Genethon

Sarepta, Genethon collaborate for Duchenne muscular dystrophy

Wednesday, June 21, 2017

Sarepta Therapeutics, Inc., 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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