Ocugen, Inc. announced today that the first patient has been dosed in its Phase 2/3 GARDian3 clinical trial for OCU410ST (AAV5-hRORA). This modifier gene therapy candidate is being developed for all Stargardt disease (ABCA4-associated retinopathies). Dosing the first patient is an especially significant milestone, bringing the company closer to addressing the unmet medical need for Stargardt patients.
The Phase 2/3 clinical trial builds upon encouraging results from the Phase 1 GARDian trial, which demonstrated 48% slower lesion growth at 12-month follow-up in evaluable treated eyes compared to untreated eyes. Additionally, evaluable treated eyes showed a statistically significant (p=0.031) and clinically meaningful improvement of nearly a 2-line gain in best corrected visual acuity (BCVA) at 12-month follow-up. OCU410ST has maintained a favorable safety and tolerability profile with no serious adverse events.
The study will enroll 51 participants, with 34 receiving a one-time subretinal injection of OCU410ST and 17 assigned to an untreated control group. The primary objective is to evaluate the reduction in atrophic lesion size, with one-year follow-up data supporting the planned Biologics License Application (BLA). This pivotal trial represents Ocugen’s second late-stage clinical program, with a BLA submission targeted for 2027.
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