliates and all Sublicensees will provide Licensor and/or its direct or indirect licensors (and their respective accountants) with access to all of the relevant books, records, and related background information as reasonably required to confirm the accuracy of the royalties,

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CONFIDENTIAL TREATMENT REQUESTED

fees, and payments paid to Licensor under this Agreement. Access will be made available: (a) during normal business hours; (b) in a manner reasonably designed to facilitate the auditing party’s review or audit without unreasonable disruption to Licensee’s business; and (c) no more than once each calendar year during the term of this Agreement and for a period of **** thereafter. Licensee will promptly pay to Licensor the amount of any underpayment determined by the review or audit, plus accrued interest. If the review or audit determines that Licensee has underpaid any payment by **** or more, then Licensee will also promptly pay the costs and expenses of Licensor and or its direct or indirect licensors and accountants in connection with the review or audit.

3.7 Currency, Interest.

3.7.1 All dollar amounts referred to in this Agreement are expressed in United States dollars. All payments to Licensor under this Agreement must be made in United States dollars.

3.7.2 If Licensee receives payment in a currency other than United States dollars for which a royalty or fee or other payment is owed under this Agreement, then (a) the payment will be converted into United States dollars at the conversion rate for the foreign currency as published in the eastern edition of the Wall Street Journal, as of the last business day of the Calendar Quarter in which the payment was received by Licensee; and (b) the conversion computation will be documented by Licensee in the applicable report delivered to Licensor under Section 3.6.

3.7.3 All amounts that are not paid by Licensee when due will accrue interest from the date due until paid at a rate equal to 1.5% per month (or the maximum allowed by law, if less).

3.8 Issuance of Common Stock. If Licensee elects to pay any amounts under Section 3.1 or 3.3.1 by the issuance of shares of Licensee’s common stock, then the provisions of this Section 3.8 will apply.

3.8.1 Each share of Licensee’s common stock will be valued at **** per share as of the Effective Date (the “Price Per Share”) and shall be issued pursuant to the terms of the Common Stock Purchase Agreement in the form attached hereto as Exhibit C (the “Stock Purchase Agreement”). If Licensee at any time or from time to time after the Effective Date effects a subdivision, split, or combination of Licensee’s outstanding common stock into a greater or lesser number of shares, then, in each such event, the Price Per Share in effect immediately prior to such subdivision, split, or combination will be increased or decreased proportionately. Licensee will provide Licensor with written notice of any such subdivision, split, or combination and the resulting Price Per Share.

3.8.2 The number of shares to be issued to Licensor will be determined by taking the amount of the payment owed under Section 3.1 or 3.3.1, as applicable, and dividing it by the Price Per Share, as calculated pursuant to Section 3.8.1. Licensee will deliver to Licensor, by no later than the date the payment (with respect to which Licensee will fulfill by the issuance of shares of Licensee’s common stock) is due, (a) a copy of the Stock Purchase Agreement (executed by both Licensor and Licensee), (b) a stock certificate in the name of Licensee for the number of shares of common stock to be issued, and (c) in connection with any issuance

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CONFIDENTIAL TREATMENT REQUESTED

pursuant to Section 3.3.1, a certificate signed by an officer of Licensee attesting that Licensee’s representations and warranties contained in Sections 8.2.1 (with respect to Licensee’s ability to issue the common stock), 8.2.4 (with respect to Licensee’s ability to issue the common stock), and 8.2.5 are true and correct as of the date of issuance of such stock with the same effect as though made on and as of such date.

ARTICLE 4: DILIGENCE

4.1 Licensee will use commercially reasonable efforts to develop, commercialize, market, promote, and sell Licensed Products in each of the XLMTM Field and the Pompe Field. Commercially reasonable efforts means efforts equivalent to those utilized by ****. Without limiting the foregoing, Licensee will meet the following:

(a)

Acceptance by the FDA of an Investigational New Drug application for a Licensed Product in the XLMTM Field by no later than ****; and

(b)

Acceptance by the FDA of an Investigational New Drug application for a Licensed Product in the Pompe Field by no later than ****.

Licensee will notify Licensor in writing as soon as Licensee believes in good faith that Licensee will not be able to achieve either milestone set forth in Section 4.1(a) or (b) by the relevant deadline date, and, upon the payment to Licensor of **** within **** of the original deadline date, the deadline date for such milestone set forth in Section 4.1(a) or (b), as applicable, will be extended for **** from the original deadline date; provided that Licensee will only be entitled to **** for the XLMTM Field and **** for the Pompe Field, each of which extensions will require a payment of **** as provided in this Section 4.1.

4.2 Within **** after the Effective Date and within **** of each December 1 thereafter, Licensee shall provide Licensor with written progress reports, setting forth in such detail as Licensor may reasonably request, the progress of the development, evaluation, testing, and commercialization of each Licensed Product. Licensee will also notify Licensor within **** of the first commercial sale by Licensee, its Affiliates, or any Sublicensees of each Licensed Product. Such a report (“Development Progress Report”), setting forth the current stage of development of Licensed Products, shall include:

4.2.1 Date of Development Progress Report and time covered by such report;

4.2.2 Major activities and accomplishments completed by Licensee, its Affiliates, and any Sublicensees relating directly to the Licensed Product since the last Development Progress Report;

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4.2.5 Projected total development remaining before product launch of each Licensed Product; and

4.2.6 Summary of significant development efforts using the Licensed Patents being performed by Third Parties, including the nature of the relationship between Licensee and such Third Parties.

4.3 The Parties agree that Development Progress Reports shall be deemed Licensee’s Confidential Information; provided that Licensor may share a copy of such reports with its direct and indirect licensors.

4.4 Simultaneously with the Development Progress Report, Licensee shall deliver a detailed description of any Licensed Back Improvements, if not previously provided pursuant to Section 2.5.2.

ARTICLE 5: CONFIDENTIALITY

5.2 Public Announcements.

5.2.1 The Parties agree they will release a joint press release in the form attached hereto as Exhibit B. Except as provided in Section 5.2.1, any other press releases by either Party with respect to the other Party or any other public disclosures concerning the existence of or terms of this Agreement shall be subject to review and approval by the other Party. Once the joint press release or any other written statement is approved for disclosure by both Parties, either Party may make subsequent public disclosure of the contents of such statement without the further approval of the other Party.

CONFIDENTIAL TREATMENT REQUESTED

5.2.2 Notwithstanding Section 5.2.1, Licensor has the right to publish (through press releases, scientific journals, or otherwise) and refer to any clinical, regulatory, or research results related to Licensee’s Licensed Product or AAV8 or AAV9 program that have been publicly disclosed by Licensee, including referring to Licensee by name as a licensee of Licensor, which publication or referral by Licensor shall not require the prior consent of Licensee.

5.3 Authorized Disclosure. Notwithstanding the provisions of Section 5.1 or 5.2, either Party may disclose Confidential Information or make such a disclosure of the existence of and/or terms of this Agreement to any ****; provided that, in each case, such recipient of Confidential Information is obligated to keep such information confidential on terms no less stringent than those set forth in this Agreement. Furthermore, Licensee agrees that Licensor may share a copy of this Agreement, reports and notices provided by Licensee to Licensor pursuant to the terms of this Agreement, and copies of sublicense agreements provided to Licensor hereunder with any of Licensor’s direct and indirect licensors of the Licensed Patents. In the event that the Receiving Party receives service of legal process that purports to compel disclosure of the Disclosing Party’s Confidential Information or becomes obligated by law to disclose the Confidential Information of the Disclosing Party or the existence of or terms of this Agreement to any governmental authority, the Receiving Party shall promptly notify the Disclosing Party, so that the Disclosing Party may seek an appropriate protective order or other remedy with respect to narrowing the scope of such requirement and/or waive compliance by the Receiving Party with the provisions of this Agreement. The Receiving Party will provide the Disclosing Party with reasonable assistance in obtaining such protective order or other remedy. If, in the absence of such protective order or other remedy, the Receiving Party is nonetheless required by law to disclose the existence of or terms of this Agreement or other Confidential Information of the Disclosing Party, the Receiving Party may disclose such Confidential Information without liability hereunder; provided that the Receiving Party shall furnish only such portion of the Confidential Information that is legally required to be disclosed and only to the extent required by law.

