MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has secured provisional approval and priority designation in The Bahamas for OCU400, creating a pathway to provide its investigational gene therapy to retinitis pigmentosa patients through an expanded access program while the treatment remains in Phase 3 development.
The Longevity and Regenerative Therapies Board granted the designations, which place OCU400 on a regulatory pathway toward full approval and patient access in The Bahamas. Ocugen plans to supply the therapy through an expanded access program and is targeting treatment of the first patient within 90 days of full approval.
The arrangement gives Ocugen another potential route to patients as it advances OCU400 toward a planned U.S. regulatory filing. The company expects topline Phase 3 data in the first quarter of 2027 and plans to submit a Biologics License Application in the second quarter.
Ocugen intends to work with the LARTA Board on access and commercial pricing for OCU400, including an approach based on cost-effectiveness for a one-time treatment of retinitis pigmentosa.
“Our partnership marks an important milestone for Ocugen — creating a unique opportunity to provide global access through the Bahamas to OCU400 for people suffering from retinitis pigmentosa,” Chairman, Chief Executive Officer and co-founder Shankar Musunuri stated.
OCU400 is designed as a modifier gene therapy targeting a broad retinitis pigmentosa population, ranging from pediatric to adult patients and from early- to late-stage disease. Ocugen states that the therapy is designed to address mutations involving more than 100 genes.
The candidate uses NR2E3, a nuclear hormone receptor gene involved in functions including photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival. Ocugen is developing the therapy to modify dysfunctional gene networks in retinal cells rather than target only a single disease-causing mutation.
OCU400 has received Regenerative Medicine Advanced Therapy and Orphan Drug designations from the U.S. Food and Drug Administration, as well as Orphan Medicinal Product designation from the European Medicines Agency.
The Bahamas’ LARTA framework covers the regulation of longevity and regenerative therapies, including gene therapies. Priority designation is intended to provide enhanced regulatory engagement and expedited coordination as a program moves toward full approval and operational readiness.
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