Four-Mechanism Gene Therapy Shows Slower GA Lesion Growth

The phase 1/2 study also showed no serious adverse events

By: Jay Chhablani, MD, Michael A. Singer, MD

Retinal Physician November 21, 2025

5 Key Takeaways

1

The phase 1/2 study of Ocugen's OCU410 gene therapy for geographic atrophy showed no serious adverse events.
2

OCU410 demonstrated a 23% slower growth of GA lesions in the study eye compared to fellow eyes at 12 months.
3

Interim results from the phase 2 study indicated a 27% slower GA lesion growth compared to fellow eyes.
4

Visual function improved or stabilized by 2 lines in the study eye, with some patients reporting significant benefits.
5

The next step for the OCU410 program is to initiate a phase 3 trial by April 2026.

Jay Chhablani

Retina Specialist

Affiliations: UPMC Vision Institute

Specialties: Ophthalmology

Bio: Jay Chhablani, MD, is a retina specialist at UPMC Vision Institute and serves on the ARVO retina program committee. His clinical and scientific interests include precision medicine, retinal imaging, artificial intelligence applications in ophthalmology, geographic atrophy biomarkers, and high-resolution imaging approaches such as adaptive optics. At ARVO 2026, Dr. Chhablani highlighted emerging work integrating genetics and artificial intelligence, validation of biomarkers such as the ellipsoid zone in geographic atrophy, and laboratory models incorporating retinal pigment epithelium, Bruch’s membrane, and choroidal vessels.

Michael A. Singer

Specialties: Ophthalmology

Bio: Michael A. Singer, MD, is a physician author covering retina topics for Retinal Physician. In a 2025 Retina Minute article, he discussed low-dose 0.01% atropine as a nonsurgical potential treatment for symptomatic vitreous floaters, including patient selection considerations and the importance of peripheral retinal examination before prescribing.

PentaVision

Retinal Physician

Four-Mechanism Gene Therapy Shows Slower GA Lesion Growth

5 Key Takeaways

1

2

3

4

5

Recommendations