Opus Genetics Debuts AAV Gene Therapy Clinical Program to Combat MERTK-Driven Retinal Degeneration

January 27, 2026-Opus Genetics has officially inaugurated a transformative clinical trial for OPGx-MERTK, a sophisticated AAV gene therapy designed to combat MERTK-related retinitis pigmentosa (RP). This milestone represents a strategic expansion of AAV-based genetic medicine into the United Arab Emirates, with clinical development set to begin at Cleveland Clinic Abu Dhabi in 2026. The AAV-mediated program specifically targets a rare form of RP affecting 60,000 people globally, for which no curative AAV or conventional treatments currently exist. By utilizing a high-efficiency AAV vector, Opus Genetics intends to deliver functional genetic material to the retina, potentially halting the progressive vision loss that characterizes this devastating condition.

The pathophysiology of MERTK-related RP involves a breakdown in the retinal pigment epithelium’s ability to recycle photoreceptor components; this AAV therapy is engineered to bypass that defect. The AAV vector carries a healthy copy of the MERTK gene into the patient’s retinal cells, essentially “rebooting” the cellular machinery required to maintain vision. Without this AAV-delivered intervention, the accumulation of cellular debris leads to inevitable blindness. This specific AAV program is of vital importance to the Middle East, where the prevalence of inherited retinal diseases is significantly higher than the global average, making the local launch of an AAV clinical trial a major public health advancement.

The trial is bolstered by the Abu Dhabi Healthcare Research and Innovation Fund, marking a significant sovereign investment into AAV biotechnology. Cleveland Clinic Abu Dhabi will provide the surgical and imaging expertise necessary to administer the AAV infusion and monitor the long-term safety of the AAV-treated patients. As the AAV field matures, this collaboration between Opus Genetics and the Department of Health – Abu Dhabi serves as a model for how global AAV innovation can be localized to address high-density genetic disease clusters. By moving this AAV candidate into the clinic, researchers hope to prove that AAV gene transfer can reliably protect and restore sight in patients who have historically had no hope for treatment.