Sarepta and Genethon Will Collaborate to Develop DMD Gene Therapy

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |


Sarepta Therpeutics and Genethon have signed a research collaboration agreement to jointly develop a gene therapy for Duchenne muscular dystrophy (DMD).

Created by the AFM-Telethon, the French Muscular Dystrophy Association, Genethon is a nonprofit organization that specializes in the discovery and development of drugs for orphan genetic diseases using its microdystrophin gene therapy approach. This approach reportedly has been shown to target most patients with DMD.

“Microdystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients. 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,” Frederic Revah, CEO of Genthon, said in a press release.

Genethon has invested significantly in the development of gene therapies for neuromuscular diseases and employs one of the world’s largest research and clinical enterprises working to advance therapies for rare diseases, including DMD.

Genethon also is affiliated with YposKesi, Europe’s largest Current Good Manufacturing Practices (CGMP) vector manufacturing facility. YposKesi employs approximately 150 experts in bio-production and is preparing to make future adjustments to meet the growing demand of gene therapy products.

“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 CEO. “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,” Kaye said.

Per the terms of the agreement, Genethon will be responsible for early development work and Sarepta will have the option to co-develop Genethon’s microdystrophin program, which includes exclusive U.S. commercial rights. Financial terms of the agreement were not disclosed.

This collaboration, as well as YposKesi’s plans to invest in gene therapy products, might mean that more gene therapies will be available soon in the European Union, not only for DMD but also for other rare genetic diseases.