Ocugen completes dosing of first cohort in trial of gene therapy for Stargardt disease
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Biotechnology company Ocugen announced that dosing is complete in the first cohort of its phase 1/2 GARDian clinical trial of OCU410ST, a modifier gene therapy candidate for treatment of Stargardt disease.
“This is an important clinical milestone for our first-in-class, potential one-time therapy for the treatment of Stargardt disease,” Arun Upadhyay, PhD, Ocugen’s chief scientific officer and head of research and development, said in a company press release. “Although Stargardt is one of the most common inherited retinal diseases, there remains no treatment option to address this condition. OCU410ST provides hope to these patients who may eventually lose their vision.”
According to the release, Stargardt disease is a genetic eye disorder and the most common form of inherited age-related macular degeneration. It affects approximately 35,000 people in the U.S.
The trial, with participants from up to 10 retinal surgery centers in the U.S., is designed to assess the safety of unilateral subretinal administration of OCU410ST in two phases, the first of which is an open-label, dose-ranging study of low (3.75×10E10 vg/mL), medium (7.5×10E10 vg/mL) and high (2.25×10E11 vg/mL) doses. This announcement marks the completion of dosing for three participants in the first cohort, who received the low dose.
The second phase is an outcome accessor-blinded, dose-expansion study of adult and pediatric patients randomized to one of two OCU410ST dose groups or an untreated group.
“I am very pleased to participate in this study and to offer a novel modifier gene therapy option to my patients,” Benjamin Bakall, MD, PhD, director of clinical research at Associated Retina Consultants and clinical assistant professor at the University of Arizona, said in the release. “Until now, we have not had any effective treatment that can prevent the vision loss in patients with Stargardt disease. Now, I believe that this approach can offer a new therapeutic option to address the disease itself.”
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