Ocugen Completes Enrollment in Pivotal Stargardt Disease Gene Therapy Study
Ocugen has completed enrollment and dosing in its phase II/III GARDian3 study for OCU410ST gene therapy targeting Stargardt disease. The study includes 63 patients and aims to address over 1,200 ABCA4 gene mutations with a single treatment. Interim data is expected in Q3 2026, with a BLA submission targeted for mid-2027.
Ocugen has announced that enrollment and dosing have been completed in the phase II/III GARDian3 pivotal confirmatory study evaluating its pipeline candidate, OCU410ST, for the treatment of Stargardt disease. The milestone was achieved in less than nine months, ahead of schedule, with the company now targeting a mid-2027 biologics license application submission for the gene therapy.
The phase II/III GARDian3 study is a randomized, pivotal study evaluating the safety and efficacy of OCU410ST in 63 patients across all forms and stages of Stargardt disease, including pediatric and adult populations. Participants in the treatment group were given OCU410ST as a one-time subretinal injection using the company's AAV5-based modifier gene therapy platform versus an untreated control group.
OCU410ST has the potential to address more than 1,200 disease-causing mutations in the ABCA4 gene linked to Stargardt disease and other ABCA4-related retinopathies with a single, one-time treatment. Stargardt disease type I is an inherited eye condition caused by biallelic mutations in the ABCA4 gene that typically begins in childhood or adolescence and leads to gradual damage of the macula, resulting in a progressive decline in central vision over time. Currently, there are no FDA-approved treatments available for this rare eye disease.
The primary endpoint focuses on the reduction in atrophic lesion size at 12 months, while the key secondary endpoints include improvements in best-corrected visual acuity and low-luminance visual acuity. Preservation of the Ellipsoid Zone is also being evaluated as an observational endpoint, serving as an early and meaningful indicator of therapeutic benefit. To date, OCU410ST has demonstrated a favorable safety and tolerability profile, with no serious adverse events of special interest reported.
Ocugen expects interim data from the GARDian3 study in the third quarter of 2026, with top-line data anticipated in the second quarter of 2027. The company targets a mid-2027 biologics license application submission for OCU410ST, aligned with its broader goal of filing three BLAs by 2028.
Year to date, shares of Ocugen have gained 32.6% compared with the industry's 4.6% growth.