Sarepta Therapeutics to Partner with Genethon on Gene Therapy for Duchenne

Biopharmaceutical company Sarepta Therapeutics and non-profit rare disease R&D company Genethon have decided to partner to develop novel therapies for Duchenne muscular dystrophy (DMD). Genethon has developed and demonstrated proof-of-concept for their micro-dystrophin gene therapy approach in an animal model of DMD.

While Genethon will be responsible for early R&D for the micro-dystrophin program, the deal will give Sarepta the option to act as co-developer and take advantage of exclusive commercial rights for the US market. The companies did not disclose the financials included in the collaboration agreement.

“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,” said Edward Kaye, Sarepta’s chief executive officer. “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.”

Last year, Sarepta’s Exondys 51 became the first FDA-approved treatment for DMD, a rare genetic disease that causes progressive muscle deterioration and weakness. While the approval was supported by members of the DMD patient community, it sparked controversy in the industry with then-FDA commissioner Dr. Robert Califf claiming that the published results of a study on Exondys 51 were misleading.

“Micro-dystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients,” said Frederic Revah, chief executive officer of Genethon. “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. This partnership brings together the highly complementary and synergistic expertises of Sarepta and Genethon, to the benefit of the patients.”

Originally developed by Marathon Pharmaceuticals, PTC Therapeutics’ Emflaza has also been approved by the FDA to treat DMD. Sarepta previously inked a partnership with biopharmaceutical company Catabasis Pharmaceuticals to develop combination therapies for DMD.