Ocugen is a biotechnology company focused on discovering, developing, and commercializing gene and cell therapies as well as vaccines. Its programs target major unmet needs, especially in eye diseases.
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.
Ocular Therapeutix's experimental drug Axpaxli showed superior vision maintenance compared to Regeneron's Eylea in a late-stage wet AMD trial. Meanwhile, Ocugen reported 12-month data showing its gene therapy OCU410 reduced geographic atrophy lesions by 31%, though less than earlier interim results. Both companies are advancing toward regulatory submissions and further clinical development.
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.
Ocugen will host a conference call on March 4, 2026 at 8:30 a.m. ET to discuss fourth quarter and full year 2025 financial results and provide a business update, with a pre-market earnings announcement the same day.
Ocugen has completed enrollment of 140 patients in its Phase 3 liMeliGhT trial for OCU400, a gene therapy for retinitis pigmentosa. Topline data expected in Q1 2027 could support potential approval later that year.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06574997 |
Expanded Access Program for RP in Adults |
NO_LONGER_AVAILABLE | |
| NCT06388200 |
A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT06018558 |
Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy |
ACTIVE_NOT_RECRUITING | PHASE1/PHASE2 |
| NCT05956626 |
A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease |
ACTIVE_NOT_RECRUITING | PHASE2/PHASE3 |
| NCT05802329 |
Phase I Study to Assess the Safety and Efficacy of OCU200 for Center-Involved Diabetic Macular Edema (DME) |
RECRUITING | PHASE1 |
| NCT05541289 |
Expanded Access Program of Whole, Inactivated COVID-19 Vaccine COVAXIN™ (BBV152) in Adults Aged 18 Years and Older |
NO_LONGER_AVAILABLE | |
| NCT05258669 |
Immuno-bridging and Broadening Study of a Whole, Inactivated COVID-19 Vaccine BBV152 in Healthy Adults |
UNKNOWN | PHASE2/PHASE3 |
| NCT05203939 |
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis |
ACTIVE_NOT_RECRUITING | PHASE1/PHASE2 |
| NCT03785340 |
Study of Brimonidine Tartrate Nanoemulsion Eye Drop Solution in the Treatment of Dry Eye Disease (DED) |
COMPLETED | PHASE3 |
| NCT03591874 |
Study of Brimonidine Tartrate Nanoemulsion Eye Drops in Patients With Ocular Graft-vs-Host Disease |
TERMINATED | PHASE3 |