5.4 Term of Confidentiality. The obligations of this Article 5 shall continue for a period of **** following the expiration or termination of this Agreement.

ARTICLE 6: TERM AND TERMINATION

6.1 Term of Agreement. This Agreement, unless sooner terminated as provided in this Agreement, expires upon the expiration, lapse, abandonment, or invalidation of the last Valid Claim to expire, lapse, or become abandoned or unenforceable in all countries of the world.

6.2 Licensee’s Right to Terminate. Licensee may, upon 90 days’ prior written notice to Licensor, terminate this Agreement for any reason, with or without cause. In exercising such termination right, Licensee may terminate the Agreement in its entirety or, if desired, Licensee may specify in the written notice that this Agreement is terminating only with respect to either the Pompe Field or the XLMTM Field.

6.3 Termination for Breach.

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6.3.1 Licensor may terminate this Agreement, if Licensee is late in paying to Licensor royalties, fees, or any other monies due under this Agreement, and Licensee does not pay Licensor in full within 15 days upon written demand from Licensor, which termination shall be effective immediately upon the expiration of such 15-day cure period.

6.3.2 Either Party may terminate this Agreement, if the other Party materially breaches

6.3.3 This Agreement and does not cure such material breach within 30 days after written notice of the breach, which termination shall be effective immediately upon the expiration of such 30-day cure period; provided that, if termination is by Licensor as a result of Licensee’s materially breaching Article 4, and if such breach only relates to either the Pompe Field or XLMTM Field, but not both, then Licensor’s termination right shall only be with respect to the Pompe Field or XLMTM Field, as applicable, with respect to which the breach related and not both. Notwithstanding the above, if Licensee disputes in good faith that such material breach exists, and gives Licensor written notice of such dispute within 30 days following Licensee’s receipt of Licensor’s notice of default, then, Licensor may not terminate this Agreement until the dispute is resolved in accordance with Section 10.6; provided that Licensor shall be entitled to terminate this Agreement at the end of the original 30-day cure period, without waiting for resolution of the dispute in accordance with Section 10.6, if the breach by Licensee of this Agreement would cause Licensor to be in breach of the GSK Agreement or the Penn Agreement.

6.4 Termination for Insolvency.

6.4.1 Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, if Licensee or any of its Affiliates experiences any Trigger Event.

6.4.2 Licensee shall include in each sublicense agreement entered into with a Sublicensee a right of Licensee to terminate such sublicense agreement if such Sublicensee experiences any Trigger Event; and Licensee shall terminate the sublicense agreement, effective immediately upon written notice to the Sublicensee, if the Sublicensee experiences any Trigger Event. In addition, if the Sublicensee’s experiencing of a Trigger Event gives Licensor’s licensor a right of termination under the Penn Agreement and such licensor provides written notice of such termination to Licensor, then, upon receipt of such notice, Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, if any Sublicensee experiences any Trigger Event.

6.4.3 For purposes of this Section 6.4, “Trigger Event” means any of the following: (a) if Licensee, any Affiliate, or any Sublicensee, as applicable, (i) becomes insolvent, becomes bankrupt, or generally fails to pay its debts as such debts become due, (ii) is adjudicated insolvent or bankrupt, (iii) admits in writing its inability to pay its debts, (iv) suffers the appointment of a custodian, receiver, or trustee for it or its property and, if appointed without its consent, such appointment is not discharged within 30 days, (v) makes an assignment for the benefit of creditors, or (vi) suffers proceedings being instituted against it under any law related to bankruptcy, insolvency, liquidation, or the reorganization, readjustment, or release of debtors and, if contested by it, not dismissed or stayed within ten days; (b) the institution or commencement by Licensee, any Affiliate, or any Sublicensee, as applicable, of any proceeding under any law related to bankruptcy, insolvency, liquidation, or the reorganization, readjustment, or release of debtors; (c) the entering of any order for relief relating to any of the proceedings

CONFIDENTIAL TREATMENT REQUESTED

described in Section 6.4.3(a) or (b) above; (d) the calling by Licensee, any Affiliate, or any Sublicensee, as applicable, of a meeting of its creditors with a view to arranging a composition or adjustment of its debts; or (e) the act or failure to act by Licensee, any Affiliate, or any Sublicensee, as applicable, indicating its consent to, approval of, or acquiescence in any of the proceedings described in Section 6.4.3(b) through (d) above.

6.5 Patent Challenge.

6.5.1 Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, upon the commencement by Licensee or any of its Affiliates of a Patent Challenge.

6.5.2 Licensee shall include in each sublicense agreement entered into with a Sublicensee a right of Licensee to terminate such sublicense agreement if such Sublicensee commences a Patent Challenge; and Licensee shall terminate the sublicense agreement, effective immediately upon written notice to the Sublicensee, if the Sublicensee commences a Patent Challenge. In addition, if the Sublicensee’s commencement of a Patent Challenge gives Licensor’s licensor a right of termination under the Penn Agreement and such licensor provides written notice of such termination to Licensor, then, upon receipt of such notice, Licensor may terminate this Agreement, effective immediately upon written notice to Licensee, if any Sublicensee commences a Patent Challenge.

6.5.3 For purposes of this Section 6.5, “Patent Challenge” means any action against Licensor, the University of Pennsylvania, or any direct or indirect licensor of Licensor (including an action for declaratory judgment) to declare, or render invalid or unenforceable the Licensed Patents, or any claim thereof.

6.6 Effects of Termination. The effect of termination by Licensee pursuant to Section 6.2, by either Party, as applicable, under Section 6.3, or by Licensor pursuant to Section 6.4 or 6.5 shall be as follows:

6.6.1 The licenses granted by Licensor hereunder shall terminate, and Licensee and its Affiliates shall cease to make, have made, use, import, sell, and offer for sale all Licensed Products and shall cease to otherwise practice the Licensed Patents; provided that Licensee shall have the right to continue to sell its existing inventories of Licensed Products for a period not to exceed **** after the effective date of such termination;

6.6.2 All sublicenses granted to Third Parties to the extent of the rights licensed to Licensee hereunder and sublicensed to the Sublicensee shall be assigned to Licensee; provided that (i) prior to such assignment, Licensee shall advise Licensor whether such Sublicensee is then in full compliance with all terms and conditions of its sublicense and continues to perform thereunder, and, if such Sublicensee is not in full compliance or is not continuing to perform, Licensor may elect not to have such sublicense assigned; and (ii) following such assignment, Licensor shall not be liable to such Sublicensee with respect to any obligations of Licensee to the Sublicensee that are not consistent with, or not required by, Licensor’s obligations to Licensee under this Agreement;

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CONFIDENTIAL TREATMENT REQUESTED

6.6.3 If termination is by Licensee pursuant to Section 6.2 or by Licensor pursuant to Section 6.3, 6.4, or 6.5, Licensee shall grant, and hereby grants to Licensor a non-exclusive, perpetual, irrevocable, worldwide, royalty-free, transferable, sublicensable license under any patentable modifications or improvements (and any intellectual property rights with respect thereto) developed by Licensee, any Affiliates (excluding any such modifications or improvements developed by a Third Party that acquired Licensee or its Affiliates, whether by merger, acquisition or assets sale, prior to the date of such acquisition), or any Sublicensees to any vector that is the subject of a claim within any of the Licensed Patents, for use by Licensor for the research, development, and commercialization of products in any therapeutic indication;

6.6.4 Licensee shall pay all monies then-owed to Licensor under this Agreement; and

6.6.5 Each Receiving Party shall, at the other Party’s request, return all Confidential Information of the Disclosing Party. Notwithstanding the foregoing, one copy may be kept by either Party for a record of that Party’s obligations.

If termination is only with respect to either the Pompe Field or the XLMTM Field, but not both, then the provisions of this Section 6.6 shall only apply with respect to the terminated Field, and this Agreement shall continue with respect to the non-terminated Field.

