Opus Genetics Treats First Participant in New Gene Therapy Trial for Retinal Disease

Opus Genetics has officially dosed the very first participant in its Phase 1/2 clinical trial for OPGx-001 — a major step forward for families affected by inherited retinal diseases. This marks the company’s first human trial and an important milestone in the effort to bring new gene therapies to people living with vision-loss conditions. What Is OPGx-001? OPGx-001 is an investigational gene therapy designed for individuals with mutations in the LCA5 gene — a rare but severe cause of Leber congenital amaurosis (LCA). Children born with LCA5-related disease typically experience profound vision loss early in life due to rapid photoreceptor degeneration. Until now, no treatment options have existed. Opus Genetics’ treatment aims to deliver a healthy copy of the LCA5 gene directly to the retina using an adeno-associated viral (AAV) vector. The hope is to preserve and potentially improve vision by restoring the function of cells that would otherwise deteriorate. About the New Clinical Trial The trial — a Phase 1/2, open-label, dose-escalation study — is designed to evaluate: Safety of the