Opus Genetics Completes Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy OPGx-LCA5

Opus Genetics 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 said it 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, according to a company news release.

“In the first cohort, OPGx-LCA5 has been well-tolerated and demonstrated clear signs of biological activity through 90 days, warranting continued evaluation in the next highest dose,” said principal investigator Tomas S. Aleman, MD, from the Center for Advanced Retinal and Ocular Therapeutics (CAROT), of the Scheie Eye Institute, Department of Ophthalmology of the Perelman School of Medicine, University of Pennsylvania. “Moreover, early anecdotal and VR challenge test feedback is encouraging and indicates that some of the patients, who have been nearly totally blind all their lives, are now able to see and identify objects for the first time.”

LCA5 is a form of an early-onset retinal degeneration that affects approximately one in 1.7 million people in the US. Currently, there are no approved treatments for individuals with LCA5-related vision loss.

“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, PhD, chief executive officer of Opus. “We’d like to thank the study participants and their families and the incredible efforts of the team at the University of Pennsylvania for reaching this clinical milestone, and we look forward to progressing the trial as we continue to dose escalate.”

For more information on the trial, visit clinicaltrials.gov (NCT05616793).