MALVERN, PA — Ocugen Inc. (Nasdaq: OCGN) has completed enrollment and dosing in its Phase 2/3 clinical trial evaluating a gene therapy candidate for Stargardt disease.
The GARDian3 trial enrolled 63 patients and was completed in less than nine months, according to the company. Topline results are expected in the second quarter of 2027, with a planned Biologics License Application submission by mid-2027.
The study is evaluating OCU410ST, a modifier gene therapy designed for Stargardt disease and other ABCA4-associated retinopathies, a group of inherited retinal conditions with no approved treatments.
According to Ocugen, the randomized, controlled trial is assessing the therapy’s ability to reduce atrophic lesion size at 12 months, with secondary endpoints including changes in visual acuity and other measures of retinal function.
Participants received a one-time subretinal injection of the therapy in the eye with poorer vision, while a control group received no treatment.
An interim analysis is planned for the third quarter of 2026 after a subset of patients completes eight months of follow-up, the company said.
Ocugen reported that the therapy has shown a favorable safety profile to date, with no serious adverse events or events of special interest observed in the trial.
The company said earlier-stage data from a Phase 1 study showed a reduction in lesion growth and improvements in visual acuity in treated eyes compared with untreated eyes.
Stargardt disease is a genetic eye disorder that typically begins in childhood or adolescence and leads to progressive central vision loss. The condition affects an estimated 100,000 people in the United States and Europe, according to the company.
Ocugen said OCU410ST is designed as a one-time treatment that targets disease pathways independent of specific genetic mutations associated with the condition.
The trial represents the company’s second late-stage clinical program as it advances multiple gene therapy candidates for inherited retinal diseases.
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