Sarepta, Genethon collaborate for Duchenne muscular dystrophy
Sarepta Therapeutics, Inc., a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Genethon, a non-profit R&D organization dedicated to the development of biotherapies for orphan genetic diseases from research to clinical validation, have signed a gene therapy research collaboration to jointly develop treatments for Duchenne muscular dystrophy (DMD). Genethon’s micro-dystrophin gene therapy approach can target the majority of patients with DMD. Genethon has demonstrated proof-of-concept of their micro-dystrophin program via robust gene expression in a large animal model of DMD.
Under the terms of the 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. Financial terms of the collaboration have not been disclosed.
Genethon has made significant investment in the development of gene therapies for neuromuscular diseases and employs one of the largest research and clinical groups in the world working to advance rare disease therapies. The Company’s European-based research laboratory has a long-term commitment to neuromuscular diseases with a central focus on DMD. In addition, Genethon is affiliated with Europe’s largest cGMP vector manufacturing facility, YposKesi, located in Evry (Essonne). YposKesi employs approximately 150 experts in bio-production at its current 54,000 square feet manufacturing facility, and plans significant future expansion 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,” stated 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.”
“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,” said Frederic Revah, chief executive officer of Genethon.