Ocugen publishes phase 1 GARDian1 gene therapy data for Stargardt disease

Ocugen has announced the publication of positive results from its phase 1 GARDian1 trial of OCU410ST, a gene therapy for Stargardt disease.

Data was published in the peer-reviewed journal Nature Eye, which is published by the Royal College of Ophthalmologists.¹

Huma Qamar, MD, MPH, CMI, chief medical officer at Ocugen, commented on the results, saying, "This publication in Eye validates the scientific approach and clinical promise of OCU410ST as a modifier gene therapy for Stargardt disease. The phase 1 GARDian1 trial demonstrated convergent functional and structural benefits. This represents a paradigm shift from any other approaches, including oral or mutation-constrained replacement approaches, to an agnostic modification strategy that can potentially benefit patients regardless of their underlying ABCA4 mutation with a potential single gene therapy for life. These results provide important support for our ongoing Phase 2/3 GARDian3 trial."

OCU410ST uses an adeno-associated virus delivery platform for the retinal delivery of the RORA gene. RORA regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.^1-2

In the trial, 9 patients with STGD1 (5 females/4 males; mean age 41.7 ± 18.1 years) received OCU410ST in the study eye, with 8 completing the 12-month visit—1 was lost to follow-up. According to the company, among 6 patients with gradable fundus autofluorescence 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 compared to untreated eyes (0.19 ± 0.026 mm/year). In 6 BCVA-evaluable patients without confounders, treated eyes gained +6 letters in BCVA (+4.5 letters) compared with −1.5 ± 2.33 letters in untreated fellow eyes at 12​ months.^1,2

The company noted that the phase 2/3 GARDian3 trial is progressing ahead of schedule with anticipated enrollment completion in Q1 of 2026. The FDA cleared the Investigational New Drug (IND) amendment from Ocugen in June 2025.^1,3

The phase 2/3 clinical trial for OCU410ST will enroll 51 participants diagnosed with Stargardt disease. Of the 51, 34 will receive a 1-time subretinal injection of OCU410ST (200 μL at a concentration of 1.5 × 10¹¹ vector genomes/mL) in the eye with poorer visual acuity. The remaining 17 will be assigned to an untreated control group. The company states the primary objective as the reduction in atrophic lesion size. Key secondary endpoints include improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA).³

The company noted it plans to file a Biologics License Application (BLA) in the first half of 2027.

References:

1. Ocugen Announces Publication of Phase 1 GARDian1 Trial Results for OCU410ST Modifier Gene Therapy. Published January 12, 2026. Accessed January 13, 2026. https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-publication-phase-1-gardian1-trial-results

2. Khanani, A.M., Vajzovic, L., Bakall, B. et al. A novel modifier gene therapy to treat Stargardt disease: Phase 1 GARDian1 Trial Insights. Eye (2026). https://doi.org/10.1038/s41433-025-04202-5

3. Harp MD. Ocugen’s IND amendment to begin phase 2/3 confirmatory trial of OCU410ST cleared by FDA. Published June 16, 2025. Accessed January 13, 2026. https://www.ophthalmologytimes.com/view/ocugen-s-ind-amendment-to-begin-phase-2-3-confirmatory-trial-of-ocu410st-cleared-by-fda