OCU400 is an investigational AAV5-hNR2E3 modifier gene therapy for retinitis pigmentosa. The program is being studied in a Phase 3 multicenter randomized trial in mutation-defined and broader RP populations. Development updates describe it as a potential one-time subretinal treatment with planned regulatory filings rather than an approved product.
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 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 |
