MALVERN, Pa. — September 25, 2026
Ocugen Inc. announced that its investigational modifier gene therapy OCU400 has received provisional approval and priority designation from the Longevity and Regenerative Therapies Board (LARTA Board) in The Bahamas for the treatment of retinitis pigmentosa (RP). Under the planned pathway, Ocugen intends to provide OCU400 through an expanded access program (EAP), with the company targeting treatment of the first RP patient within 90 days following full LARTA approval. The company said the collaboration is intended to expand access to an investigational one-time gene therapy for patients with significant unmet medical needs while OCU400 continues through its global clinical development program.
LARTA Designation Supports Expedited Regulatory Engagement
The LARTA Priority Designation recognizes the scientific and clinical potential of OCU400 and its potential to address significant unmet medical need, according to Ocugen. When combined with provisional approval, the designation provides a structured pathway involving enhanced regulatory engagement and expedited coordination toward full approval and operational readiness. Ocugen plans to work with the LARTA Board on the expanded access framework and patient treatment process. The company also said its approach is informed by cost-effectiveness modeling for a one-time broad treatment of retinitis pigmentosa, supporting its stated objective of addressing access challenges associated with serious retinal diseases.
OCU400 Uses Modifier Gene Therapy Approach for RP
OCU400 is an investigational modifier gene therapy designed to address the underlying biology of retinitis pigmentosa rather than a single disease-causing mutation. The candidate is based on NR2E3, a nuclear hormone receptor involved in retinal functions including photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival. Ocugen says OCU400 is designed to reset dysfunctional gene networks in retinal cells and restore cellular homeostasis. The company is developing the candidate for a broad RP population spanning early- to late-stage disease and pediatric and adult patients, with a potential reach across mutations associated with more than 100 genes. OCU400 has also received FDA Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations, as well as Orphan Medicinal Product designation from the EMA.
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Phase 3 Development Continues Toward 2027 Regulatory Filing
OCU400 is currently being evaluated in Phase 3 clinical development, with Ocugen expecting topline data in the first quarter of 2027 and planning a Biologics License Application (BLA) submission in the second quarter of 2027. The Bahamas access program is therefore progressing in parallel with the candidate’s pivotal development and planned U.S. regulatory pathway. The company is positioning OCU400 as part of its broader modifier gene therapy platform for inherited retinal diseases and other causes of blindness, including Stargardt disease and geographic atrophy. If full LARTA approval is obtained, Ocugen intends to use the expanded access program to begin providing OCU400 to eligible RP patients in The Bahamas while the therapy remains investigational and its Phase 3 and regulatory activities continue.
Source :Ocugen press release
