Ocugen, Inc. (OCGN) delivered some encouraging news Thursday about its eye gene therapy, but investors weren't buying it. The company shared preliminary phase 2 results for OCU410, a gene therapy designed to treat geographic atrophy, a progressive form of age-related macular degeneration that leads to vision loss.
A Meaningful Reduction in Vision Damage
The early data from the ArMaDa clinical trial showed a 46% reduction in lesion growth compared to the control group after 12 months. But here's where it gets interesting: the medium dose of OCU410 performed even better, achieving a 54% reduction in lesion size. The high dose came in at 36%, which suggests that more isn't always better when it comes to gene therapy dosing.
What makes this particularly compelling is the safety profile. Across both phase 1 and phase 2 trials, no serious adverse events were reported. For a gene therapy, that's exactly what you want to see alongside efficacy data.
Geographic atrophy affects millions of people worldwide, and current treatments require frequent injections. A one-time gene therapy that could slow disease progression by this magnitude would represent a significant advancement in how we treat this condition.
Ocugen plans to release complete phase 2 data later this quarter and aims to launch a phase 3 trial in 2026. If everything stays on track, the company is eyeing a Biologics License Application filing for OCU410 in 2028.
Expanding the Pipeline
The company has been busy beyond just the geographic atrophy program. Earlier this week, Ocugen announced that phase 1 trial results for OCU410ST, a modifier gene therapy for Stargardt disease, were published in Nature Eye. The peer-reviewed publication included 12-month safety, tolerability, and exploratory efficacy data from the first-in-human trial, with the company reporting robust outcomes that support continued development.
Last September, Ocugen also signed a licensing deal with Kwangdong Pharmaceutical, granting exclusive Korean rights to OCU400, another modifier gene therapy, this one targeting retinitis pigmentosa.




