Sarepta Therapeutics and Genethon announce a gene therapy research collaboration for the treatment of Duchenne muscular dystrophy | DuchenneXchange

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Sarepta Therapeutics and Genethon announce a gene therapy research collaboration for the treatment of Duchenne muscular dystrophy


Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Genethon, a non-profit R&D organization dedicated to the development of biotherapies for orphan genetic diseases from research to clinical validation, have signed a gene therapy research collaboration to jointly develop treatments for Duchenne muscular dystrophy (DMD). Genethon’s micro-dystrophin gene therapy approach can target the majority of patients with DMD. Genethon has demonstrated proof-of-concept of their micro-dystrophin program via robust gene expression in a large animal model of DMD.

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