Opus Genetics Completes Dosing in Phase 1/2 Gene Therapy Trial for Inherited Retinal Dystrophy LCA5

In a recent update, Opus Genetics, a company focused on gene therapy for inherited retinal diseases, reported positive results from its Phase 1/2 clinical trial. The trial is testing OPGx-LCA5, a gene therapy candidate for Leber congenital amaurosis (LCA), a severe inherited retinal dystrophy caused by mutations in the LCA5 gene. The first group of adult patients has completed treatment with the subretinal administration of OPGx-LCA5, which utilizes an adeno-associated virus 8 (AAV8) vector for precise gene delivery to the retina.
The initial data indicate that OPGx-LCA5 is safe and has shown biological activity, prompting the company to plan for a higher dosage in upcoming trials. The next phase is expected to begin in mid-2024, with an expansion to include older children and adolescents in the study. The principal investigator, Dr. Tomas S. Aleman, has noted that patients who were nearly completely blind have started to perceive and recognize objects, which is a significant advancement.
LCA5 affects roughly one in 1.7 million individuals in the U.S. and currently lacks any approved treatments. The company's CEO, Dr. Ben Yerxa, expressed optimism about the potential impact of OPGx-LCA5 on patients' lives and gratitude towards the participants and the University of Pennsylvania team for their contributions to the trial's progress.
For further details on the clinical trial, interested parties can visit the clinicaltrials.gov website under the identifier NCT05616793. Opus Genetics, which is supported by the RD Fund, is dedicated to developing treatments for rare forms of inherited blindness, leveraging cutting-edge science and manufacturing efficiencies to bring innovative therapies to patients. The company is headquartered in Research Triangle Park, North Carolina, and is committed to transparently advancing transformative treatments.

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