Phase 1/2 Gene Therapy Trial for LCA5 Disease Completes Initial Dosing

Opus Genetics, a clinical-stage gene therapy company, has reported positive preliminary results from its Phase 1/2 clinical trial for OPGx-LCA5, a treatment for Leber congenital amaurosis (LCA) caused by biallelic mutations in the LCA5 gene. The first group of adult patients has completed dosing and shown good tolerance to the therapy, with indications of biological activity observed within 90 days.

The OPGx-LCA5 treatment utilizes an adeno-associated virus 8 (AAV8) vector to deliver a functional version of the LCA5 gene to the outer retina. The first cohort's positive response has prompted the company to proceed with a higher dose, with the next phase expected to begin in mid-2024. The study may also be expanded to include participants as young as 13 years old.

Principal investigator Dr. Tomas S. Aleman from the University of Pennsylvania highlighted that the initial findings are encouraging, with some patients who were nearly completely blind now being able to see and identify objects. LCA5 is a rare form of retinal degeneration that affects approximately one in 1.7 million people in the U.S., and currently, there are no approved treatments available.

Ben Yerxa, Ph.D., CEO of Opus Genetics, expressed enthusiasm for the potential impact of OPGx-LCA5 on affected patients' lives. He acknowledged the contributions of the study participants, their families, and the research team at the University of Pennsylvania in achieving this milestone. The company is committed to advancing the trial and escalating the dosing regimen.

OPGx-LCA5 is designed to target an early-onset severe inherited retinal dystrophy caused by mutations in the LCA5 gene. The ongoing Phase 1/2 clinical trial at the University of Pennsylvania is aimed at assessing the safety and preliminary efficacy of the treatment in patients with inherited retinal degeneration due to these mutations.

Opus Genetics is dedicated to developing gene therapies for inherited retinal diseases, with a focus on addressing the needs of patients with orphan retinal diseases. The company's portfolio includes gene therapy programs for various forms of inherited blindness, with the LCA5 program currently in clinical trials. Opus Genetics is based in Research Triangle Park, N.C., and is supported by the RD Fund, the venture arm of Foundation Fighting Blindness. The company aims to develop transformative treatments for patients by leveraging the best scientific knowledge and the expertise of pioneers in ocular gene therapy.