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Ocugen Completes Dosing of Stargardt Disease Patients in Phase 1/2 GARDIAN Trial for OCU410ST Gene Therapy

Thursday, May 16, 2024

Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on developing novel gene and cell therapies as well as vaccines, has announced the completion of dosing in the second cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA). This treatment is intended as a one-time therapy for Stargardt disease, aimed at modifying the gene associated with the condition.

Dr. Huma Qamar, Ocugen’s Chief Medical Officer, highlighted the importance of reaching this milestone in the trial. She expressed optimism regarding the positive safety and tolerability profile demonstrated by OCU410ST, indicating the potential for higher doses as the study progresses. The company plans to share initial safety and efficacy data from Phase 1 soon.

Stargardt disease, which lacks FDA-approved treatments, presents a significant medical challenge. Dr. Benjamin Bakall, Director of Clinical Research at Associated Retina Consultants, emphasized the potential of OCU410ST to provide hope for patients with this condition. The completion of dosing for Cohort 2 represents a crucial advancement.

The trial’s Data and Safety Monitoring Board will meet next month to review safety data before advancing to Cohort 3, which involves administering the highest dose. The GARDian trial evaluates the safety and efficacy of OCU410ST in subjects with Stargardt disease, conducted in two phases.

Stargardt disease affects approximately 100,000 individuals in the United States and Europe, leading to progressive vision loss due to retinal degeneration. OCU410ST employs an AAV delivery platform to deliver the RORA gene, targeting pathways associated with Stargardt disease.

Ocugen is dedicated to addressing the unmet needs of individuals with inherited retinal diseases. An update on the OCU410ST clinical trial is anticipated in the third quarter of 2024.

 

Source: ocugen.com

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