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Genethon and Sarepta Strengthen Duchenne Collaboration

Genethon And Sarepta Strengthen Duchenne Collaboration
Genethon And Sarepta Strengthen Duchenne Collaboration
Josh Argall
Community Manager

Genethon Announces Expanded Collaboration with Sarepta Therapeutics
The agreement paves the way for a clinical trial to begin in 2020.

Genethon, a French laboratory dedicated to the design and development of gene therapy products for rare diseases, and Sarepta Therapeutics announced an expansion of their collaboration to develop a gene therapy for Duchenne muscular dystrophy.

Preclinical studies of the microdystrophin gene therapy, currently referred to by Genethon as GNT0004, have demonstrated significant expression of microdystrophin protein levels and stabilization of symptoms, including restoration of muscle function in canines naturally affected by the disease.

The approach used for this particular gene therapy is the result of technology developed by Genethon researchers in collaboration with a team led by George Dickson, Professor of Molecular Cell Biology at Royal Holloway – University of London (RHUL).

Under the terms of the agreement, Genethon will be responsible for commercializing GNT0004 in Europe (excluding the United Kingdom). Sarepta will be responsible for commercialization in the United Kingdom and throughout the rest of the world.

According to the press release issued by Genethon, the agreement paves the way for a clinical trial to begin in 2020.