CAMBRIDGE, Mass., July 27, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced the publication of data validating Genethon’s micro-dystrophin gene therapy approach in an animal model for Duchenne muscular dystrophy (DMD). The results were featured in the July 25, 2017 online issue of Nature Communications. Sarepta announced in June 2017 that it entered into an exclusive gene therapy research collaboration with Genethon to jointly develop treatments for DMD.
“While early, these data highlight the potential for Genethon’s micro-dystrophin gene therapy program and once again underscore the significance of dystrophin production in the treatment of DMD,” said Douglas Ingram, Sarepta’s president and chief executive officer. “As the leader in the research and development of new treatments for DMD, we are taking a multi-front approach to advancing therapies for those afflicted with this debilitating disease.”
The study was conducted in 12 dogs naturally affected by DMD and treated with intravenous micro-dystrophin, a shortened version of the dystrophin protein combined with an AAV-type viral vector. At two-year follow-up, muscle function was significantly restored and clinical symptoms had stabilized. Additionally, researchers noted that dystrophin expression had returned to a high level in the high-dose group. No immunosuppressive treatment was administered beforehand, and no side-effects were observed.
Under the terms of the previously announced collaboration, Genethon will be responsible for the early development work. Sarepta has the option to co-develop Genethon’s micro-dystrophin program, which includes exclusive U.S. commercial rights. For more information, and to see a video illustrating the results of the aforementioned study, please click here.
Le Guiner, C. et al. Long-term micro-dystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. Nature Communications. 2017: Accepted Article, ahead of print. DOI: 10.1038/ncomms161.