Ocugen, Inc. announced that dosing is complete in the second cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA)?a modifier gene therapy candidate being developed for Stargardt disease as a one-time treatment for life. Six patients with Stargardt disease have been dosed in the Phase 1/2 clinical trial to date. An additional three patients will be dosed with the high dose (Cohort 3) of OCU410ST in the dose-escalation phase.

A Data and Safety Monitoring Board meeting will convene next month to review the 4-week safety data of the medium dose cohort before proceeding with Cohort 3 (high dose), which is the final dose in the Phase 1 dose-escalation study. The GARDian clinical trial will assess the safety and efficacy 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 consisting of three dose levels [low dose (3.75×1010 vg/mL), medium dose (7.5×1010 vg/mL), and high dose (2.25×1011 vg/mL)].

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 group. Ocugen is committed to finding solutions for people with inherited retinal disease for whom no effective treatment options exist. While an orphan disease, Stargardt affects approximately 100,000 people in the United States and Europe combined.

The Company expects to provide a clinical trial update for OCU410ST in the third quarter of 2024. 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.