6.7 Survival. Licensee’s obligation to pay all monies due and owed to Licensor under this Agreement which have matured as of the effective date of termination or expiration shall survive the termination or expiration of this Agreement. In addition, the provisions of Section 2.2, (Retained Rights), 2.3 (Government Rights), 2.5 (Improvements), Article 3 (Consideration) (with respect to any final reports or to the extent any amounts are due but unpaid), Section 3.6 (Reports and Records), Article 5 (Confidentiality), Article 6 (Term and Termination), Section 8.3 (Disclaimer of Warranties, Damages), Section 8.4 (Indemnification), Section 8.5 (Insurance), Article 9 (Use of Name), and Article 10 (Additional Provisions) shall survive such termination or expiration of this Agreement in accordance with their respective terms.

ARTICLE 7: PATENT MAINTENANCE; PATENT INFRINGEMENT

7.1 Prosecution of Licensed Patents. As between Licensor and Licensee, the Parties agree as follows:

7.1.1 Licensor shall have the sole right, but not the obligation, to Prosecute patent applications and issued patents within Licensed Patents, in Licensor’s sole discretion. Subject to Section 7.1.3, Licensor shall provide Licensee with a reasonable opportunity to review and provide comments in connection with the Prosecution of the Licensed Patents; and Licensor shall keep Licensee reasonably informed as to all material developments with respect to such Licensed Patents and shall supply to Licensee copies of material communications received and filed in connection with the Prosecution of such Licensed Patents.

CONFIDENTIAL TREATMENT REQUESTED

7.1.3 Licensee acknowledges that the University of Pennsylvania controls Prosecution of the Licensed Patents, with Licensor having certain rights to review. Licensee acknowledges and agrees the rights and obligations under this Section 7.1 are subject to the rights of Licensor’s direct and indirect licensors with respect to the Licensed Patents and Licensor’s obligations under this Agreement only apply to the extent of Licensor’s rights with respect to participation in Prosecuting the Licensed Patents under its agreements with its direct and indirect licensors.

7.2 Infringement Actions Against Third Parties.

7.2.1 Licensee is responsible for notifying Licensor promptly of any infringement of Licensed Patents (other than Retained Rights) that may come to Licensee’s attention. Licensee and Licensor shall consult one another in a timely manner concerning any appropriate response to the infringement.

7.2.2 As between Licensor and Licensee, Licensor shall have the first right, but not the obligation, to prosecute any such infringement at its own expense. In any action to enforce any of the Licensed Patents, Licensee, at the request and expense of Licensor, shall cooperate to the fullest extent reasonably possible, including in the event that, if Licensor is unable to initiate or prosecute such action solely in its own name, Licensee shall join such action voluntarily and shall execute all documents necessary to initiate litigation to prosecute and maintain such action.

7.2.3 If Licensor elects not to pursue any infringement of a Licensed Patent, then, to the extent that a Licensed Product is covered by any such License Patent and such Licensed Patent is being infringed by another product in the Field (such infringement, the “Competitive Infringement”), Licensee shall have the second right, but not the obligation, to prosecute such Competitive Infringement with respect to such other product in the Field, at Licensee’s own expense. In any such action to enforce any of the Licensed Patents, Licensor, at the request and expense of Licensee, shall cooperate to the fullest extent reasonably possible, including in the event that, if Licensee is unable to initiate or prosecute such action solely in its own name, Licensor shall join such action voluntarily and shall execute all documents necessary to initiate litigation to prosecute and maintain such action. In prosecuting any such Competitive Infringement, Licensee (a) shall not take any actions that would be detrimental to the Licensed Patents and Licensor’s rights with respect thereto outside the Field and (b) shall not settle any such Competitive Infringement without the prior consent of Licensor.

7.2.4 Any recovery of damages by Licensor for any infringement other than a Competitive Infringement shall be ****. Any recovery of damages by the Party undertaking enforcement or defense of a suit for Competitive Infringement shall be applied, as between Licensor and Licensee but subject to the obligations to Licensor’s direct and indirect licensors, first to reimburse each such Party for costs and expenses (including reasonable attorneys’ fees and costs) incurred by such Party in connection with such suit, and the balance remaining, if any, from any such recovery shall be ****.

7.2.5 Licensee acknowledges and agrees that (a) the rights and obligations under this Section 7.2 are subject to the rights of Licensor’s direct and indirect licensors of the Licensed Patents (including any consent or approval rights or rights to control or participate in any enforcement actions); and (b) Licensor’s obligations under this Agreement only apply to the

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extent that Licensor has any rights with respect to enforcing the Licensed Patents under its agreements with its direct and indirect licensors. Furthermore, Licensee acknowledges the following:

7.2.5.1 All monies recovered upon the final judgment or settlement of any action with respect to Competitive Infringement will also need to be allocated to Licensor’s direct and indirect licensors (a) to reimburse the costs and expenses (including reasonable attorneys’ fees and costs) of such licensors, (b) to take into account the royalties payable to such licensors; and (c) to take into account the relative extent of such licensors’ financial participation in such action, if applicable.

7.2.5.2 Licensor’s direct and indirect licensors retain the continuing right to intervene at their own expense and join Licensor or Licensee in any claim or suit for infringement of the Licensed Patents.

7.2.5.3 In any infringement prosecuted by Licensors’ direct and indirect licensors, all financial recoveries will be ****.

7.2.5.4 In any infringement prosecuted by Licensor’s direct and indirect licensors, Licensee agrees, at the request and expense of such licensors, to cooperate to the fullest extent reasonably possible, to the same extent as though Licensor were prosecuting such suit (as provided in this Section 7.2, including Section 7.2.2).

7.2.5.5 The written consent of Licensor’s direct and indirect licensors will be required (a) for any decision that would have a materially adverse affect on the validity, scope of patent claims, or enforceability of the Patent Rights and (b) for any settlement or compromise of any infringement suit that would impose any obligations or restrictions on any of its direct or indirect licensors, or grants any rights to the Licensed Patents other than rights that Licensee has the right to grant under this Agreement.

7.3 Defense of Infringement Claims. In the event Licensee or Licensor becomes aware that Licensee’s or any of its Affiliates’ or any Sublicensees’ practice of the Licensed Patents is the subject of a claim for patent infringement by a Third Party, that Party shall promptly notify the other, and the Parties shall consider the claim and the most appropriate action to take. Licensee shall cause each of its Affiliates and each Sublicensee to notify Licensee promptly in the event such entity becomes aware that its practice of the Licensed Patents is the subject of a claim of patent infringements by another. To the extent Licensor takes any action, Licensor (or its direct or indirect licensors) shall have the right to require Licensee’s reasonable cooperation in any such suit, upon written notice to Licensee; and Licensee shall have the obligation to participate upon Licensor’s request, in which event, Licensor shall bear the cost of Licensee’s participation. Without Licensor’s prior written permission, Licensee must not settle or compromise any such suit in a manner that imposes any material obligations or restrictions on Licensor or any of its direct or indirect licensors or grants any rights to the Licensed Patents other than rights that Licensee has the right to grant under this Agreement.

