OCU410ST is an investigational AAV5-hRORA modifier gene therapy candidate for Stargardt disease. It is being studied in a pivotal multicenter randomized Phase 2/3 program and earlier phase data suggest acceptable tolerability and visual function stabilization signals. The program is designed for potential BLA pathway progression rather than current approved use.
Ocugen has completed enrollment and dosing in its phase II/III GARDian3 study for OCU410ST gene therapy targeting Stargardt disease. The study includes 63 patients and aims to address over 1,200 ABCA4 gene mutations with a single treatment. Interim data is expected in Q3 2026, with a BLA submission targeted for mid-2027.
Ocugen reported Phase 2 data for its geographic atrophy gene therapy OCU410 showing 31% lesion growth reduction versus control. The company plans Phase 3 initiation in Q3 2026 and maintains its goal of three BLA filings in three years. Shares fell 11.48% following the data release which trailed earlier interim results.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT05956626 |
A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease |
ACTIVE_NOT_RECRUITING | PHASE2/PHASE3 |
