Release Details

ROCKVILLE, Md., Oct. 1, 2021 /PRNewswire/ --

• Data for Cohort 1 with three months of follow-up from the RGX-314 Phase II trial for the treatment of diabetic retinopathy (ALTITUDE^®) to be presented

REGENXBIO Inc. (Nasdaq: RGNX) today announced that data from the RGX-314 clinical trials will be presented in three oral presentations at the American Society of Retina Specialists Annual Meeting taking place in San Antonio, TX, from October 8 to 12, 2021. The presentations will include interim results at three months of follow-up for patients in Cohort 1 (dose level: 2.5x10¹¹ genome copies per eye (GC/eye)) of the Phase II ALTITUDE^® trial in patients with diabetic retinopathy (DR). This trial is designed to evaluate the in-office, suprachoroidal delivery of RGX-314.

The American Society of Retina Specialists Annual Meeting presentations include:

Title: Suprachoroidal Delivery of RGX-314 for Diabetic Retinopathy Without CI-DME: Early Results From the Phase II ALTITUDE Study
Presenter: Dennis Marcus, M.D., F.A.S.R.S., Southeast Retina Center
Session Title: Diabetic Retinopathy 2 Symposium
Date/Time: Saturday, October 9, 2021, from 11:31 to 11:35 a.m. CT
Type: Oral presentation

Title: Subretinal Delivery of RGX-314 for Neovascular AMD: End of Study Phase I/IIa Results (Encore Presentation)
Presenter: Lejla Vajzovic, M.D., F.A.S.R.S., Surgical & Medical Retina Specialist & Director of Duke Eye Center Continuing Medical Education
Session Title: Wet AMD 1 Symposium
Date/Time: Monday, October 11, 2021, from 8:38 to 8:44 a.m. CT
Type: Oral presentation

Title: Suprachoroidal Delivery of RGX-314 for Neovascular AMD: Initial Results From the Phase II AAVIATE Study (Encore Presentation)
Presenter: Mark Barakat, M.D., F.A.S.R.S., Director of Research, Retinal Consultants of Arizona; Clinical Assistant Professor, University of Arizona College of Medicine, Phoenix
Session Title: Wet AMD 1 Symposium
Date/Time: Monday, October 11, 2021, from 8:44 to 8:50 a.m. CT
Type: Oral presentation

About RGX-314

RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector^® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV^® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

SCS Microinjector^® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

Contacts:

Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com

Investors:
Brendan Burns, 212-600-1902
brendan@argotpartners.com

Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com

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SOURCE REGENXBIO Inc.