RESEARCH TRIANGLE PARK, N.C. — Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced the launch and funding of its clinical trial evaluating a gene therapy for MERTK-related retinitis pigmentosa (RP), a rare inherited eye disease that causes progressive vision loss and eventual blindness.
The study is being launched in Abu Dhabi, the capital of the United Arab Emirates in collaboration with the Department of Health – Abu Dhabi (DoH), Cleveland Clinic Abu Dhabi, the Innovative Research Oversight and Support (IROS) division of the M42 group, and the Authority of Social Contribution – Ma’an. Cleveland Clinic Abu Dhabi will serve as the clinical site, equipped with advanced diagnostic imaging, surgical expertise and specialized retinal disease clinics.
The clinical trial will evaluate the safety and efficacy of OPGx-MERTK, an investigational adeno-associated virus (AAV)-based gene therapy developed by Opus Genetics. The therapy is designed to deliver a functional copy of the MERTK gene to retinal cells. Mutations in MERTK impair the retina’s ability to recycle photoreceptor components, leading to progressive degeneration and vision loss. There are currently no approved treatments for MERTK-related retinitis pigmentosa.
The Opus MERTK program is expected to commence clinical development activities in 2026, marking a major milestone for rare disease research and precision medicine in Abu Dhabi and the wider region, where inherited retinal diseases affect an estimated 5% of the population. MERTK-related retinitis pigmentosa affects approximately 60,000 patients worldwide.
George Magrath, M.D., Chief Executive Officer of Opus Genetics said: “Launching our clinical trial for a MERTK gene therapy is a defining moment for patients and for the field of inherited retinal disease. This collaboration with the Department of Health – Abu Dhabi and our partners brings together scientific innovation, clinical excellence, and a shared commitment to addressing serious unmet needs. For patients living with MERTK-related retinitis pigmentosa, this trial represents the first real opportunity to potentially change the course of a disease that has historically led to inevitable vision loss.”
H.E. Dr. Noura Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, said: “By hosting the first MERTK gene therapy clinical trial in the United Arab Emirates (UAE), Abu Dhabi is demonstrating its leadership in precision medicine and rare disease innovation while turning scientific possibility into tangible impact for patients. In collaboration with our international and local partners, we are proud to support the development of a novel therapy that could transform the lives of individuals living with inherited blindness. This milestone brings renewed hope to families and reinforces Abu Dhabi’s position as a trusted destination for advanced healthcare and life sciences research.”
Dr. Fahed Al Marzooqi, Chief Executive Officer of M42’s Integrated Health Solutions, added: “This trial represents a pivotal step in strengthening Abu Dhabi’s position as a global hub for biotech innovation. Through strategic partnerships and clinical excellence, we are translating scientific breakthroughs into real-world treatments for patients with urgent, unmet needs. This collaboration reflects our mission to accelerate access to transformative therapies and to shape the future of rare disease research from the heart of the region.”
Through this trial, Opus Genetics and its partners reaffirm their commitment to advancing precision medicine, accelerating rare disease research, and bringing transformative gene therapies to patients with urgent unmet needs.
About Opus Genetics
Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs). The Company is developing durable, one-time treatments designed to address the underlying genetic causes of severe retinal disorders. The Company’s pipeline includes seven AAV-based programs, led by OPGx-LCA5 for LCA5-related mutations and OPGx-BEST1 for BEST1-related retinal degeneration, with additional candidates targeting RHO, CNGB1, RDH12, NMNAT1, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75%, an approved small-molecule therapy for pharmacologically induced mydriasis, with additional potential indications in presbyopia and low-light visual disturbances following keratorefractive surgery. The Company is based in Research Triangle Park, NC. For more information, visit www.opusgtx.com.
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