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Collaborations & Alliances

PTC Therapeutics, Odylia Therapeutics Enter Gene Therapy Alliance

Expands PTC's gene therapy portfolio to rare ophthalmic diseases

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By: Kristin Brooks

Managing Editor, Contract Pharma

PTC Therapeutics, Inc. and Odylia Therapeutics entered a strategic collaboration to develop novel gene therapies in rare inherited retinal diseases (IRDs) utilizing the Anc80 vector system developed by researchers at Massachusetts Eye and Ear. The collaboration augments PTC’s expertise in localized gene therapy applications in the Central Nervous System (CNS) with the ophthalmic experience at Odylia across a broad portfolio of IRD targets. The lead program in the collaboration will be RP-GRIP1, a ciliary protein that is defective in Leber Congenital Amaurosis 6 (LCA6), a rare early onset childhood retinal dystrophy.

“We are excited to expand our gene therapy efforts to include targets in rare, inherited retinal diseases, for which there is tremendous unmet need,” said Mark Pykett, V.M.D., Ph.D., chief scientific officer of PTC Therapeutics. “This partnership combines PTC’s strength in developing and commercializing treatments for rare diseases with Odylia’s novel vector platform and breadth of expertise in rare ophthalmic diseases. As with our CNS gene therapy programs, we will apply a targeted, micro-dosing gene therapy strategy to our IRD research and development.”

Scott Dorfman, chief executive officer of Odylia Therapeutics, added, “PTC is the ideal partner with their expertise in developing and commercialization of rare disorder treatments but most importantly, their commitment to patients. We look forward to the clinical translation of the Anc80 AAV platform and the RP-GRIP1 Program, as well as future programs in the partnership.”

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