Ocugen, Inc. Announces First Patient Dosed in Phase 2/3 GARDian3 Pivotal Confirmatory Trial for OCU410ST—Novel Modifier Gene Therapy Candidate for Stargardt Disease

Core Viewpoint - Ocugen, Inc. has initiated dosing for the first patient in its Phase 2/3 GARDian3 clinical trial for OCU410ST, a gene therapy aimed at treating Stargardt disease, marking a significant milestone in addressing the unmet medical needs of patients suffering from this condition [1][2]. Company Overview - Ocugen, Inc. is a biotechnology company focused on developing gene therapies for blindness diseases, aiming to provide innovative solutions for patients globally [9]. Clinical Trial Details - The Phase 2/3 GARDian3 trial will enroll 51 participants diagnosed with Stargardt disease, with 34 receiving a one-time subretinal injection of OCU410ST and 17 in a control group [4]. - The primary objective of the trial is to evaluate the reduction in atrophic lesion size, with key secondary endpoints including improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA) [4]. Previous Trial Results - The Phase 1 GARDian trial showed promising results, with a 48% slower lesion growth at 12-month follow-up in treated eyes compared to untreated eyes, and a statistically significant improvement in BCVA [2][3]. Future Plans - Ocugen plans to submit a Biologics License Application (BLA) for OCU410ST in 2027, as part of its strategy to file three BLAs over the next three years [5]. Disease Background - Stargardt disease is the most common form of inherited macular degeneration, leading to progressive vision loss due to the degeneration of photoreceptor cells in the retina [7][8].