Ocugen reported that dosing has been completed ahead of schedule in its phase 2/3 GARDian3 trial evaluating OCU410ST for Stargardt disease. OCU410ST is a modifier gene therapy designed to deliver the RORA gene using an AAV5 vector, with the aim of addressing multiple disease pathways independent of ABCA4 genotype.

The randomized, masked, multicenter study enrolled 63 patients across a range of ages and disease stages. Participants in the treatment arm received a single subretinal injection in the eye with worse visual acuity, while controls remained untreated. The primary endpoint is reduction in atrophic lesion size at 12 months, with secondary endpoints including best-corrected visual acuity and low-luminance visual acuity. Preservation of the ellipsoid zone is being assessed as an early structural marker of treatment effect.

Company officials said interim data from the trial are expected in the third quarter of 2026, with 1-year results intended to support a planned biologics license application in 2027. To date, no serious adverse events or adverse events of special interest have been reported. Earlier phase 1 data suggested slower lesion growth and stabilization or improvement in visual acuity in treated eyes compared with untreated fellow eyes, although these findings require confirmation in the ongoing pivotal study.

OCU410ST is a disease-specific extension of the company’s OCU410 modifier gene therapy platform, which is currently being studied for geographic atrophy secondary to dry age-related macular degeneration. Ocugen recently reported positive 12-month results from its phase 2 ArMaDa trial in GA, and announced plans to launch a phase 3 registrational trial later in 2026. RP