The trial results revealed that all nine evaluable treated subjects demonstrated improvement or preservation in visual function compared to untreated eyes over a two-year period.

Importantly, OCU400 has shown a favorable long-term safety and tolerability profile, with no serious adverse events reported related to the treatment.

Dr. Syed M. Shah emphasized the significance of OCU400's effectiveness across various mutations, suggesting its potential for broader applications in treating retinal diseases.

Shankar Musunuri, CEO of Ocugen, expressed enthusiasm over the trial results, highlighting the therapy's potential to transform the lives of patients with inherited retinal disorders.

Ocugen, Inc. recently announced a positive update on the Phase 1/2 OCU400 clinical trial for treating retinitis pigmentosa (RP), which took place on January 13, 2025.

Moreover, all treated subjects exhibited improvement or stabilization in mobility testing within the first year of treatment.

Designed as a one-time therapy, OCU400 aims to address the needs of approximately 2 million patients globally suffering from RP, with around 300,000 of those in the U.S. and EU.

This improvement in visual function was statistically significant, with a p-value of 0.01, indicating robust results across various genetic mutations.

Additionally, the treatment led to a meaningful enhancement of two lines (10 letters) on the ETDRS chart in low-luminance visual acuity for the treated eyes.