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ARTICLE 8: WARRANTIES; INDEMNIFICATION

8.1 Warranty by Licensor. Licensor represents and warrants to Licensee as of the Effective Date:

8.1.1 Licensor has the right, power, and authority to enter into this Agreement and to grant to Licensee the rights specified in this Agreement;

8.1.2 This Agreement when executed shall become the legal, valid and binding obligation of it, enforceable against it, in accordance with its terms;

8.1.3 There are no actions, suits, proceedings, or arbitrations pending or, to the Licensor’s knowledge, threatened against Licensor relating to the Licensed Patents that would impact activities under this Agreement;

8.1.4 To Licensor’s knowledge, (a) the Licensed Patents are solely owned by the University of Pennsylvania, and (b) no Third Party (other than Licensor’s direct and indirect licensors) has any right, interest, or claim in or to such Licensed Patents in the Field that are inconsistent with those granted to Licensee herein;

8.1.5 To Licensor’s knowledge, Licensor does not Control as of the Effective Date any patent or patent application (other than the Licensed Patents (as defined in Section 1.13(a)) that would necessarily be infringed by the use or sale of AAV8 or AAV9 in the Field. If it is determined, in accordance with the procedure of this Section 8.1.5, that Licensor Controls as of the Effective Date a patent or patent application (other than the Licensed Patents) that would necessarily be infringed by the use or sale of AAV8 or AAV9 in the Field, then Licensee’s sole remedy shall be the inclusion of the applicable patent or patent application as a “Licensed Patent” hereunder but solely to the extent of the claim(s) that would necessarily be infringed by the use or sale of AAV8 or AAV9. At any time during the term of this Agreement, Licensee may notify Licensor in writing of any such patent or patent application that Licensee believes should be included as a “Licensed Patent” pursuant to this Section 8.1.5. Such written notice shall identify the relevant patent or patent application and relevant claim(s) and shall explain briefly why Licensee, in good faith, believes it should be included as a “Licensed Patent.” Licensor has **** following Licensor’s receipt of Licensee’s written notice to dispute the inclusion of such patent or patent application or the scope of the remedy; in which event, such dispute will be resolved in accordance with Section 10.6. Upon the Parties’ agreement (or a resolution, in favor of Licensee, of the dispute pursuant to Section 10.6), the applicable claim(s) of the applicable patent or patent application will be deemed a “Licensed Patent” hereunder. For the avoidance of doubt, Licensor makes no representation or warranty under this Section 8.1.5 as to any claim of a patent or patent application covering the manufacture of AAV8 or AAV9, and Licensee acknowledges that manufacturing claims of any patents or patent applications will not be added as “Licensed Patents” pursuant to the procedure set forth in this Section 8.1.5. For the purpose of this Section 8.1.5, “Control” means the possession by Licensor (whether by ownership or license, other than pursuant to this Agreement) of the ability to grant to Licensee access, a license, or a sublicense (as applicable) to the applicable patent or patent application on the terms and conditions set forth herein without violating the terms of any agreement or other arrangement with any Third Party;

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8.1.6 To Licensor’s knowledge, no Third Party is infringing any of the Licensed Patents in the Field; and

8.1.7 Licensor has not received any written notice from any Third Party patentee alleging infringement of, and to Licensor’s knowledge Licensor has not been sued for patent infringement of, Third Party technology by the practice of the Licensed Patents in the Field.

8.2 Warranty by Licensee. Licensee represents and warrants to Licensor as of the Effective Date that:

8.2.1 Licensee has the right, power, and authority to enter into this Agreement, to grant the rights granted by it hereunder, and to issue Licensee’s common stock to Licensor in accordance with this Agreement;

8.2.2 This Agreement when executed shall become the legal, valid and binding obligation of it, enforceable against it, in accordance with its terms;

8.2.3 Licensee has the ability and the resources, including financial resources, necessary to carry out its obligations under this Agreement;

8.2.4 There are no actions, suits, proceedings, or arbitrations pending or, to the Licensee’s knowledge, threatened against Licensee that would impact activities under this Agreement; and

8.2.5 Licensee’s common stock, when issued and delivered in accordance with the terms of Article 3, (a) will be duly and validly authorized and issued, fully paid and non-assessable, and free from all taxes, liens, and charges created by Licensee in respect of the issuance thereof, (b) will be issued in compliance with all applicable federal and state securities laws, and (c) will be free of transfer restrictions (other than the transfer restrictions imposed by any federal or state securities laws and liens or encumbrances created by or imposed by Licensor).

8.3 Disclaimer of Warranties, Damages. EXCEPT AS SET FORTH IN SECTION 8.1, THE LICENSED PATENTS, LICENSED PRODUCTS, AND ALL RIGHTS LICENSED UNDER THIS AGREEMENT ARE PROVIDED ON AN “AS IS” BASIS, AND LICENSOR MAKES NO REPRESENTATIONS OR WARRANTIES, EXPRESS OR IMPLIED, WITH RESPECT THERETO. BY WAY OF EXAMPLE BUT NOT OF LIMITATION, LICENSOR MAKES NO REPRESENTATIONS OR WARRANTIES, AND HEREBY DISCLAIMS ALL EXPRESS AND IMPLIED REPRESENTATIONS AND WARRANTIES, (i) OF COMMERCIAL UTILITY, ACCURACY, COMPLETENESS, PERFORMANCE, MERCHANTABILITY, FITNESS FOR A PARTICULAR PURPOSE, VALIDITY OR ENFORCEABILITY OF THE LICENSED PATENTS, AND PROFITABILITY; OR (ii) THAT THE USE OF THE LICENSED PATENTS OR LICENSED PRODUCTS WILL NOT INFRINGE ANY PATENT, COPYRIGHT, TRADEMARK, OR OTHER PROPRIETARY RIGHTS OF THIRD PARTIES. EXCEPT AS SET FORTH HEREIN, NONE OF LICENSOR OR ANY OF LICENSOR’S DIRECT OR INDIRECT LICENSORS SHALL BE LIABLE TO LICENSEE, LICENSEE’S SUCCESSORS OR ASSIGNS, ANY SUBLICENSEES, OR ANY THIRD PARTY WITH RESPECT TO: (a) ANY CLAIM ARISING FROM USE OF THE LICENSED PATENTS,

CONFIDENTIAL TREATMENT REQUESTED

LICENSED PRODUCTS, AND ANY OR ALL RIGHTS LICENSED UNDER THIS AGREEMENT OR FROM THE DEVELOPMENT, TESTING, MANUFACTURE, USE, OR SALE OF LICENSED PRODUCTS; OR (b) ANY CLAIM FOR LOSS OF PROFITS, LOSS OR INTERRUPTION OF BUSINESS, OR FOR INDIRECT, SPECIAL, INCIDENTAL, PUNITIVE, OR CONSEQUENTIAL DAMAGES OF ANY KIND, INCLUDING ANY ARISING FROM OR RELATING TO ANY BREACH OF THIS AGREEMENT OR THE EXERCISE OF RIGHTS HEREUNDER, REGARDLESS OF ANY NOTICE OF SUCH DAMAGES. NOTHING IN THIS SECTION 8.3 IS INTENDED TO LIMIT OR RESTRICT THE INDEMNIFICATION RIGHTS OR OBLIGATIONS OF EITHER PARTY UNDER SECTION 8.4 OR TO LIMIT A PARTY’S LIABILITY FOR BREACHES OF ITS OBLIGATION REGARDING CONFIDENTIALITY UNDER ARTICLE 5.

8.4 Indemnification.

8.4.1 By Licensee. Licensee shall defend, indemnify, and hold harmless Licensor, its direct and indirect licensors of the Licensed Patents, and their respective shareholders, members, officers, trustees, faculty, students, contractors, agents, and employees (individually, a “Licensor Indemnified Party” and, collectively, the “Licensor Indemnified Parties”) from and against any and all Third Party liability, loss, damage, action, claim, fee, cost, or expense (including attorneys’ fees) (individually, a “Third Party Liability” and, collectively, the “Third Party Liabilities”) suffered or incurred by the Licensor Indemnified Parties from claims of such Third Parties that results from or arises out of: ****; provided, however, that Licensee shall not be liable for claims based on any breach by Licensor of the representations, warranties, or obligations of this Agreement or the gross negligence or intentional misconduct of any of the Licensor Indemnified Parties. Without limiting the foregoing, Licensee must defend, indemnify, and hold harmless the Licensor Indemnified Parties from and against any Third Party Liabilities resulting from:

8.4.1.1 any **** or other claim of any kind related to the **** by a Third Party of a Licensed Product that **** by Licensee, its Affiliates, any Sublicensees, their respective assignees, or vendors;

8.4.1.2 any claim by a Third Party that the ****; and

8.4.1.3 **** conducted by or on behalf of Licensee, its Affiliates, any Sublicensees, their respective assignees, or vendors relating to the Licensed Patents or Licensed Products, including any claim by or ****.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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8.4.2 By Licensor. Licensor shall defend, indemnify, and hold harmless Licensee, its shareholders, members, officers, contractors, agents, and employees (individually, a “Licensee Indemnified Party” and, collectively, the “Licensee Indemnified Parties”) from and against any and all Third Party Liabilities suffered or incurred by the Licensee Indemnified Parties from claims of such Third Parties that results from or arises out of: ****; provided, however, that Licensor shall not be liable for claims based on any breach by Licensee of the representations, warranties, or obligations of this Agreement or the gross negligence or intentional misconduct of any of the Licensee Indemnified Parties.

