ASRS 2025: Phase 1/2 study update on OCU-410 for geographic atrophy

Jay Chhablani, MD, Vice Chair at the University of Pittsburgh Medical Center, presented the phase 1/2 clinical trial data for Ocugen's OCU410, at the 2025 annual meeting of the American Society of Retina Specialists. This meeting was held in Long Beach, California from July 30 through August 2, 2025.

The presentation highlighted several key findings from the research. The study's safety profile was particularly noteworthy, with no serious adverse events reported during the 12-month follow-up period. Researchers observed significant therapeutic benefits, including a more than 40% decrease in lesion size and a four-line improvement in low luminance visual acuity.

Chhablani emphasized the unique characteristics of this gene therapy approach. The treatment was delivered subretinally, requiring a single surgical intervention rather than repeated monthly injections. He described it as an almost "one-and-done" therapy that could effectively slow disease progression. The subretinal delivery method involved introducing the drug in the peripheral retinal area.

While some pigmentary changes were expected in the initial follow-up period, these changes were not anticipated to significantly impact patient vision due to the treatment's specific location. The research team was optimistic about the therapy's potential.

The Phase 2 enrollment had already been completed, and Ocugen is preparing to launch Phase 3 trials in the first quarter of 2026. This progression suggested promising next steps for the treatment's potential clinical application. The gene therapy's approach represented a potentially transformative method for managing dry macular degeneration. By offering a single surgical intervention that could provide long-term benefits, the treatment offered hope for patients seeking alternatives to ongoing, repetitive treatments. The presentation concluded with a sense of excitement about the future possibilities of OCU410, highlighting its potential to provide a more convenient and potentially more effective treatment option for patients suffering from this degenerative eye condition.