REGENXBIO Announces New License Agreement with Pfizer for the Treatment of Friedreich's Ataxia Using NAV® AAV9 Vector

ROCKVILLE, MD, USA I July 31, 2019 I REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced it entered into a license agreement with Pfizer Inc.

Under the terms of the agreement, REGENXBIO has granted Pfizer a non-exclusive worldwide license, with rights to sublicense, to REGENXBIO's NAV AAV9 vector for the development and commercialization of gene therapies for the treatment of Friedreich's ataxia, the most common hereditary ataxia. In return for these rights, REGENXBIO will receive an upfront payment, and has the potential to receive ongoing fees, development and commercial milestone payments, and royalties on net sales of products incorporating the licensed intellectual property.

"This license agreement further validates the strength of our intellectual property portfolio and the potential of NAV AAV9 for the treatment of systemic and CNS manifestations of movement disorders," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We are pleased to establish our relationship with Pfizer as they advance this program to develop a potential gene therapy treatment for Friedreich's ataxia."

"We are excited to partner with REGENXBIO on the use of the NAV AAV9 vector for the treatment of Friedreich's ataxia, a condition with significant unmet medical need," said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer's Rare Disease Research Unit. "We believe the AAV9 vector has the potential to have a profound impact on patients with severe and debilitating diseases where treatment options are limited today."

"Friedreich's ataxia is a debilitating, life-shortening, degenerative neuro-muscular disorder that affects about one in 50,000 people in the United States, and there are currently no treatments available," said Ronald J. Bartek, President, Director and Co-Founder of the Friedreich's Ataxia Research Alliance. "We applaud these efforts to develop new potential NAV Technology-based gene therapies for our under-served patient community."

About Friedreich's Ataxia

Friedreich's ataxia (FA) is the most common hereditary ataxia.  FA patients have a genetic mutation in the FXN gene, which limits the production of the protein frataxin, causing a variety of debilitating symptoms and complications including loss of coordination and balance, muscle weakness, impaired vision, hearing and speech, scoliosis, diabetes, and cardiomyopathy. 


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.


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