Ocugen doses first patient in with modifier gene therapy in phase 3 GA trial

Ocugen has dosed its first patient in the global phase 3 registrational trial of OCU410, its first-in-class modifier gene therapy candidate for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).

The initiation of dosing follows the successful completion of a Type B End-of-Phase 2 (EOP2) meeting with CBER in July 2026, resulting in alignment on all critical phase 3 design elements, including primary and secondary endpoints, dose, adaptive design, and a single pivotal trial pathway to support a BLA. The BLA filing is anticipated in 2028.

Back in March, Ocugen announced positive 12-month data from the phase 2 ArMaDa clinical trial evaluating OCU410. 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.

The FDA recently granted OCU410 RMAT designation, providing enhanced agency engagement throughout development and eligibility for accelerated approval and priority review.

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.​