The US Food and Drug Administration has cleared Ocugen to begin a pivotal phase 2/3 trial of its investigational treatment OCU410ST for Stargardt disease. The trial will evaluate 51 patients, with 34 receiving a one-time subretinal injection of the gene therapy and 17 serving as untreated controls.

The new study follows phase 1 results, in which OCU410ST showed no serious adverse events and achieved a 48% reduction in lesion growth and statistically significant visual improvements over 12 months. The goal of the phase 2/3 study is to reduce lesion size, with secondary endpoints measuring best corrected and low luminance visual acuity.

Stargardt disease, an inherited retinal disorder linked to mutations in the ABCA4 gene, currently has no approved treatments. Ocugen’s candidate uses an AAV-based delivery of the RORA gene, designed to modulate biological pathways such as inflammation, oxidative stress, and cell survival.

The FDA has designated OCU410ST as both an Orphan Drug and a Rare Pediatric Disease treatment. A Biologics License Application is expected in 2027. Ocugen aims to submit 3 such applications over the next three years as part of its broader development strategy, the company said in a press release. RP