First Patient Dosed in Ocugen's Phase 1/2 Trial Evaluating Safety and Efficacy of OCU41OST Modifier Gene Therapy for Stargardt Disease

Ocugen announced that the first patient has been dosed in its phase 1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease.

“There is a significant unmet medical need for the approximate 35,000 patients in the US living with Stargardt disease,” Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen, said in a company news release. “It is critical to our mission to develop innovative treatments for inherited retinal diseases and this milestone is an important step in bringing our novel modifier gene therapies to people who desperately need them.”

This phase 1/2 trial will assess the safety of unilateral subretinal administration of OCU410ST in subjects with Stargardt Disease and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose ranging study. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to an untreated control group.

OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks.