2014 Press Releases

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11/18/14Dr. James M. Wilson Joins REGENXBIO as Chief Scientific Advisor
Renowned gene therapy pioneer to take active role in accelerating development of groundbreaking treatmentsWASHINGTON, DC November 18, 2014 – REGENXBIO Inc. today announced that James M. Wilson, M.D., Ph.D. has been named its Chief Scientific Advisor, effective immediately. Dr. Wilson is also the director of the Gene Therapy Program and professor of Pathology and Laboratory Medicine at the Perelman School of Medicine at the University of Pennsylvania (Penn).REGENXBIO’s NAV® Technology, which incl... 
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10/28/14REGENXBIO Appoints Dr. Stephen Yoo as Chief Medical Officer
WASHINGTON, DC October 28, 2014 – REGENXBIO Inc. today announced the appointment of Stephen Yoo, M.D. as Chief Medical Officer, effective immediately. Dr. Yoo will oversee the development of REGENXBIO’s AAV gene therapy treatments for lysosomal storage disorders and ocular diseases and serve on the company’s management team.“We are pleased to welcome Stephen as our first Chief Medical Officer,” said Ken Mills, President and CEO of REGENXBIO. “Stephen’s extensive experience leading clinical devel... 
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09/29/14REGENXBIO’S NAV® Technology Demonstrates Correction of Cardiovascular Symptoms of MPS I
Results highlight potential for development of novel AAV-mediated gene therapy treatments for lysosomal storage diseases WASHINGTON, DC September 29, 2014 – REGENXBIO Inc. today announced that gene transfer mediated by REGENXBIO’s NAV® AAV8 vectors resulted in sustained serum α-L-iduronidase (IDUA) expression, as well as correction of systemic features of MPS I, or Hurler syndrome, a lysosomal storage disease (LSD) caused by the body’s inability to produce the IDUA enzyme. Data from a study p... 
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08/27/14Study Using NAV Vector Highlights Potential for Gene Therapy To Treat Diseases That Affect The CNS
Results support REGENX’s plans to advance gene therapies for MPS I and MPS II into human clinical trialsWASHINGTON, DC August 27, 2014 – REGENX Biosciences, LLC today announced that gene transfer mediated by REGENX’s NAV® AAV9 vector can be accomplished in vivo and globally correct central nervous system (CNS) manifestations of MPS I, a lysosomal storage disease caused by the body’s inability to produce the α-L-iduronidase (IDUA) enzyme.  Data from a study performed by researchers at the Pe... 
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06/02/14REGENX Biosciences and Voyager Therapeutics Announce License Agreement
Voyager acquires rights to REGENX’s proprietary NAV® vectors in multiple CNS disordersEighth third-party commercial license of REGENX’s NAV vectors since 2010REGENX to receive undisclosed upfront payment, milestones and royalties in exchange for non-exclusive worldwide licenseWASHINGTON, DC and CAMBRIDGE, Mass.  June 2, 2014 – REGENX Biosciences, LLC and Voyager Therapeutics today announced that they have entered into a license agreement for use of REGENX’s proprietary NAV® vectors for the devel... 
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04/29/14REGENX Biosciences enters into agreement with AAVLife 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 ... 
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04/01/14REGENX Biosciences and AveXis Enter into Exclusive Agreement for Development of Treatments for SMA
Washington, DC and Dallas, TX - REGENX Biosciences, LLC (REGENX) and AveXis, Inc. (AveXis) announce that they have entered into an exclusive agreement for the development and commercialization of products to treat Spinal Muscular Atrophy (SMA) using NAV rAAV9 vectors. Under the terms of the agreement, REGENX granted AveXis an exclusive, worldwide license, with rights to sublicense, to REGENX’s NAV rAAV9 vector for treatment of SMA disease in humans. In return for these rights, REGENX receives a... 
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02/28/14REGENX Biosciences and ESTEVE enter into license agreement for rare lysosomal storage disorder
WASHINGTON, DC and BARCELONA, SPAIN February 28, 2014 – REGENX Biosciences, LLC (REGENX) and Laboratorios Dr. Esteve, S.A. (ESTEVE) announce that they have entered into an agreement enabling the development and commercialization of products to treat mucopolysaccharidosis type IIIA (MPSIIIA or Sanfilippo syndrome Type A) using NAV rAAV9.Under the terms of the Agreement, REGENX granted ESTEVE a non-exclusive worldwide license, with rights to sublicense, to REGENX’s N... 
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