Ocugen Receives FDA Clearance for Pivotal Phase 2/3 Trial of AAV Gene Therapy OCU410ST for Stargardt Disease

MALVERN, Pa. – June 16, 2025 – Ocugen, Inc. announced today that the FDA has cleared its Investigational New Drug (IND) amendment to initiate a Phase 2/3 pivotal confirmatory trial for OCU410ST, an AAV-based modifier gene therapy candidate for all Stargardt disease (ABCA4-associated retinopathies).

This decision significantly expedites the clinical development of OCU410ST, a therapy that has already shown favorable safety and tolerability in its Phase 1 GARDian trial, alongside slower lesion growth and improved visual function.

OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA gene, aiming to address the complex underlying biological mechanisms of Stargardt disease. The Phase 2/3 trial will enroll 51 participants, with data from the one-year follow-up intended to support a Biologics License Application (BLA) in 2027. This marks Ocugen’s second late-stage clinical program, addressing a significant unmet medical need for patients with this debilitating genetic eye disorder.