Newly Published Data Validates Micro-Dystrophin Gene Therapy Approach in Animal Model for Duchenne

PPMD, at the start of the year, made its biggest investment ever, $2.2M to Jerry Mendell at Nationwide Children’s Hospital, for a Phase 1 Safety trial of gene therapy in children with Duchenne. Sarepta made an additional investment in this trial, leveraging PPMD's initial investment to make the results even more robust.

Last week, gene therapy made headlines as researchers in France announced that they had successfully introduced microdystrophin into a dog with muscular dystrophy via a systemic intravascular injection using a viral vector delivery and restored dystrophin production to achieve functional improvement in the dogs.

The effect lasted for over two years and is ongoing. This was a first.

For us, this proof that the technology works very well in dogs gives added evidence that gene therapy is ready to go into humans and supports our decision to invest heavily in gene therapy. While dogs and humans are not exactly the same, these results are so very encouraging.

We look forward to working with Dr. Mendell and his team at Nationwide and Sarepta, our co investors, to move this trial forward.

Read the Press Release from Sarepta Therapeutics: 

Sarepta Therapeutics Announces its Partner, Genethon, Published New Micro-Dystrophin Gene Therapy Data in Nature Communications
July 27, 2017

Data show for the first time a systemic therapeutic effect in DMD dogs using a rAAV2/8 micro-dystrophin gene therapy approach without immunosuppressive treatment

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.

About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, please visit

About Genethon
Created by the AFM-Telethon, the French Muscular Dystrophy Association (AFM), Genethon, located in Evry, France, is a non-profit R&D organization dedicated to the development of biotherapies for orphan genetic diseases, from the research to clinical validation. Genethon, is specialized in the discovery and development of gene therapy drugs and has multiple ongoing programs at clinical, preclinical and research stage for neuromuscular, blood, immune system, liver and eye diseases.

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