Ocugen Reports Positive Phase 1 Results for AAV Gene Therapy OCU410ST in Stargardt Disease

MALVERN, Pa., Jan. 12, 2026 (GLOBE NEWSWIRE) — Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on gene therapies for blinding retinal diseases, announced the publication of positive Phase 1 clinical data for OCU410ST, an AAV-based modifier gene therapy, in the peer-reviewed journal Eye, published by the Royal College of Ophthalmologists.

The publication reports 12-month safety, tolerability, and exploratory efficacy results from the first-in-human Phase 1 GARDian1 trial evaluating OCU410ST, an adeno-associated virus (AAV) gene therapy, in patients with early to advanced Stargardt disease. OCU410ST uses an AAV5 vector to deliver the RORA gene to retinal cells, representing a mutation-agnostic AAV gene therapy approach for inherited retinal degeneration.

Key findings from the AAV gene therapy trial include a 54% reduction in atrophic lesion growth in AAV-treated eyes compared with untreated fellow eyes, as measured by fundus autofluorescence over 12 months. Lesion expansion in AAV-treated retinas was approximately 50% slower than untreated eyes and below published natural history rates. Among best-corrected visual acuity (BCVA)–evaluable patients, AAV gene therapy–treated eyes demonstrated an average gain in visual acuity, while untreated eyes declined.

Importantly, the AAV gene therapy demonstrated a favorable safety profile, with no drug-related serious adverse events or adverse events of special interest observed during the study period. All treated eyes either stabilized or improved visual acuity following AAV administration, supporting the potential of AAV-based modifier gene therapy in Stargardt disease.

OCU410ST is designed to address multiple disease-driving pathways through AAV-mediated gene delivery, including lipofuscin accumulation, oxidative stress, inflammation, complement activation, and photoreceptor degeneration. Unlike traditional gene replacement strategies, this AAV modifier gene therapy is not constrained by ABCA4 mutation subtype, enabling broader applicability across the Stargardt disease population.

Stargardt disease is the most common inherited macular degeneration, affecting more than 100,000 people across the United States and Europe, with no approved therapies currently available. The positive Phase 1 results support continued clinical development of AAV-based gene therapy as a disease-modifying approach for this unmet medical need.

Ocugen’s AAV gene therapy program is progressing into the Phase 2/3 GARDian3 trial, with enrollment expected to complete in the first quarter of 2026. The company remains on track for a potential Biologics License Application (BLA) submission in the first half of 2027, positioning AAV gene therapy as a cornerstone of its retinal disease pipeline.