Ocugen, Inc. announced that dosing is complete in the first cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA)?a modifier gene therapy candidate being developed for Stargardt disease. Stargardt disease is an orphan blindness disease that affects approximately 35,000 people in the United States alone. Up to 10 leading retinal surgery centers across the United States are participating in the GARDian clinical trial.

In the first cohort, 3 subjects received 200L single subretinal administration of the low dose (3.75x1010 vg/mL) of OCU410ST. The GARDian clinical 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 consisting of three dose levels [low dose (3.75×10E10 vg/mL), medium dose (7.5×10E10 vg/mL), and high dose (2.25×10E11 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. The Company will continue to provide clinical updates.