Date: 2017-06-21

Type of information: Research agreement

Compound: micro-dystrophin gene therapy

Company: Sarepta Therapeutics (USA - MA) Genethon (France)

Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases

Type agreement: R&D - research - development

Action mechanism: gene therapy. 

Disease: Duchenne muscular dystrophy (DMD)


  • • On June 21, 2017, Sarepta Therapeutics 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. 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.
  • 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 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.

Financial terms: Financial terms of the collaboration have not been disclosed.

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