Sarepta signs another Duchenne gene therapy pact as it aims for wider treatment quoting : FierceBiotech

Sarepta Therapeutics has penned its second DMD gene therapy pact this year as it announces a tie-up with France's Genethon, a nonprofit R&D org. It also penned an exclusive license agreement with Nationwide for their Galgt2 gene therapy program, originally developed by researcher Paul Martin. This early-stage program aims to research a potential surrogate gene therapy approach to DMD, whereby the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin. "This partnership brings together our collective experience in Duchenne drug development and Genethon's particular expertise in gene therapy for rare diseases. An initial phase 1/2a trial for the microdystrophin gene therapy is slated to begin at the end of the year and will be done at Nationwide Children's.




Source: Sarepta Therapeutics, Inc.Media and Investors:Sarepta Therapeutics, Inc.Ian Estepan, 617-274-4052iestepan@sarepta.comorW2O GroupBrian Reid, 212-257-6725breid@w2ogroup.comGenethonStéphanie BARDON - +331. Genethon's micro-dystrophin gene therapy approach can target the majority of patients with DMD. "Microdystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients. "This partnership brings together our collective experience in Duchenne drug development and Genethon's particular expertise in gene therapy for rare diseases. 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.

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