Sarepta gets option for Genethon's DMD gene therapy

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said it gained an option to obtain exclusive U.S. rights from non-profit organization Genethon (Evry, France) to a gene therapy to treat Duchenne muscular dystrophy.

The collaboration covers a Genethon preclinical program that delivers a transgene encoding microdystrophin, a shortened but functional copy of dystrophin, via an adeno-associated virus serotype 2 (AAV2) vector. Genethon will be responsible for early development, and Sarepta may opt to co-develop the program. Sarepta spokesperson Ian Estepan declined to disclose financial terms...