8.4.3 Indemnification Procedure. Each Party, as an indemnifying party (a “Indemnifying Party” , shall not be permitted to settle or compromise any claim or action giving rise to Third Party Liabilities in a manner that imposes any restrictions or obligations on any indemnified party (a “Indemnified Party”) without the other Party’s prior written consent or, if Licensee is the Indemnifying Party, that grants any rights to the Licensed Patents or Licensed Products other than those Licensee has the right to grant under this Agreement without Licensor’s prior written consent. The Indemnifying Party shall be permitted to control any litigation or potential litigation involving the defense of any claim subject to indemnification pursuant to this Section 8.4, including the selection of counsel, with the reasonable approval of the Indemnified Party. If an Indemnifying Party fails or declines to assume the defense of any such claim or action within **** after notice thereof, the Indemnified Party may assume the defense of such claim or action at the cost and risk of the Indemnifying Party, and any Third Party Liabilities related thereto shall be conclusively deemed a Third Party Liability of the Indemnifying Party. The indemnification rights of a Indemnified Party contained in this Agreement are in addition to all other rights which such Indemnified Party may have at law or in equity or otherwise. The Indemnifying Party will pay directly all Third Party Liabilities incurred for defense or negotiation of any claim hereunder or will reimburse the Indemnified Party for all documented Third Party Liabilities incident to the defense or negotiation of any such claim within **** after the Indemnifying Party’s receipt of invoices for such fees, expenses, and charges.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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adequacy of the minimum amounts of insurance for each coverage required by this Section 8.5, and Licensor reserves the right to require Licensee to adjust the limits accordingly. The required minimum amounts of insurance do not constitute a limitation on Licensee’s liability or indemnification obligations to the Licensor Indemnified Parties under this Agreement. The policies of insurance required by this Section 8.5 will be issued by an insurance carrier with an A.M. best rating of **** or better and will name Licensor as an additional insured with respect to Licensee’s performance (and its Affiliates’ and any Sublicensees’) under this Agreement. Licensee will provide Licensor with insurance certificates evidencing the required coverage within **** after the Effective Date and the commencement of each policy period and any renewal periods. Upon Licensor’s written request, each certificate will provide that the insurance carrier will notify Licensor in writing at least **** prior to the cancellation or material change in coverage. Licensee will cause all Sublicensees to comply with the terms of this Section 8.5 to the same extent as Licensee.

ARTICLE 9: USE OF NAME

ARTICLE 10: ADDITIONAL PROVISIONS

10.2 Assignment. The rights and obligations of Licensee and Licensor hereunder shall inure to the benefit of, and shall be binding upon, their respective permitted successors and assigns. Licensee may not assign this Agreement or any of its rights or obligations under this Agreement without the prior written consent of Licensor; provided, however, that Licensee may assign this Agreement, without Licensor’s prior written consent, pursuant to a merger or sale of all or substantially all of the assets to which the Agreement relates; provided that, as part of any permitted assignment, (a) Licensee provides Licensor with notice of such assignment at least 5 business days prior to the effectiveness of such assignment, and (b) Licensee requires any such assignee to agree in writing to be legally bound by this Agreement to the same extent as Licensee and provides Licensor with a copy of such assignee undertaking. Licensor may assign this Agreement and its rights and obligations without the consent of Licensee. No assignment shall relieve the assigning Party of responsibility for the performance of any accrued obligations which it has prior to such assignment. Any attempted assignment by Licensee in violation of this Section 10.2 shall be null and void and of no legal effect.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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If for Licensor:		with a copy to:

	ReGenX Biosciences, LLC		ReGenX Biosciences, LLC
	50 17th Street, NW		750 17th Street, NW
	Suite 1100		Suite 1100
	Washington, DC 20006		Washington, DC 20006
	Attn: Chief Executive Officer		Attn: General Counsel
	Telephone: 202-785-7438		Telephone: 202-785-7438
	Facsimile: 202-785-7439		Facsimile: 202-785-7439

If for Licensee:		with a copy to:

	Audentes Therapeutics, Inc.		Fenwick and West, LLP.
	****		1191 Second Avenue, 10th Floor
	San Francisco, California, ****		Seattle, WA 98101
	Attn: Matthew Patterson, President &		Attn: Effie Toshav
	CEO
	Telephone: 646-712-1001		Telephone: 206.389.4510
	Email: mpatterson@audentestx.com		Facsimile: 206-389-4511

Either Party may change its official address upon written notice to the other Party.

10.5 Applicable Law. This Agreement shall be construed and governed in accordance with the laws of the State of Delaware, without giving effect to conflict of law provisions that may require the application of the laws of another jurisdiction. Subject to Section 10.6, the Parties hereby submit to the exclusive jurisdiction of and venue in the courts located in the State of Delaware with respect to any and all disputes concerning the subject of this Agreement.

10.6 Dispute Resolution. In the event of any controversy or claim arising out of or relating to this Agreement, the Parties shall first attempt to resolve such controversy or claim through good faith negotiations for a period of not less than **** following notification of such controversy or claim to the other Party. If such controversy or claim cannot be resolved by means of such negotiations during such period, then such controversy or claim shall be resolved by binding arbitration administered by the American Arbitration Association (“AAA”) in accordance with the Commercial Arbitration Rules of the AAA in effect on the date of commencement of the arbitration, subject to the provisions of this Section 10.6. The arbitration shall be conducted as follows:

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10.6.1 The arbitration shall be conducted by three arbitrators, each of whom by training, education, or experience has knowledge of the research, development, and commercialization of biological therapeutic products in the United States. The arbitration shall be conducted in English and held in New York, New York.

10.6.4 The arbitrators will render their award in writing and, unless all Parties agree otherwise, will include an explanation in reasonable detail of the reasons for their award. Judgment upon the award rendered by the arbitrators may be entered in any court having jurisdiction thereof, including in the courts described in Section 10.5. The arbitrators will have the authority to grant injunctive relief and other specific performance; provided that the arbitrators will have no authority to award damages in contravention of this Agreement, and each Party irrevocably waives any claim to such damages in contravention of this Agreement. The arbitrators will, in rendering their decision, apply the substantive law of the State of Delaware, without giving effect to conflict of law provisions that may require the application of the laws of another jurisdiction. The decision and award rendered by the arbitrators will be final and non-appealable (except for an alleged act of corruption or fraud on the part of the arbitrator).

10.6.5 The Parties shall use their reasonable efforts to conduct all dispute resolution procedures under this Agreement as expeditiously, efficiently, and cost-effectively as possible.

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irreparable harm that may be caused by the other Party’s actual or threatened breach of this Agreement.

10.7 No Discrimination. Licensee, its Affiliates, and any Sublicensees, in their respective activities under this Agreement, shall not discriminate against any employee or applicant for employment because of race, color, sex, sexual, or affectional preference, age, religion, national, or ethnic origin, handicap, or because he or she is a disabled veteran or a veteran (including a veteran of the Vietnam Era).

10.9 Entire Agreement. This Agreement embodies the entire understanding between the Parties relating to the subject matter hereof and supersedes all prior understandings and agreements, whether written or oral, including that certain Mutual Non-Disclosure Agreement between the Parties dated December 3, 2012. All “Confidential Information” disclosed by the Parties pursuant to such Mutual Non-Disclosure Agreement shall be deemed “Confidential Information” under this Agreement (unless and until it falls within one of the exclusions set forth in Section 1.5). This Agreement may not be varied except by a written document signed by duly authorized representatives of both Parties.

10.10 Marking. Licensee, its Affiliates, and any Sublicensees shall mark any Licensed Product (or their containers or labels) made, sold, or otherwise distributed by it or them with any notice of patent rights necessary or desirable under applicable law to enable the Licensed Patents to be enforced to their full extent in any country where Licensed Products are made, used, sold, offered for sale, or imported.

