Collaboration expands PTC’s Gene Therapy portfolio to rare ophthalmic diseases
SOUTH PLAINFIELD, N.J. and BOSTON, July 1, 2019 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) and Odylia Therapeutics today announced 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 existing expertise in localized gene therapy applications in the Central Nervous System (CNS) with the deep 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.” About PTC Therapeutics, Inc. About Odylia Therapeutics, Inc. For More Information: PTC Therapeutics, Inc. Media: Odylia Therapeutics, Inc. Massachusetts Eye and Ear Forward-Looking Statements: PTC’s actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including the factors discussed in the “Risk Factors” section of PTC’s Annual Report on Form 10-K for the year ended December 31, 2018 as well as any updates to these risk factors filed from time to time in PTC’s other filings with the SEC. You are urged to carefully consider all such factors. The forward-looking statements contained herein represent PTC’s views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.
SOURCE PTC Therapeutics, Inc. | ||
Company Codes: NASDAQ-NMS:PTCT |