On September 25, 2026, Ocugen, Inc. announced that its AAV-based gene therapy OCU400 received provisional approval and priority designation from the LARTA Board, the regulatory agency responsible for reviewing and approving longevity and regenerative therapy programs within the Commonwealth of The Bahamas. Ocugen will provide OCU400 through an expanded access program (EAP) to treat the first retinitis pigmentosa (RP) patient within 90 days, following full LARTA approval.

The design of OCU400 (AAV5-hNR2E3) differs fundamentally from traditional gene replacement therapies. Rather than targeting a specific disease-causing gene, it delivers NR2E3, a nuclear receptor gene described as a “master regulator” of retina-specific physiological functions. NR2E3 regulates diverse physiological functions within the retina, such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation, and cell survival.

OCU400 is currently in Phase 3 clinical development and has received FDA Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation (ODD), as well as EMA Orphan Medicinal Product Designation (OMPD). It has not yet received marketing approval from the FDA or EMA. The provisional approval under the LARTA framework provides an accelerated pathway for patient access to gene therapies that have not yet completed confirmatory clinical trials.

Unlike approved therapies such as Luxturna, which target specific gene mutations, OCU400 is designed to address more than 100 gene mutations, potentially covering the vast majority of RP patients. If ultimately approved, its potential market could be substantially larger than that of single-gene RP therapies.

As part of the broader gene therapy ecosystem, Genevoyager provides end-to-end CRO/CDMO services, from drug discovery to large-scale manufacturing, leveraging both HEK293 and our proprietary Bac/Sf9 AAV production platforms to produce high-quality AAV vectors with minimal impurities and no detectable rcAAV. With 30+ drug development projects and multiple IIT/IND submissions supported, Genevoyager has helped advance AAV-based gene therapies and recombinant protein vaccines into clinical development, including programs targeting Parkinson’s disease, refractory epilepsy, hepatitis B, and cancer, with selected programs receiving FDA IND clearance and entering Phase I trials. Backed by extensive experience, we are committed to delivering safe, high-quality solutions to academic, pharmaceutical, and biotech partners worldwide.

Source:

https://ir.ocugen.com/news-releases/news-release-details/ocugen-receives-provisional-approval-and-priority-designation

https://ocugen.com/science-and-technology/gene-therapies/

Image source:

https://www.frontiersin.org/journals/medicine/articles/10.3389/fmed.2025.1679619/full