10.11 Severability and Reformation. If any provision of this Agreement is held to be invalid or unenforceable by a court of competent jurisdiction, then such invalid or unenforceable provision will be automatically revised to be a valid or enforceable provision that comes as close as permitted by law to the Parties’ original intent; provided that, if the Parties cannot agree upon such valid or enforceable provision, the remaining provisions of this Agreement will remain in full force and effect, unless the invalid or unenforceable provisions are of such essential importance to this Agreement that it is to be reasonably assumed that the Parties would not have entered into this Agreement without the invalid or unenforceable provisions.

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10.12 Further Assurances. Each Party hereto agrees to execute, acknowledge, and deliver such further instruments, and to do all other acts, as may be necessary or appropriate in order to carry out the purposes and intent of this Agreement.

10.13 Interpretation; Construction. The captions to the several Articles and Sections of this Agreement are included only for convenience of reference and shall not in any way affect the construction of, or be taken into consideration in interpreting, this Agreement. In this Agreement, unless the context requires otherwise, (a) the word “including” shall be deemed to be followed by the phrase “without limitation” or like expression; (b) references to the singular shall include the plural and vice versa; (c) references to masculine, feminine, and neuter pronouns and expressions shall be interchangeable; (d) the words “herein” or “hereunder” relate to this Agreement; (e) “or” is disjunctive but not necessarily exclusive; (f) the word “will” shall be construed to have the same meaning and effect as the word “shall”; (g) all references to “dollars” or “$” herein shall mean U.S. Dollars; (h) unless otherwise provided, all reference to Sections and exhibits in this Agreement are to Sections and exhibits of and in this Agreement; and (i) whenever this Agreement refers to a number of days, such number shall refer to calendar days unless business days are specified. Business days shall mean a day on which banking institutions in Washington, D.C. are open for business. Each Party represents that it has been represented by legal counsel in connection with this Agreement and acknowledges that it has participated in the drafting hereof. In interpreting and applying the terms and provisions of this Agreement, the Parties agree that no presumption will apply against the Party which drafted such terms and provisions.

10.15 Counterparts. This Agreement may be executed in one or more counterparts, each of which will be deemed an original, but all of which together will constitute one and the same instrument.

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IN WITNESS WHEREOF, the Parties, intending to be legally bound, have caused this License Agreement to be executed by their duly authorized representatives.


REGENX BIOSCIENCES, LLC		AUDENTES THERAPEUTICS, INC.

By:	/s/ Kenneth T. Mills	By:	/s/ Matthew Patterson
Name:	Kenneth T. Mills	Name:	Matthew Patterson
Title:	President & CEO	Title:	President & CEO

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Exhibit A

Licensed Patents

Part 1, Licensed AAV8 Patents


Application #	Patent #	Filing Date*	Country	Status
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****

*	International Filing Date, where national stage application or foreign divisional thereof

Part 2, Licensed AAV9 Patents


Application #	Patent #	Filing Date	Country	Status
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****
****	****	****	****	****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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Exhibit B

Press Release

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LOGO

REGENX Biosciences and Audentes Therapeutics Enter into Exclusive License Agreement for

Development of Treatments for Serious, Rare Muscle Diseases Using NAV^TM Vectors

WASHINGTON & SAN FRANCISCO—(BUSINESS WIRE)—REGENX Biosciences, LLC and Audentes Therapeutics, Inc. announce that they have entered into an agreement for the development and commercialization of products to treat X-Linked Myotubular Myopathy (XLMTM) and Pompe disease using NAV vectors.

Under the terms of the Agreement, REGENX granted Audentes an exclusive worldwide license, with rights to sublicense, to REGENX’s NAV rAAV8 and rAAV9 vectors for treatment of XLMTM and Pompe disease in humans. In return for these rights, REGENX receives an up-front payment, certain milestone fees and royalties on net sales of products incorporating NAV rAAV8 and rAAV9.

“We believe this exclusive license agreement is important to the successful development of NAV-based gene delivery treatments for patients with XLMTM and Pompe disease,” said Ken Mills, President and CEO of REGENX. “As a leader in gene therapy, we are pleased to be cooperating with the team at Audentes in its pursuit of developing innovative treatments for patients with serious, rare muscle diseases through the application of NAV technology. REGENX has a continued interest to provide commercial partners that evidence outstanding leadership, expertise, resources and a strong commitment to patients, such as Audentes, with access to our NAV technology.”

“Audentes is committed to the development of new treatments for patients with XLMTM and Pompe disease using AAV gene therapy technology and we feel rAAV8 and rAAV9 are the most promising vectors to achieve this goal,” said Matthew R. Patterson, President and CEO of Audentes. “We are very pleased to enter into this agreement with REGENX, which we believe offers us the best path to expeditiously develop novel therapies for patients.”

About X-Linked Myotubular Myopathy (XLMTM)

X-Linked Myotubular Myopathy (XLMTM) is a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment. It is caused by mutations in the MTM1 gene, which encodes an enzyme called myotubularin. Myotubularin is thought to be involved in the development and maintenance of muscle cells. XLMTM affects approximately 1 in 50,000 newborn males worldwide.

About Pompe Disease

Pompe Disease is a rare, inherited disorder characterized by progressive muscle weakness and respiratory impairment. It is caused by mutations in a gene that encodes an enzyme called acid alpha-glucosidase (GAA), which is needed by the body to break down glycogen — a stored form of sugar used for energy. Pompe Disease affects approximately 1 in every 40,000 births.

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About REGENX Biosciences

REGENX Biosciences is leading the effort to translate promising gene delivery applications into a pipeline of next generation personalized therapies for a range of severe diseases with serious unmet needs. We believe that the NAV technology to which we have exclusive rights represents the potential promise of curing the root cause of disease rather than the symptoms, and we are committed to establishing best in class standards for our NAV vectors. Our intent is to initially develop treatments for a number of rare, genetic diseases including hypercholesterolemias, the mucopolysaccharidoses, and retinitis pigmentosa and ensure continuing access for our NAV technology through innovative partnerships, license opportunities and the expansion of our growing team of global collaborators. REGENX holds exclusive rights to a portfolio of over 100 patents and patent applications pertaining to its NAV technology and related applications.

For more information regarding REGENX, please visit www regenxbio.com.

About Audentes Therapeutics, Inc.

Audentes^TM is a biotechnology company committed to the development and commercialization of innovative new treatments for people with serious, rare muscle diseases through the application of adeno-associated virus (AAV) gene therapy technology. The company consists of a focused, experienced, and passionate team driven by the goal of improving the lives of patients. Audentes takes pride in strong, global relationships with the patient, research, and medical communities.

For more information regarding Audentes, please visit www.audentestx corn.

Contacts

REGENX Biosciences

Vit Vasista, 202-785-7438

vvasista@regenxbio com

Audentes Therapeutics, Inc.

Matthew Patterson, 646-712-1001

mpatterson@audentestx com

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EXHIBIT C

****

[9 pages omitted]

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****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

EX-10.16

Exhibit 10.16

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LICENSE AGREEMENT

This LICENSE AGREEMENT (“Agreement”) is entered into as of March 21, 2014 (“Effective Date”) by and between ReGenX Biosciences, LLC, a limited liability company organized under the laws of the State of Delaware, with offices at 750 17th Street, NW, Suite 1100, Washington, DC 20006 (“Licensor”), and AveXis, Inc. (formerly known as BioLife Cell Bank, Inc.), a corporation organized under the laws of the State of Delaware, with offices at 4925 Greenville Avenue, Suite 604, Dallas, TX 75206 (“Licensee”). Licensor and Licensee are hereinafter referred to individually as a “Party” and collectively as the “Parties.”

WHEREAS, Licensor has rights under certain Licensed Patents (as defined herein) pertaining to adeno-associated virus serotype 9; and

WHEREAS, Licensee desires to obtain an exclusive license under the Licensed Patents under the terms set forth herein;

NOW, THEREFORE, in consideration of the promises and covenants contained in this Agreement, and intending to be legally bound, the Parties hereby agree as follows:

ARTICLE 1: DEFINITIONS

1.1 “AAV9” means (a) the recombinant adeno-associated virus serotype 9 vector with the specified sequence set forth in GenBank **** and (b) any recombinant adeno-associated virus derivatives of such serotype 9 vector that are covered by the claims of the Licensed Patents.

