Ocugen Completes Dosing of Stargardt Disease Patients in Phase 1/2 GARDIAN Trial for OCU410ST Gene Therapy
Overview
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.
Words from CMO: Ocugen
- 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 & OCU410ST
- 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.
Safety Data Review Meeting
- 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
- 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
- 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.
