Date: 2017-01-10
Type of information: Licensing agreement
Compound: Galgt2 gene therapy program
Company: Sarepta Therapeutics (USA - MA) Nationwide Children’s Hospital (USA - OH)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Type agreement: R&D licensing
Action mechanism: gene therapy
Disease: Duchenne muscular dystrophy
Details: * On January 10, 2017, Sarepta Therapeutics announced it has entered an exclusive license agreement with Nationwide Children’s Hospital, for their Galgt2 gene therapy program developed by researcher Dr. Paul Martin, Ph.D. The experimental program explores the potential surrogate gene therapy approach to Duchenne muscular dystrophy. As a “surrogate gene therapy approach”, the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin, with the goal of producing a muscle cell that can function normally even when dystrophin is absent. It has the potential to be used broadly in several muscular dystrophies.
“We are pleased to have this opportunity to help advance our Galgt2 gene therapy program,” said Dr. Kevin Flanigan, the Principal Investigator leading the clinical trial. “Our goal is to have this program in the clinic during 2017 and begin to evaluate a therapy that has the potential to treat patients of all ages and disease severity.”
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