Ocugen Reports Positive 2-Year Results for OCU400 Gene Therapy in Retinitis Pigmentosa

Ocugen, Inc. has announced encouraging 2-year safety and efficacy results from its Phase 1/2 clinical trial of OCU400, a novel gene therapy for the treatment of retinitis pigmentosa (RP) across early to advanced stages in both pediatric and adult populations.

Key Findings

       • Visual Acuity Improvement:
          OCU400 demonstrated a 2-line gain (10 letters) on the ETDRS chart in low-luminance visual acuity (LLVA) in treated eyes compared to untreated fellow eyes.

               • This improvement was statistically significant (p=0.01) across all participants, regardless of genetic mutation.

               • These findings validate OCU400’s gene-agnostic mechanism of action, making it a groundbreaking approach for RP treatment.

       • Durability of Results:
          The 2-year findings align with results observed at the 1-year mark, confirming the long-term potential of OCU400 in preserving and improving visual function.

Expert Insights

Dr. Syed M. Shah, Vice Chair for Research and Digital Medicine at Gundersen Health System, highlighted the significance of OCU400:

“The broad spectrum of genes and mutations causing RP presents a unique challenge in developing treatments for this unmet need. This is where the promise of mutation-agnostic therapies becomes particularly compelling. OCU400’s demonstrated effectiveness across multiple mutations not only offers hope to RP patients but also opens new possibilities for treating other retinal diseases."

Dr. Huma Qamar, Chief Medical Officer at Ocugen, emphasized the therapy’s durability:

“Establishing the long-term safety and efficacy of OCU400 demonstrates the durability of this novel gene therapy. These 2-year low light visual acuity (LLVA) findings, which are the most sensitive measure of visual function, are consistent with the results observed at one year.”

Next Steps

Ocugen is advancing the Phase 3 OCU400 liMeliGhT clinical trial, which remains on track for a Biologics License Application (BLA) submission in the first half of 2026.

Conclusion

The 2-year results of OCU400 underscore its potential to address the challenges of RP with a mutation-agnostic approach, offering hope to patients across diverse genetic backgrounds. This innovative therapy is a promising step forward in treating one of the most challenging retinal diseases.