Ocugen (NASDAQ:OCGN) announced Monday that it has completed patient enrollment for its pivotal Phase 3 liMeliGhT clinical trial, evaluating OCU400 for the treatment of retinitis pigmentosa (RP).
The trial is the first of its kind to utilize a gene-agnostic "modifier" approach, aiming to treat patients regardless of the specific genetic mutation causing their vision loss.
The study randomized 140 patients in a 2:1 ratio into treated and untreated control arms.
Unlike traditional gene therapies that replace a single defective gene, OCU400 delivers a functional copy of the NR2E3 gene.
This "master regulator" helps reset dysfunctional gene networks in the retina, potentially stabilizing or improving vision across the more than 100 different genetic mutations that cause RP.
Alongside the enrollment update, Ocugen released positive long-term data from its Phase 1/2 study.
After three years of follow-up, OCU400 continued to show a favorable safety profile with no new treatment-related serious adverse events.
Efficacy remained durable, with 88% (7/8) of evaluable treated subjects demonstrating improvement or preservation of vision.