Sarepta Therapeutics and Genethon Announce DMD Treatment Research Collaboration

Sarepta Therapeutics has announced a new collaboration with Genethon for gene therapy research in efforts to develop new Duchenne muscular dystrophy (DMD) treatments.

Genethon, a nonprofit organization working toward developing biotherapies for orphan genetic diseases, has a micro-dystrophin gene therapy approach that can target DMD patients. Genethon’s research laboratory has also been focused on producing gene therapies for neuromuscular diseases, including DMD.

There is currently no cure for DMD, which is a fatal muscle disease that causes muscle degeneration and mostly affects boys. The disease has an average life expectancy of just 26 years. Last year, the FDA approved the first DMD treatment, Exondys 51 developed by Sarepta, which many insurers are declining to cover. Another drug approved more recently, deflazacort, has a price tag of $35,000 a year, but families had been importing it from abroad for years for less than $2000 a year.

“Our agreement with Genethon strengthens our ongoing commitment to patients and is aligned with our strategy of building the industry’s most comprehensive franchise in DMD,” Edward Kaye, Sarepta’s chief executive officer, said in a statement. “This partnership brings together our collective experience in Duchenne drug development and Genethon’s particular expertise in gene therapy for rare diseases. We look forward to working with Genethon given their knowledge, large infrastructure and state-of the-art manufacturing capabilities to advance next generation therapies for DMD.”

The agreement places Genethon responsible for the beginning development efforts, while Sarepta will have the option to co-develop the micro-dystrophin program, including exclusive U.S. commercial rights. Genethon’s micro-dystrophin program has demonstrated its potential through the gene expression in an animal model of DMD.

“Microdystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients,” Frederic Revah, chief executive officer of Genethon, said in a statement. “In order to accelerate the development of a treatment, we are very pleased to partner with Sarepta Therapeutics, which has demonstrated commitment and success for innovative therapies for Duchenne muscular dystrophy.”