Ocugen modifier gene therapy hits phase 2 goals in geographic atrophy

Ocugen announced positive 12-month data from the phase 2 ArMaDa clinical trial evaluating OCU410 (AAV5-RORA), its novel modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).

Key findings from phase 2 include: 31% reduction in lesion growth in the optimal dose (medium) group compared to control; 27% slower rate of ellipsoid zone loss compared to control, indicating structural preservation of photoreceptors, which correlates with visual function; 55% of treated patients demonstrated ≥30% lesion size reduction vs. control.

GA — an advanced, late-stage form of dAMD — is a multifactorial disease with a complex etiology that involves genetic and environmental factors. There are limited options for patients with dAMD in the U.S. and current therapies require 6-12 injections per year indefinitely.

Current treatment options for GA in the U.S. are limited to those targeting a single mechanism — the complement pathway.  By contrast, OCU410 is a first-in-class retinoid-related orphan receptor alpha (RORA)-based gene therapy designed to support central retina and photoreceptor integrity through a multi-pathway mechanism — targeting drusen, inflammation, oxidative stress, and complement activation.​

Ocugen plans to initiate the OCU410 phase 3 registrational trial in the third quarter of 2026 in line with the company’s goal of three BLA filings in three years.