Ocugen, Inc. announced that the Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial recently convened and approved to proceed with dosing the high dose of OCU410ST in the dose-escalation phase of the study. OCU410ST (AAV5-hRORA) is a modifier gene therapy candidate being developed for Stargardt disease. Stargardt disease affects approximately 100,000 people in the U.S. and Europe combined.

Six patients with Stargardt disease have been dosed in the Phase 1/2 clinical trial to date in the low dose cohort and medium dose cohort. An additional three patients will be dosed with the high dose in cohort 3. The Phase 1/2 GARDian clinical trial will include up to 42 subjects?30 adults and 12 children with Stargardt disease?who exhibit mild to moderate disease symptoms and will assess the safety of unilateral subretinal administration of OCU410ST. The clinical trial is being conducted in two phases.

Phase 1 is a multicenter, open-label, dose-ranging/dose-escalation 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 enrolled in a 1:1:1 ratio to randomize subjects into two different treatment groups at varying dose levels, or a control (untreated group), allowing for a comprehensive assessment of the treatment's efficacy across different dosages. Currently, patients with Stargardt disease have no FDA-approved therapeutic options.

Ocugen is dedicated to providing a gene-agnostic treatment approach for patients living with inherited retinal diseases and is encouraged that the Phase 1/2 GARDian trial for OCU410ST remains on track.