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Ocugen Doses First Patient in Gene Therapy Trial for Inherited Eye Disorder

NEW YORK – Ocugen on Friday said it has dosed the first patient in a Phase I/II trial of OCU410ST, a gene therapy it is evaluating as a treatment for Stargardt disease.

In the GARDian trial, the Malvern, Pennsylvania-based biotech firm is assessing the safety and efficacy of OCU410ST in treating the rare and inherited eye disease, which causes patients to progressively lose vision. The first phase of the study is a multicenter, open-label, dose-ranging study, while Phase II is a dose-expansion study in which investigators will randomize adult and pediatric patients to receive either one of two OCU410ST doses or to an untreated control group.

OCU410ST uses an adeno-associated viral vector to deliver a functional copy of the RORA gene, which is thought to regulate pathways associated with the disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival networks. In the GARDian trial, patients will receive a dose of the gene therapy in one eye, administered subretinally.