PTC Therapeutics and Odylia Therapeutics Partner to Develop Rare Retinal Disease Gene Therapies

Rare Daily Staff

PTC Therapeutics and Odylia Therapeutics are teaming up to develop novel gene therapies in rare inherited retinal diseases utilizing a vector system developed by researchers at Massachusetts Eye and Ear.

The collaboration enhances PTC’s expertise in central nervous system gene therapy applications with Odylia’s ophthalmic capabilities across a broad portfolio inherited rare disease targets. The lead program in the collaboration will be RP-GRIP1, a ciliary protein that is defective in Leber Congenital Amaurosis 6, 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, 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. Similarly to our CNS gene therapy programs, we will apply a targeted, micro-dosing gene therapy strategy to our inherited rare disease research and development.”

Odylia Therapeutics was established in 2017 as a nonprofit with support from Massachusetts Eye and Ear and the Usher 2020 Foundation to bring treatments for rare eye diseases from the lab into human clinical trials using AAV technology and other gene delivery techniques.

There are more than 250 genetic forms of inherited rare diseases that lead to blindness. Most are extremely rare, affecting only one in a million people worldwide. Today, there is preclinical proof-of-concept for treatments for more than 30 of these diseases, with more being discovered every day.

“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,” said Scott Dorfman, co-founder and CEO of Odylia and a member of Usher 2020 Foundation.

Photo: Scott Dorfman, co-founder and CEO of Odylia

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