Opus Genetics Announces Completion of Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in Patients with Rare Inherited Retinal Disease LCA5
RESEARCH TRIANGLE PARK, N.C., March 26, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that the first cohort has completed dosing in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).
- Based on positive safety and efficacy data from the first cohort of three adult patients, the Company will advance OPGx-LCA5 into the next highest dose.
- Opus anticipates initiating the next cohort mid-2024.
- There are also future plans to expand the study population to include subjects 13 years or older.
- “Based on these early clinical data, we’re excited for the potential of OPGx-LCA5 to transform the lives of patients affected by LCA5,” said Ben Yerxa, Ph.D., chief executive officer of Opus.