Ocugen Announces Publication Of Positive Phase 1 Gardian1 Trial Results For OCU410ST, Modifier Gene Therapy For Stargardt Disease, In Peer-Reviewed Journal Nature Eye

• Study supports favorable safety and tolerability profile and clinically meaningful functional and structural benefits in Stargardt disease patients

MALVERN, Pa., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ:OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced the publication of positive Phase 1 GARDian1 trial results for OCU410ST, its novel modifier gene therapy for Stargardt disease, in the peer-reviewed journal Nature Eye—published by the Royal College of Ophthalmologists.

The publication, A novel modifier gene therapy to treat Stargardt disease: Phase 1 GARDian1 Trial Insights, was authored by Arshad M. Khanani, MD, MA, FASRS, Director of Clinical Research, and Director of Fellowship at Sierra Eye Associates and Clinical Professor at the University of Nevada, Reno School of Medicine; Lejla Vajzovic, MD, FASRS, Professor of Ophthalmology, Director of CME-Ophthalmology, Duke University School of Medicine; Benjamin A. Bakall, MD, PhD. Associated Retina Consultants, President and Founder, Retina Research Foundation of America, Clinical Assistant Professor, University of Arizona College of Medicine; and Ocugen researchers Dr. Murthy Chavali and Dr. Huma Qamar. The publication reports comprehensive 12-month safety, tolerability, and exploratory efficacy data from the first-in-human Phase 1 trial evaluating OCU410ST in patients with early to advanced Stargardt disease.

The Phase 1 GARDian1 trial demonstrated robust efficacy and safety outcomes supporting the clinical development of OCU410ST:

Key Findings include:

• Among six patients with gradable Fundus Auto Fluorescence images, atrophic lesion growth was reduced by 54% (0.55 ± 0.27 mm²) in treated eyes, compared to untreated fellow eyes (1.19 ± 0.31 mm²) over 12 months​
• Lesion expansion was 50% slower (0.10 ± 0.039 mm/year) in treated eyes versus untreated eyes (0.19 ± 0.026 mm/year), below published natural history rates​ (0.14–0.18 mm/year)
• Among six BCVA-evaluable patients without confounders, treated eyes gained +6 letters in BCVA (+4.5 letters) compared to a −1.5 letter decline in untreated fellow eyes at 12​ months
• 100% of treated eyes either stabilized (±4 letters) or improved (≥5 letters) in visual acuity​
• No drug-related serious adverse events or adverse events of special interest were observed​.

Stargardt disease is the most common form of inherited macular degeneration, affecting more than 100,000 people in the United States and Europe combined. The disease is characterized by progressive central vision loss due to photoreceptor degeneration caused by toxic lipofuscin accumulation in the retinal pigment epithelium. Currently, no approved treatment exists for this devastating condition, representing a critical unmet medical need.