Opus Genetics announced the launch and funding of a clinical trial evaluating an investigational gene therapy for MERTK-related retinitis pigmentosa, a rare inherited retinal disease that leads to progressive vision loss and eventual blindness. Cleveland Clinic Abu Dhabi will serve as the clinical site, providing advanced retinal imaging, surgical capabilities, and specialized inherited retinal disease clinics. Clinical development activities are expected to begin in 2026.

The trial will assess the safety and efficacy of OPGx-MERTK, an adeno-associated virus–based gene therapy designed to deliver a functional copy of the MERTK gene to retinal cells. Mutations in MERTK disrupt photoreceptor outer segment recycling, resulting in retinal degeneration. There are currently no approved therapies for MERTK-related retinitis pigmentosa, which affects an estimated 60,000 patients worldwide.

Opus Genetics is developing multiple gene therapy programs targeting inherited retinal diseases, including candidates for LCA5-, BEST1-, and RHO-related disorders. RP