Opus Genetics Launches Gene Therapy Clinical Trial for MERTK-Related Retinitis Pigmentosa

Opus Genetics has initiated enrollment in a Phase 1/2 clinical trial evaluating OPGx-MERTK, an investigational gene therapy for retinitis pigmentosa caused by mutations in the MERTK gene. The trial represents a significant step forward for patients with this rare form of inherited retinal disease, which currently has no approved treatments.

The multicenter, open-label study will enroll participants across three dose-escalation cohorts to evaluate the safety, tolerability, and preliminary efficacy of a single subretinal injection of OPGx-MERTK. The main trial site will be the Cleveland Clinic in Abu Dhabi, in collaboration with the Department of Health in Abu Dhabi and other local support groups.

Mutations in the MERTK gene cause approximately 1–2% of cases of RP, representing thousands of individuals worldwide living with progressive vision loss that begins in childhood or early adulthood. Patients typically experience night blindness, peripheral vision loss, and eventual central vision loss.

OPGx-MERTK uses an adeno-associated virus (AAV) vector to deliver a functional copy of the MERTK gene directly to retinal cells, addressing the underlying genetic cause of the disease.

"The launch of this clinical trial demonstrates the power of sustained research investment in rare retinal diseases," said Jason Menzo, CEO of the Foundation Fighting Blindness. "Each new clinical trial brings us closer to our mission of providing treatments and cures for blinding diseases."

Through the generosity of donors, the Foundation Fighting Blindness has been instrumental in advancing research into MERTK-related RP and supporting innovative gene therapy approaches. The Foundation's commitment to funding early-stage research and clinical development has helped pave the way for promising treatments like OPGx-MERTK to reach clinical trials.

In 2021, the RD Fund, the venture philanthropy arm of the Foundation Fighting Blindness, launched Opus Genetics as a patient-focused gene therapy company targeting inherited retinal diseases. In 2024, Opus Genetics was acquired and is now publicly traded.