1.2 “Affiliate” means any legal entity directly or indirectly, during the term of this Agreement, controlling, controlled by, or under common control with another entity. For purposes of this Agreement, “control” means the direct or indirect ownership of more than 50% of the outstanding voting securities of a legal entity, or the right to receive more than 50% of the profits or earnings of a legal entity, or the right to control the policy decisions of a legal entity. An entity may be or become an Affiliate of an entity and may cease to be an Affiliate of an entity, in each case, during the term of this Agreement.

1.3 “Calendar Quarter” means each three-month period or any portion thereof, beginning on January 1, April 1, July 1, and October 1.

1.4 “Confidential Information” means and includes all technical information, inventions, developments, discoveries, software, know-how, methods, techniques, formulae, animate and inanimate materials, data, processes, finances, business operations or affairs, and other proprietary ideas, whether or not patentable or copyrightable, of either Party that are (a) marked or otherwise identified as confidential or proprietary at the time of disclosure in writing; or (b) if disclosed orally, visually, or in another non-written form, identified as confidential at the time of disclosure and summarized in reasonable detail in writing as to its general content within 30 days after original disclosure. The Parties acknowledge that (i) the terms and conditions of this Agreement and (ii) the records and reports referred to in Section 3.6 will be deemed the Confidential Information of both Parties, regardless of whether such information is marked or identified as confidential. In addition, information provided to Licensee pursuant to the

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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provisions of Section 7.1 will be deemed the Confidential Information of Licensor, regardless of whether such information is marked or identified as confidential. Notwithstanding the foregoing, Confidential Information will not include the following, in each case, to the extent evidenced by competent written proof of the Receiving Party:

1.4.1 information that was already known to the Receiving Party, other than under an obligation of confidentiality, at the time of disclosure by the Disclosing Party;

1.4.2 information that was generally available to the public or otherwise part of the public domain at the time of its disclosure to the Receiving Party;

1.4.3 information that became generally available to the public or otherwise part of the public domain after its disclosure, other than through any act or omission of the Receiving Party in breach of this Agreement;

1.4.4 information that is independently discovered or developed by the Receiving Party without the use of Confidential Information of the Disclosing Party; or

1.4.5 information that was disclosed to the Receiving Party, other than under an obligation of confidentiality, by a Third Party who had no obligation to the Disclosing Party not to disclose such information to others.

1.5 “Disclosing Party” has the meaning set forth in Section 5.1.

1.6 “Domain Antibody” ****.

1.7 “FDA” means the United States Food and Drug Administration, or a successor agency in the United States with responsibilities comparable to those of the United States Food and Drug Administration.

1.8 “Field” means the treatment of spinal muscular atrophy in humans by in vivo gene therapy using AAV9.

1.9 “GSK Agreement” means that certain License Agreement entered into between Licensor and SmithKline Beecham Corporation, effective on March 6, 2009, as amended by that certain Amendment to License Agreement dated April 15, 2009, and as amended from time to time.

1.10 “Licensed Patents” means, to the extent they cover AAV9, (a) all United States patents and patent applications listed in Exhibit A, and (b) any re-examination certificates thereof, and their foreign counterparts and extensions, continuations, divisionals, and re-issue applications.

1.11 “Licensed Product” means (a) any AAV9 product that is made, made for, used, sold, offered for sale, or imported by Licensee, its Affiliates, and any of its or their Sublicensees, the manufacture, use, sale, offer for sale, or import of which product, in the absence of the license granted pursuant to this Agreement, would infringe or is covered by at least one Valid Claim in the country of manufacture, use, sale, offer for sale, or import, including products manufactured

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

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by a process that would infringe or is covered by at least one Valid Claim in the country of manufacture, use, sale, offer for sale, or import; or (b) any service sold by Licensee, its Affiliates, and any of its or their Sublicensees with respect to the administration of any AAV9 product to patients that, in the absence of the licenses granted pursuant to this Agreement, would infringe or is covered by at least one Valid Claim in the country of sale.

1.12 “NDA” means a New Drug Application filed with the FDA as described in 21 C.F.R. § 314, a Biological License Application (BLA) pursuant to 21 C.F.R. § 601.2, or any equivalent or any corresponding application for regulatory approval in any country or regulatory jurisdiction other than the United States.

1.13 “Net Sales” means the gross receipts from sales or other disposition of a Licensed Product (including fees for services within the definition of “Licensed Product”) by Licensee and/or its Affiliates and/or any Sublicensees to Third Parties less the following deductions that are directly attributable to a sale, specifically and separately identified on an invoice or other documentation and actually borne by Licensee, its Affiliates, or any Sublicensees: ****. In the event consideration other than cash is paid to Licensee, its Affiliates, or any Sublicensees, for purposes of determining Net Sales, the Parties shall use the cash consideration that Licensee, its Affiliates, or any Sublicensees would realize from an unrelated buyer in an arm’s length sale of an identical item sold in the same quantity and at the time and place of the transaction, as determined jointly by Licensor and Licensee based on transactions of a similar type and standard industry practice, if any.

1.14 “Penn Agreement” means that certain License Agreement entered into between Licensor and The Trustees of the University of Pennsylvania, effective on February 24, 2009, as amended by that letter agreement dated March 6, 2009, and as amended from time to time.

1.15 “Phase 3 Clinical Trial” means a pivotal clinical trial in humans performed to gain evidence with statistical significance of the efficacy of a product in a target population, and to obtain expanded evidence of safety for such product that is needed to evaluate the overall benefit-risk relationship of such product, to form the basis for approval of an NDA and to provide an adequate basis for physician labeling, as described in 21 C.F.R. § 312.21(c) or the corresponding regulation in jurisdictions other than the United States.

1.16 “Prosecute” means preparation, filing, and prosecuting patent applications and maintaining patents, including any reexaminations, reissues, oppositions, inter partes review, and interferences.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

1.17 “Receiving Party” has the meaning set forth in Section 5.1.

1.18 “ReGenX Licensors” means SmithKline Beecham Corporation (or any successor thereto under the GSK Agreement) and The Trustees of the University of Pennsylvania (or any successor thereto under the Penn Agreement).

1.19 “Retained Rights” has the meaning set forth in Section 2.2.

1.20 “Sublicensee” means (i) any Third Party or Affiliate to whom Licensee grants a sublicense of some or all of the rights granted to Licensee under this Agreement as permitted by this Agreement; and (ii) any other Third Party or Affiliate to whom a sublicensee described in clause (i) has granted a further sublicense as permitted by this Agreement.

1.21 “Third Party” means any person or entity other than a Party to this Agreement or Affiliates of a Party to this Agreement.

1.22 “Valid Claim” means a claim of an issued and unexpired patent (including any patent claim the term of which is extended by any extension, supplementary protection certificate, patent term restoration, or the like) included within the Licensed Patents or a claim of a pending patent application included within the Licensed Patents, which has not lapsed, been abandoned, been held revoked, or been deemed unenforceable or invalid by a non-appealable decision or an appealable decision from which no appeal was taken within the time allowed for such appeal of a court or other governmental agency of competent jurisdiction.

ARTICLE 2: LICENSE GRANT

2.1 License Grant. Subject to the terms and conditions of this Agreement, including the Retained Rights, Licensor hereby grants to Licensee an exclusive, sublicensable (as provided in Section 2.4 only), non-transferable (except as provided in Section 10.2), royalty-bearing, worldwide license, under the Licensed Patents to make, have made, use, import, sell, and offer for sale Licensed Products solely in the Field, including, for the avoidance of doubt, the right to conduct research and development.

