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

The multicenter, randomized trial will enroll 237 patients who will be assigned 2:1 to receive a single 200-µL subretinal injection of OCU410 or no treatment. The primary endpoint is the rate of change in square root–transformed GA lesion area measured by fundus autofluorescence through month 12. Secondary endpoints include low-luminance visual acuity loss and ellipsoid zone area loss on spectral-domain optical coherence tomography.

OCU410 uses an adeno-associated virus serotype 5 vector to deliver the retinoid-related orphan receptor α (RORA) gene. Ocugen is developing the therapy as a one-time treatment intended to affect several pathways implicated in GA.

The phase 3 program follows a phase 2 study in which the selected dose was associated with a 31% reduction in GA lesion growth at 12 months compared with control among patients meeting the phase 3 lesion-size criteria. No OCU410-related serious adverse events or adverse events of special interest were reported, according to the company.

The US Food and Drug Administration (FDA) granted OCU410 Regenerative Medicine Advanced Therapy designation in July 2026. Ocugen said the agency has agreed that a single adequate and well-controlled phase 3 trial could support a biologics license application, which the company anticipates filing in 2028. RP