Ocugen disclosed the results of its Phase 1 GARDian1 trial for the OCU410ST gene‑therapy candidate targeting Stargardt disease. The data, published in the peer‑reviewed journal Nature Eye on January 12 2026, confirm the safety and tolerability of the therapy and provide the first evidence of clinical benefit in this patient population.
In the 12‑month study, treated eyes showed a 54 % reduction in atrophic lesion growth compared with untreated fellow eyes, and the rate of lesion expansion was 50 % slower. Best‑corrected visual acuity improved by an average of six letters, while untreated eyes declined by 1.5 letters. All treated eyes either maintained or improved visual acuity, and no drug‑related serious adverse events were reported. These results demonstrate that a single‑dose, AAV5‑mediated delivery of the RORA gene can modify disease progression in a gene‑agnostic manner, potentially benefiting patients regardless of their underlying ABCA4 mutation.
OCU410ST has received FDA orphan drug and rare pediatric disease designations, and the European Medicines Agency has accepted a single U.S. trial for its marketing authorization application. The company plans to file a biologics license application in the first half of 2027, positioning the therapy for a first‑in‑class approval that could address a market of more than 100,000 patients in the U.S. and Europe.
The positive Phase 1 data support the ongoing Phase 2/3 GARDian3 trial, which is ahead of schedule with enrollment expected to finish in the first quarter of 2026. Ocugen’s strategy to file three BLAs in three years—covering Stargardt disease, geographic atrophy, and retinitis pigmentosa—relies on the platform’s ability to deliver a single, lifelong treatment across multiple retinal disorders.
Chief Medical Officer Dr. Huma Qamar said the publication validates the company’s modifier‑gene‑therapy approach and underscores the potential to treat a broad patient population with a single therapy. Chairman, CEO, and co‑founder Dr. Shankar Musunuri highlighted the company’s progress at investor conferences and expressed confidence that the data will accelerate the pipeline and attract strategic partners.
Investors have reacted positively to the data, citing the robust safety profile, meaningful efficacy signals, and clear regulatory pathway as key drivers. The market’s enthusiasm reflects confidence that Ocugen’s gene‑agnostic platform can deliver a first‑in‑class treatment for an unmet medical need, potentially reshaping the retinal gene‑therapy landscape.
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