Ocugen Achieves Key Enrollment Target in Stargardt Disease Gene Therapy Trial

Ocugen finishes patient dosing for Phase 2/3 gene therapy trial (OCU410ST) for Stargardt disease early. Two late-stage ocular programs advance, with financial runway extended into 2027.

Biotechnology firm Ocugen, Inc. has completed patient dosing ahead of schedule in a critical late-stage clinical study. The company announced that the dosing phase for its pivotal Phase 2/3 trial, named GARDian3, is now finished. All 63 participants were enrolled and received treatment in under nine months. The trial is evaluating OCU410ST, a one-time gene therapy candidate for Stargardt disease, a hereditary retinal degeneration that currently has no FDA-approved treatment.

Dual Late-Stage Programs Advance

This milestone marks the second Ocugen late-phase program to move into its follow-up observation period. Recently, the company also completed enrollment for its liMeliGhT Phase 3 study, which is testing OCU400 for retinitis pigmentosa. The progress places two of the company’s ocular gene therapy assets in advanced clinical development simultaneously.

OCU410ST utilizes an AAV5 platform and is designed to deliver the hRORA gene via a single subretinal injection. Its mechanism represents a shift from classic gene replacement strategies. Rather than targeting individual genetic mutations, the therapy aims to address entire gene networks. This approach could potentially make it applicable to a broader subset of patients within the estimated 100,000 individuals affected in the U.S. and Europe, irrespective of their specific mutation in the ABCA4 gene.

Financial Runway Extended Through Early 2027

Alongside these clinical developments, Ocugen has bolstered its financial position. The company reported a $15 million capital infusion from the exercise of warrants by an institutional investor. Combined with previous financing activities, management believes its current liquidity is sufficient to fund operations into the first quarter of 2027.

Interim Readout Scheduled for 2026

An interim analysis for the GARDian3 study is planned for the third quarter of 2026. This evaluation will focus on data from 24 subjects who have been monitored for eight months post-treatment. To date, no serious adverse events have been reported. The primary efficacy endpoint of the trial is a reduction in atrophic lesions after twelve months.

If the data continues to show a positive trend, Ocugen intends to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration. The submission target, based on the one-year results, is set for mid-2027.