Phase 1/2 Trial for Stargardt: OCU410ST Dosing Complete in First Cohort

Ocugen, a biotech firm, has reached a significant phase in its clinical trial for a gene therapy treatment for Stargardt disease, a rare form of blindness. The first group of participants has completed dosing in the Phase 1/2 GARDian trial, which is testing OCU410ST—a potential one-time therapy. The condition affects approximately 35,000 individuals in the United States and currently lacks an effective treatment.

Dr. Arun Upadhyay, Ocugen's Chief Scientific Officer, highlighted the importance of this milestone, emphasizing that OCU410ST offers hope to patients who face the prospect of vision loss. The trial, which involves up to 10 prominent retinal surgery centers in the U.S., has administered a low dose of the therapy to three subjects via a single subretinal injection.

Benjamin Bakall, MD, PhD, from Associated Retina Consultants and the University of Arizona, expressed his enthusiasm for the study, noting the absence of effective treatments to prevent vision loss in Stargardt patients. He believes the new approach could provide a therapeutic alternative.

The GARDian trial is divided into two phases. Phase 1 is an open-label, dose-ranging study across three levels, while Phase 2 is a randomized, dose-expansion study with adult and pediatric subjects. The trial will evaluate the safety of the subretinal administration of OCU410ST.

Stargardt disease is characterized by the degeneration of photoreceptor cells in the retina's macula, leading to progressive vision loss. The disease typically emerges during childhood or adolescence and affects the retinal pigment epithelium, a supportive layer for photoreceptors.

OCU410ST is an AAV-based gene therapy that delivers the RORA gene to the retina. This approach is centered on the Nuclear Hormone Receptor RORA, which is linked to pathways associated with Stargardt disease, such as lipofuscin formation and oxidative stress.

Ocugen is dedicated to developing innovative therapies and vaccines to improve global health. Their breakthrough gene therapy platform aims to treat various retinal diseases and is also being explored for infectious and orthopedic diseases.

The company's commitment to innovation is driving new scientific frontiers, with the potential to make a substantial impact on patient care. Ocugen's mission is to forge new paths in science, leveraging both intellectual and human resources to bring about transformative treatments for a range of conditions.