GlobeNewswire·Mar 2, 2026, 7:02 AM ET

Ocugen Announces Phase 3 liMeliGhT Enrollment Completion for OCU400, a Novel Modifier Gene Therapy for Broad Retinitis Pigmentosa

Enrollment is complete for the liMeliGhT Phase 3 clinical trial, the largest gene therapy trial for broad retinitis pigmentosa (RP) patients, involving 140 randomized participants across RHO & gene-agnostic arms.
Topline Phase 3 data is expected in the first quarter of 2027 to support a Biologics License Application (BLA) filing & potential approval for OCU400 in 2027.
The European Medicines Agency (EMA) has accepted the U.S.-based trial for submission of a Marketing Authorization Application (MAA).
Ocugen plans to file a rolling BLA in the third quarter of 2026, with the trial designed as a one-year study assessing 12-month visual function changes via the proprietary LDNA test.
Positive 3-year Phase 1/2 data demonstrates durable safety & tolerability, with 88% of treated subjects showing visual improvement or preservation & approximately a 2-line gain in visual function.
OCU400 is a gene-agnostic modifier gene therapy targeting the NR2E3 nuclear hormone receptor to reset dysfunctional retinal gene networks across multiple RP mutations.
The therapy aims to address the 98% of RP patients currently ineligible for existing single-mutation gene replacement therapies.
Dr. Shankar Musunuri, Ocugen's CEO, stated the milestone brings the company closer to delivering a one-time treatment for thousands of RP patients globally.