Opus Genetics Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in Patients with Rare Inherited Retinal Disease LCA5 

Penn spinout Opus Genetics, a patient-first, clinical-stage gene therapy company that develops treatments for inherited retinal diseases, announced that the first patient has been dosed in its Phase 1/2, first-in-human clinical trial of OPGx-LCA5.

The Phase 1/2, open-label, dose-escalation trial aims to evaluate the subretinal delivery of OPGx-LCA5 in nine adult patients with LCA5, an early-onset retinal degeneration that affects approximately one in 1.7 million people in the U.S. The goal of the trial is to evaluate the safety and preliminary efficacy of OPGx-LCA5 in patients who have inherited retinal degeneration due to biallelic mutations in the LCA5 gene.

Learn more about the trial and Opus Genetics here.

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