First Patient Dosed in LCA5 Gene Therapy Clinical Trial Launched by Opus Genetics

Opus Genetics, a company developing gene therapies for people with inherited retinal diseases, has dosed the first patient in its Phase 1/2 gene therapy clinical for Leber congenital amaurosis 5 (LCA5), which causes significant vision loss in children. The Phase 1/2 clinical trial, enrolling nine adult patients, is being conducted at the University of Pennsylvania. Once safety in adults has been established and confirmed by the US Food and Drug Administration, Opus plans to dose pediatric patients.

Known as OPGx-001, the gene therapy uses a human-engineered adeno-associated virus (AAV) to deliver healthy copies of the LCA5 gene to the retinas of patients, augmenting the mutated copies causing vision loss. The therapy is administered through a one-time injection underneath the retina. Researchers believe gene therapies will be effective for many years, perhaps the life of the patient.

The LCA5 gene-therapy clinical trial is the first launched by Opus, a company originally conceived and formed by the Foundation Fighting Blindness. Founded in 2021, Opus received $19 million in seed funding from the Foundation’s RD Fund, a venture philanthropy fund for emerging retinal disease therapies in or nearing early-stage clinical trials. The company is led by Ben Yerxa, PhD, former chief executive officer of the Foundation.