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REGENXBIO Announces First FDA Approval of a Gene Therapy Based on Its Proprietary NAV® Technology Platform
ROCKVILLE, Md., May 24, 2019 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through
About this update from Regenxbio Inc.
[{"type":"text","content":"ROCKVILLE, Md., May 24, 2019 /PRNewswire/ -- 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 the first U.S. Food and Drug Administration (FDA) approval of a gene therapy that leverages REGENXBIO's proprietary NAV Technology Platform, Novartis AG's ZOLGENSMA® (onasemnogene abeparvovec-xioi; AVXS-101). ZOLGENSMA was approved by the FDA as a one-time infusion for pediatric patients with spinal muscular atrophy (SMA) who are less than two years of age.\n\"Today's FDA approval of ZOLGENSMA marks a major milestone for NAV Technology, gene therapy and patients and families facing SMA, a debilitating and potentially deadly disease,\" said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. \"We celebrate this important milestone and the benefit that ZOLGENSMA can bring to patients suffering from SMA and their families with the SMA community. ZOLGENSMA is truly an innovative therapy that is designed to correct an underlying genetic defect with one-time treatment by providing a functional copy of the SMN1 gene.\" \nIn 2009, REGENXBIO secured exclusive rights to key intellectual property covering novel recombinant adeno-associated viral vectors discovered at the University of Pennsylvania in the lab of James Wilson, M.D., Ph.D. AveXis, Inc., a Novartis company, licensed REGENXBIO's NAV adeno-associated virus 9 (AAV9) vector in 2014 and entered into an expanded license for rights to the entire NAV Technology Platform for the treatment of SMA in 2018. \nMr. Mills added: \"ZOLGENSMA is based on our novel NAV Technology Platform, which was created and validated by REGENXBIO's founders and collaborators at the University of Pennsylvania led by Dr. Jim Wilson. Its approval demonstrates the transformational impact that NAV Technology can have on the treatment of genetic diseases with significant unmet needs. We are inspired by the innovative work of the founders at AveXis, led by Dr. Brian Kaspar, to create and develop ZOLGENSMA. ZOLGENSMA is the first of many NAV Technology gene therapy product candidates in our research and clinical development pipeline with the potential to treat a large range of genetic and chronic diseases. We are excited to see our NAV Tech...