Ocugen Completes Dosing In Phase 2/3 GARDian3 Trial For Stargardt Gene Therapy, Moves Toward BLA Filing After Successful Trial Progress

Ocugen, Inc. announced that dosing has been completed ahead of schedule in its Phase 2/3 GARDian3 pivotal trial evaluating OCU410ST, a gene therapy candidate for Stargardt disease. The study is designed to assess the efficacy and safety of the therapy across patients with ABCA4-associated retinal disorders.

“This enrollment milestone for a pivotal trial underscores the tremendous progress our team is making toward bringing a transformative therapy to people living with multiple ABCA4-related gene mutations including Stargardt disease,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-founder of Ocugen. “The efficient and accelerated execution of this trial reflects the strong engagement of investigators and patients. It reinforces our confidence in OCU410ST as a potential one-time treatment option for all Stargardt patients who are desperately seeking rescue from blindness with no approved therapies to date.”

OCU410ST is a one-time subretinal injection using an AAV5-based platform to deliver the hRORA gene, aiming to slow or halt retinal degeneration and preserve vision. The trial enrolled 63 patients, with treated participants receiving the therapy in the eye with poorer vision, while the control group remained untreated.

"I am encouraged by the enthusiastic response and rapid enrollment in the GARDian3 registrational clinical trial for Stargardt disease, a devastating pediatric-onset retinal disorder affecting approximately 100,000 patients in the U.S. and Europe," said Dr. Huma Qamar, Chief Medical Officer of Ocugen. “Our trial encompasses pediatric to adult, and early to advanced stage subjects to address critical unmet medical need.”

The primary endpoint focuses on reducing atrophic lesion size at 12 months, while secondary endpoints include improvements in visual acuity and low-light vision. Additional observational measures, such as preservation of the ellipsoid zone, are expected to provide early indicators of therapeutic benefit. Interim results are anticipated in the third quarter of 2026.

“As a treating retina specialist, I see how the natural history of Stargardt disease leads to relentless enlargement of atrophic lesions and gradual loss of central visual acuity, often at a young age," said Christine Kay, MD, Vitreo Retinal Associates, Florida and a principal investigator in the GARDian3 trial. "The opportunity to intervene at an early stage of disease with a one-time subretinal gene therapy like OCU410ST that can potentially slow lesion growth, preserve visual function over time, and save vision before irreversible damage represents an exciting and much-needed shift from watching patients decline to proactively altering the course of their disease."

The therapy has shown a favourable safety and tolerability profile so far, with no serious adverse events reported. Ocugen plans to use one-year data from the study to support a Biologics License Application (BLA), targeting a mid-2027 submission as part of its broader pipeline strategy.