Ocugen, Inc., a biotechnology company focused on gene and cell therapies, recently announced the completion of dosing in the first cohort of its Phase 1/2 clinical trial for OCU410ST, a gene therapy candidate for Stargardt disease. Stargardt disease is a rare genetic eye disorder affecting around 35,000 individuals in the United States.

In this milestone event, three subjects received a low dose of OCU410ST through subretinal administration. The trial involves 10 leading retinal surgery centers across the US and is aimed at assessing the safety and efficacy of this potential one-time therapy for Stargardt disease.

Dr. Arun Upadhyay, Ocugen's Chief Scientific Officer, expressed optimism about the progress, highlighting the lack of effective treatment options currently available for Stargardt disease patients. The modifier gene therapy, OCU410ST, represents a beacon of hope for those at risk of vision loss due to this debilitating condition.

The GARDian clinical trial will consist of two phases: a dose-ranging study with three dose levels and a randomized, outcome accessor-blinded dose-expansion study. Through the utilization of an AAV delivery platform, OCU410ST targets the RORA gene to address various pathways linked to Stargardt disease.

Stargardt disease manifests as progressive vision loss caused by the degeneration of photoreceptor cells in the macula. This disease typically emerges during childhood or adolescence, leading to decreased central vision while preserving some peripheral vision.

Ocugen, Inc. continues its dedication to developing innovative gene and cell therapies, with a particular focus on retinal diseases. For further updates and information on Ocugen's groundbreaking medical advancements, visit www.ocugen.com.