Phase 2 of OCU410ST GARDian Trial for Stargardt Disease Approved

Ocugen, Inc. (Ocugen), a biotechnology company specializing in gene and cell therapies, biologics, and vaccines, has announced that the Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial has given the green light for the second phase of the Phase 1/2 clinical trial. OCU410ST, a novel modifier gene therapy candidate, is under development to treat Stargardt disease. Charles Wykoff, MD, PhD, Director of Research at Retina Consultants of Texas & Retina Consultants of America and lead study investigator, mentioned that the DSMB's recommendation to move forward with Phase 2 enrollment is based on safety data showing that OCU410ST appears to be safe and well-tolerated to date.

The initial phase of the Phase 1/2 clinical trial was an open-label, dose-ranging study involving nine patients who were administered either a low, medium, or high dose of OCU410ST via subretinal injection. The DSMB determined that the high dose of OCU410ST is the maximum tolerated dose, and no serious adverse events have been reported.

Stargardt disease is the most prevalent form of inherited macular dystrophy, characterized by bilateral central vision loss that begins in childhood and gradually worsens over time. Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen, expressed optimism about the potential of OCU410ST as a one-time novel modifier gene therapy for addressing this significant unmet medical need affecting approximately 100,000 patients in the U.S. and Europe.

The GARDian clinical trial is being conducted at six leading retinal surgery centers across the United States.

Stargardt disease causes retinal degeneration and vision loss due to the degeneration of photoreceptor cells in the macula, the central part of the retina. While some peripheral vision is often preserved, the hallmark of Stargardt disease is decreased central vision. The disorder typically develops during childhood or adolescence, though the age of onset and progression rate can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in individuals with Stargardt disease.

OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA gene. Ocugen's modifier gene therapy approach, based on Nuclear Hormone Receptor RORA, aims to regulate pathways linked to Stargardt disease, including lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks.

Ocugen, Inc. is dedicated to discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines to improve health and offer hope to patients worldwide. The company's breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product. Additionally, Ocugen is advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs.