Ocugen, Inc.

Ocugen is a biotechnology company focused on developing gene therapies to treat vision-related medical needs. The company's primary technology is its Novel Modifier Gene Therapy Platform, which utilizes nuclear hormone receptors (NHRs). This platform aims to address major blindness diseases, including rare genetic diseases like Retinitis Pigmentosa (RP), using a gene-agnostic approach. A key product in their pipeline is OCU400, designed for early to advanced cases of RP, encompassing both syndromic and non-syndromic forms of the disease, regardless of clinical or genetic diagnosis. In January 2025, Ocugen announced positive two-year data for multiple mutations from the Phase 1/2 clinical trial for OCU400. In February 2025, the European Commission (EC) provided a positive opinion from the European Medicines Agency's (EMA) Committee for Advanced Therapies for OCU400 Advanced Therapy Medicinal Product (ATMP) classification. The company has completed enrollment in the Phase 3 liMeliGhT clinical trial for OCU400. Long-term, 3-year Phase 1/2 data for OCU400 demonstrated sustained clinically meaningful, approximately 2-line luminance visual acuity (LLVA) gain, reinforcing durable gene-agnostic benefits. Visual function benefits were consistently observed over 3 years, with 88% (7/8) of evaluable treated subjects showing improvement or preservation versus untreated fellow eyes. An approximately 2-line gain (N=8) was observed across multiple mutation types in treated eyes compared to untreated eyes at 3 years. OCU400 maintained a favorable durability, safety, and tolerability profile with no new treatment-related serious adverse events or adverse events of interest. Ocugen is on track to begin a rolling Biologics License Application (BLA) submission in the third quarter of 2026.