Cleveland Clinic and Cleveland Clinic Abu Dhabi are expanding access to potentially curative gene therapies for patients with sickle cell disease and transfusion-dependent beta-thalassemia (TDT) in the Middle East.
Both inherited blood disorders are highly prevalent across the region and can cause severe complications, chronic anemia, painful vaso-occlusive crises and reduced life expectancy. Until recently, patients seeking advanced gene therapy often had to travel abroad for treatment.
The collaboration between Cleveland Clinic and Cleveland Clinic Abu Dhabi aims to establish a comprehensive local treatment pathway, reducing the need for international travel while bringing established clinical protocols and multidisciplinary expertise closer to patients.
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Several gene therapies have received U.S. Food and Drug Administration approval since 2022, including Zynteglo for TDT, Lyfgenia for sickle cell disease, and Casgevy for both sickle cell disease and TDT.
Gene therapy involves collecting a patient’s own blood-forming stem cells, modifying them outside the body and reinfusing the cells following conditioning chemotherapy. The process requires extensive screening, stem-cell collection, cell modification, treatment and long-term follow-up and can take up to a year.
“Because these transformational therapies were not widely available in the Middle East at that time, we began considering how to serve patients there safely but also as conveniently as possible,” says Rabi Hanna, MD, Chair of Pediatric Hematology, Oncology and Bone Marrow Transplantation at Cleveland Clinic. “That’s what initiated our collaboration.”
Initially, patients were assessed in Abu Dhabi before travelling to Cleveland for stem-cell collection and treatment, followed by continued care in the UAE. Cleveland Clinic Abu Dhabi is now working toward delivering the complete treatment journey locally.
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The program also encourages early referral for specialist evaluation, particularly for patients with significant disease complications or those without a suitable matched sibling donor. The long-term vision is to develop Cleveland Clinic Abu Dhabi into a regional gene therapy hub serving patients from the Middle East, Africa and India, while supporting the development of future cellular and genetic therapies.
