Sarepta Therapeutics and Genethon Announce Gene Therapy Research Collaboration for Duchenne Muscular Disease (DMD) Therapies

Sarepta Therapeutics, 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, 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 via robust gene expression with their micro-dystrophin program in a large animal model of DMD.