Ocugen Inc., based in Malvern, Pennsylvania, has received provisional approval from the Longevity and Regenerative Therapies Board (LARTA) in The Bahamas for its gene therapy candidate OCU400, designed to treat retinitis pigmentosa (RP). This approval follows prior designations from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), including Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designations. The therapy targets mutations in over 100 genes affecting photoreceptor development and survival in the retina, addressing both pediatric and adult patients across early and late-stage disease spectrums.
The provisional approval paves the way for an expanded access program, enabling Ocugen to initiate treatment for the first RP patient within 90 days of full regulatory clearance. Topline results from its Phase 3 clinical trial are anticipated in the first quarter of 2027, with plans to submit a Biologics License Application in the second quarter of 2027. Dr. Shankar Musunuri, Ocugen’s Chairman and CEO, emphasized the milestone as a step toward expanding access to its one-time, innovative treatments for serious retinal diseases.
Ocugen’s NR2E3-based approach leverages a nuclear hormone receptor to restore retinal function, offering a potential breakthrough for patients with RP—a degenerative condition affecting vision. The Bahamas’ provisional designation aligns with broader regulatory momentum, underscoring the therapy’s potential to redefine therapeutic options for retinal disorders globally.