2.2 Retained Rights. Except for the rights and licenses specified in Section 2.1, no license or other rights are granted to Licensee under any intellectual property of Licensor, whether by implication, estoppel, or otherwise and whether such intellectual property is subordinate, dominant, or otherwise useful for the practice of the Licensed Patents. Notwithstanding anything to the contrary in this Agreement, Licensor may use and permit others to use the Licensed Patents for any research, development, commercial, or other purposes outside of the Field. Without limiting the foregoing, and notwithstanding anything in this Agreement to the contrary, Licensee acknowledges and agrees that the following rights are retained by Licensor and the ReGenX Licensors (individually and collectively, the “Retained Rights”), whether inside or outside the Field:

2.2.1 The rights and licenses granted in Section 2.1 shall not include any right (and Licensor and the ReGenX Licensors retain the exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents to make, have made, use, sell, offer to sell, and import Domain Antibodies that are expressed by an adeno-associated vector, including AAV9.

CONFIDENTIAL TREATMENT REQUESTED

2.2.2 Licensor and the ReGenX Licensors retain the following rights with respect to the Licensed Patents:

(a)

(b)

A non-exclusive right for the ReGenX Licensors (which right is sublicensable by the ReGenX Licensors) to use the Licensed Patents for non-commercial research purposes and to use the Licensed Patents for such ReGenX Licensors’ discovery research efforts with non-profit organizations and collaborators.

2.2.3 The rights and licenses granted in Section 2.1 shall not include any right (and Licensor retains the exclusive (even as to Licensee), fully sublicensable right) under the Licensed Patents:

(a)

to conduct commercial reagent and services businesses, which includes the right to make, have made, use, sell, offer to sell, and import research reagents, including any viral vector construct; provided that, for clarity, such rights retained by Licensor shall not include the right to conduct clinical trials in humans in the Field; or

(b)

2.2.4 Licensor retains the fully sublicensable right under the Licensed Patents to grant non-exclusive research and development licenses to Affiliates and Third Parties; provided that such development rights granted by Licensor shall not include the right to conduct clinical trials in humans in the Field or any rights to sell products in the Field.

2.2.5 The Trustees of the University of Pennsylvania may use and permit other non-profit organizations or other non-commercial entities to use the Licensed Patents for educational and research purposes.

2.4 Sublicensing.

CONFIDENTIAL TREATMENT REQUESTED

2.4.2 The right to sublicense granted to Licensee under this Agreement is subject to the following conditions:

(a)

Licensee may only grant sublicenses pursuant to a written sublicense agreement with the Sublicensee; ****. Licensor must receive written notice as soon as practicable following execution of any such sublicenses. Any further sublicenses granted by any Sublicensees (to the extent permitted hereunder) must comply with the provisions of this Section 2.4 (including Section 2.4.2) to the same extent as if Licensee granted such sublicense directly.

(b)

(c)

The official language of any sublicense agreement shall be English.

(d)

Within **** after entering into a sublicense, Licensor must receive a copy of the sublicense written in the English language for Licensor’s records and to share with the ReGenX Licensors. The copy of the sublicense may be redacted to exclude confidential information of the applicable Sublicensee, but such copy shall not be redacted to the extent that it impairs Licensor’s (or the ReGenX Licensors’) ability to ensure compliance with this Agreement; provided that, if either of the ReGenX Licensors requires a complete, unredacted copy of the sublicense, Licensee shall provide such complete, unredacted copy.

(e)

2.5 Improvements.

2.5.1 Licensee hereby grants to Licensor a non-exclusive, worldwide, royalty-free, transferable, sublicensable, irrevocable, perpetual license:

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

(a)

to use any Licensed Back Improvements (and any intellectual property rights with respect thereto) consummate in scope to the Retained Rights, and

(b)

to practice the Licensed Back Improvements (and any intellectual property rights with respect thereto) in connection with AAV9, including the right to research, develop, make, have made, use, offer for sale, and sell products and services; provided that Licensor shall have no right, under the license in this Section 2.5.1(b), to practice the Licensed Back Improvements in the Field.

2.5.2 For purposes of this Agreement, “Licensed Back Improvements” means any patentable modifications or improvements developed by Licensee, any Affiliates, or any Sublicensees to any vector that is the subject of a claim within the Licensed Patents.

2.5.3 Licensee agrees to provide prompt notice to Licensor upon the filing of any patent application covering any Licensed Back Improvement, together with a reasonably detailed description of or access to such Licensed Back Improvement to permit the practice of any such invention or improvement.

ARTICLE 3: CONSIDERATION

3.1 Initial Fee. In consideration of the license granted to Licensee under Section 2.1, Licensee shall pay Licensor an initial fee of $2,000,000, which shall be payable as follows: (i)**** upon the Effective Date, (ii) **** within **** after the Effective Date, and (iii) **** within **** after the Effective Date; provided that any unpaid portion of the initial fee will be immediately payable upon any termination of this Agreement.

3.3 Milestone Fees. In consideration of the license granted to Licensee under Section 2.1, Licensee shall pay Licensor the following milestone payments:


Milestone	Milestone Payment
1. First treatment of the **** human subject in a clinical trial (i.e., **** patient, first dose)		****
2. First treatment in Phase 3 Clinical Trial (i.e., first patient, first dose)		****
3. First NDA submission for a Licensed Product in the United States		****
4. First NDA submission for a Licensed Product in the European Union		****
5. First NDA approval for a Licensed Product in the United States		****

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED


6. First NDA approval for a Licensed Product in the European Union		****

Total:	$	12,250,000.00

For clarity, the milestone payments set forth in this Section 3.3 are payable **** with respect to each milestone event, ****.

3.4 Royalties. In further consideration of the license granted to Licensee under Section 2.1, Licensee shall pay to Licensor the following royalties based upon Net Sales of Licensed Products, subject to the reductions in royalty rates set forth in Section 3.4.1:


Cumulative Annual Net Sales of all Licensed Products Worldwide	Royalty Percentage
Portion of Net Sales in a calendar year less than ****		****
Portion of Net Sales in a calendar year between (and including) ** through (and including) **		****
Portion of Net Sales in a calendar year greater than ****		****

By way of example only, if Licensee receives $700,000,000 in cumulative Net Sales of all Licensed Products in a calendar year, then the royalties payable by Licensee to Licensor under this Section 3.4 during such calendar year would be calculated as follows:

= (****)(****) + (****)(****) + (****)(****)

= (****) + (****) + (****)

= ****

STACKING ROYALTY CALCULATIONS

R = (C * (A / (A+B)))

Where

R = reduction of Licensor royalty,

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT TO THE OMITTED PORTIONS.

CONFIDENTIAL TREATMENT REQUESTED

A = unreduced Licensor royalty,

B = sum of all Third Party royalties,

C = increment of projected total royalty above ****

Example Calculation:


Assume		i) all Third Party royalties = ****
		ii) unreduced Licensor royalty = ****
		iii): projected total royalty = ****

R = (**** – ****) * (**** / (**** + ****))

R = (**** * ****)

R = ****

Licensor Stacked Royalty = **** – **** = **** (but subject to the cap described below)

3.4.2 Royalty Payment Period. Licensee’s obligation hereunder for payment of a royalty under this Section 3.4 on the Net Sales of Licensed Products in a given country will end on a country-by-country basis when the Licensed Product ceases to infringe or be covered by a Valid Claim in that country.

3.5 Sublicense Fees.

3.5.1 In further consideration of the license granted to Licensee under Section 2.1, Licensee will pay Licensor **** of any sublicense fees (****) received by Licensee or its Affiliates from a Third Party for the Licensed Patents from any Sublicensee or from any Third Party granted any option to obtain a sublicense.

3.5.2 With respect to the obligations under this Section 3.5, Licensee shall not be required to submit any amounts received from a Third Party for the following:

(a)

Reimbursement for research, development, and/or manufacturing activities performed by Licensee or its Affiliates corresponding directly to the development of Licensed Products pursuant to a specific agreement;

(b)

Any and all amounts paid to Licensee or its Affiliates by a Sublicensee as royalties on sales of Licensed Product sold by the Sublicensee under a sublicense agreement; and

(c)

Consideration received for the purchase of an equity interest in Licensee or its Affiliates at fair market value.

****CERTAIN INFORMATION HAS BEEN OMITTED AND FILED SEPARATELY WITH THE COMMISSION. CONFIDENTIAL TREATMENT HAS BEEN REQUESTED WITH RESPECT