MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has announced that the first patient has been dosed in its Phase 2/3 GARDian3 clinical trial evaluating OCU410ST, a gene therapy candidate designed to treat Stargardt disease — a rare, inherited form of macular degeneration that currently lacks FDA-approved treatment options.
“Dosing the first patient is an especially significant milestone and brings us closer to our goal of addressing the unmet medical need that exists for all Stargardt patients—100,000 in the U.S. and Europe and 1 million worldwide,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Progressing our second modifier gene therapy candidate into a registration clinical trial is a pivotal step in potentially providing a one-time therapy for life for the millions of patients affected by inherited retinal diseases.”
OCU410ST builds on results from the earlier Phase 1 GARDian trial, where treated eyes demonstrated 48% slower lesion growth and nearly two lines of visual acuity improvement at 12 months, compared to untreated eyes. The improvements were statistically significant, with a p-value of 0.031.
The company’s Chief Medical Officer, Dr. Huma Qamar, emphasized the design of the current trial: “Initiating dosing in this pivotal Phase 2/3 study is an important advancement for Ocugen and more importantly for the Stargardt community. The adaptive design of this trial, including a masked interim analysis at 8 months on 24 subjects, enables us to efficiently evaluate early signals of efficacy and safety while optimizing study conduct. This ensures we generate robust and meaningful data to support our regulatory submissions for approvals.”
The study will enroll 51 patients. Of those, 34 will receive a single subretinal injection of OCU410ST in the eye with poorer visual acuity. The remaining 17 will serve as untreated controls. The primary goal is to assess reduction in lesion size, while secondary endpoints will track improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA).
“Treating the first patient with this novel gene therapy in the GARDian3 trial is a proud and hopeful moment for our team and for families affected by Stargardt disease,” said Dr. Victor H. Gonzalez, Principal Investigator and retinal surgeon at Valley Retina Institute in McAllen, Texas. “For decades, patients have faced the progressive loss of central vision with no approved treatment options. The encouraging Phase 1 results give us confidence that OCU410ST could meaningfully slow disease progression and help preserve vision. This trial brings us closer to the possibility of a one-time gene therapy that could transform patients’ quality of life for years to come.”
OCU410ST employs an AAV delivery system to transport the RORA gene to the retina, part of Ocugen’s modifier gene therapy platform. The approach targets multiple biological pathways implicated in retinal disease, including inflammation, oxidative stress, and photoreceptor cell survival.
The company aims to use the data from this study to support a Biologics License Application (BLA) in 2027. The trial represents Ocugen’s second late-stage clinical program as it pursues regulatory filings for three gene therapies over the next three years.
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