MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) said newly published clinical trial data support the safety and early effectiveness of its experimental gene therapy for Stargardt disease, a rare inherited form of macular degeneration with no approved treatments.
Results from Ocugen’s Phase 1 GARDian1 trial of OCU410ST were published in the peer-reviewed journal Eye, published by the Royal College of Ophthalmologists. The study reports 12-month safety, tolerability, and exploratory efficacy findings from the first-in-human evaluation of the modifier gene therapy in patients ranging from early to advanced stages of the disease.
According to the publication, treated eyes showed a 54% reduction in atrophic lesion growth compared with untreated fellow eyes, with lesion expansion occurring at roughly half the rate observed in untreated eyes and below published natural history benchmarks. Visual acuity outcomes also favored treatment, with treated eyes gaining letters on standard vision tests while untreated eyes declined.
All treated eyes either stabilized or improved visual acuity over the 12-month period, and no drug-related serious adverse events or adverse events of special interest were reported, the company said.
Stargardt disease is the most common inherited macular degeneration, affecting more than 100,000 people across the United States and Europe. The condition causes progressive central vision loss as toxic lipofuscin accumulates in the retina, ultimately damaging photoreceptors. There are currently no FDA-approved therapies for the disease.
“This publication validates the scientific approach and clinical promise of OCU410ST as a modifier gene therapy for Stargardt disease,” said Dr. Huma Qamar, Ocugen’s chief medical officer. She said the results demonstrate both structural and functional benefits and support a treatment strategy designed to work regardless of a patient’s specific genetic mutation.
Unlike mutation-specific approaches, OCU410ST delivers the RORA gene using an AAV5 viral vector to restore key nuclear hormone receptor signaling pathways in the retina. Ocugen said the therapy is designed to address multiple disease mechanisms, including oxidative stress, inflammation, complement activation, and photoreceptor survival, independent of the underlying ABCA4 genotype.
Lead study author Dr. Arshad M. Khanani said the consistency of benefits across structural and functional endpoints highlights the potential of the modifier gene therapy platform to change outcomes for patients who currently have no disease-modifying options.
The Phase 2/3 GARDian3 trial is progressing ahead of schedule, with enrollment expected to be completed in the first quarter of 2026. Ocugen said it remains on track to pursue a biologics license application in the first half of 2027 as part of a broader regulatory strategy.
If successful in later-stage trials, OCU410ST could become the first approved treatment for Stargardt disease, a milestone researchers say would mark a major shift in care for patients facing progressive vision loss.
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