MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focusing on innovative therapies, announced the completion of dosing in the second cohort of its groundbreaking GARDian clinical trial for OCU410ST, a gene therapy candidate targeting Stargardt disease. This disease is a leading cause of inherited retinal degeneration with no FDA-approved treatments, affecting the central vision crucial for tasks such as reading and driving.
The therapy, OCU410ST (AAV-hRORA), represents a novel approach in treating Stargardt disease through a one-time modifier gene therapy aimed at providing lifelong benefits. “The completion of dosing for Cohort 2 participants signifies an important clinical milestone for our pioneering modifier gene therapy,” stated Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen. The company reports encouraging data on the therapy’s safety and tolerability, facilitating the move to higher doses in ongoing studies.
To date, six patients have received the therapy in this Phase 1/2 trial, which is designed to evaluate the safety and efficacy of OCU410ST. An additional three patients are slated to receive a higher dose in the next phase of the study. Benjamin Bakall, MD, PhD, underscored the acute need for effective treatments for Stargardt disease, praising the potential of OCU410ST to fill this void.
The trial is structured into two phases, with Phase 1 focused on determining the optimal dosage through a multicenter, open-label, dose-ranging study. This involves three levels of dosage, culminating with a high dose that is yet to be administered. Phase 2 will expand this evaluation, comparing outcomes between treated and untreated groups across different doses.
A Data and Safety Monitoring Board is scheduled to review the medium dose cohort’s 4-week safety data next month before proceeding to the high-dose phase, marking the final stage of the Phase 1 dose-escalation study.
Ocugen’s GARDian trial aims to offer a beacon of hope for patients with Stargardt disease by potentially providing a viable and long-lasting treatment option. With preliminary safety and efficacy data expected in the third quarter of 2024, Ocugen’s efforts could mark a significant advance in the field of genetic eye diseases, addressing a critical unmet medical need and paving the way for future gene therapy applications.
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