Pfizer Partners with REGENXBIO to Develop AAV9-Based Gene Therapy for Friedreich’s Ataxia

Today, REGENXBIO Inc. announced that it has entered into a research and licensing agreement with Pfizer Inc., granting Pfizer the right to use REGENXBIO’s proprietary NAV adeno-associated virus 9 (AAV9) vector to develop gene therapies for the treatment of Friedreich’s ataxia (FA), thereby improving patients’ quality of life.

Friedreich’s ataxia (FA) is the most common form of hereditary ataxia, affecting 1 in every 50,000 individuals. FA results from mutations in the FXN gene, which restrict the production of frataxin and lead to various debilitating symptoms and complications, including loss of coordination and balance, muscle weakness, visual impairment, hearing and speech disorders, scoliosis, diabetes, and cardiomyopathy.

REGENXBIO Inc. is a biotechnology company dedicated to unlocking the potential of gene therapy to improve patients’ lives. The company’s NAV Technology Platform is a proprietary adeno-associated virus (AAV) gene delivery platform comprising more than 100 novel AAV vectors, including AAV7, AAV8, AAV9, and AAVrh10. REGENXBIO and its partners are leveraging this platform to develop and investigate candidate therapies across multiple therapeutic areas. Notably, Zolgensma, approved by the U.S. Food and Drug Administration (FDA) this year for the treatment of spinal muscular atrophy (SMA), utilizes REGENXBIO’s AAV vector technology.

▲Illustration of gene therapy (Image source: REGENXBIO Inc. official website)

Under the license agreement, REGENXBIO grants Pfizer a non-exclusive global license to develop and commercialize gene therapies for the treatment of FA using the NAV AAV9 vector. REGENXBIO will receive an upfront payment, potential subsequent payments based on development and commercialization milestones, and royalties on sales of products incorporating its intellectual property.

Reference: [1] REGENXBIO Announces New License Agreement with Pfizer for the Treatment of Friedreich's Ataxia Using NAV^® AAV9 Vector. Retrieved July 31, 2019, from https://www.prnewswire.com/news-releases/regenxbio-announces-new-license-agreement-with-pfizer-for-the-treatment-of-friedreichs-ataxia-using-nav-aav9-vector-300893950.html

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