Ocugen Announces Publication of Phase 1 GARDian1 Trial Results for OCU410ST Modifier Gene Therapy

Core Insights - Ocugen, Inc. announced positive Phase 1 GARDian1 trial results for OCU410ST, a novel modifier gene therapy for Stargardt disease, published in the peer-reviewed journal Nature Eye [1][2] Group 1: Clinical Trial Results - The Phase 1 GARDian1 trial demonstrated robust efficacy and safety outcomes, indicating a favorable safety and tolerability profile for OCU410ST [3][4] - Among six patients with gradable Fundus Auto Fluorescence images, atrophic lesion growth was reduced by 54% in treated eyes compared to untreated eyes over 12 months [5] - Treated eyes showed a 50% slower lesion expansion rate compared to untreated eyes, below published natural history rates [5] - Visual acuity improved in treated eyes, with a gain of +6 letters in Best Corrected Visual Acuity (BCVA) compared to a decline in untreated eyes [5] - No drug-related serious adverse events were reported during the trial [5] Group 2: Disease Background - Stargardt disease is the most common form of inherited macular degeneration, affecting over 100,000 individuals in the U.S. and Europe, characterized by progressive central vision loss [3][7] - Currently, there are no FDA-approved treatments for Stargardt disease, highlighting a critical unmet medical need [3][7] Group 3: Future Developments - The ongoing Phase 2/3 GARDian3 trial is progressing ahead of schedule, with anticipated enrollment completion in the first quarter of 2026 [4] - The company plans to file a Biologics License Application (BLA) in the first half of 2027, aiming for three regulatory submissions within three years [4] Group 4: Technology Overview - OCU410ST utilizes an AAV5 delivery platform to deliver the RORA gene to the retina, addressing multiple pathophysiological pathways linked to Stargardt disease [6] - The therapy aims to provide a gene-agnostic modification strategy that could benefit patients regardless of their underlying ABCA4 mutation [4][6] Group 5: Company Overview - Ocugen, Inc. focuses on discovering and developing novel gene therapies for blindness diseases, aiming to address significant unmet medical needs through innovative approaches [8]