Department of Health – Abu Dhabi, through Abu Dhabi Stem Cells Center, introduces advanced gene-editing therapy for sickle cell disease and thalassemia for first time in UAE

Abu Dhabi, 28 March 2025: The Department of Health – Abu Dhabi (DoH) has announced the introduction of CASGEVY, the first CRISPR/Cas9 gene-editing therapy in the UAE, offering innovative treatment for patients suffering from sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).

The innovative treatment, provided by Abu Dhabi Stem Cells Center (ADSCC), in coordination with DoH and in collaboration with Vertex Pharmaceuticals, a leading biotechnology company, positions Abu Dhabi as a leading destination for life sciences. 

The first patient is scheduled to begin the groundbreaking therapy at Yas Clinic Hospital in April of this year.

CRISPR/Cas9 gene-editing is an innovative technology that enables precise genome editing to treat certain genetic diseases by targeting and altering specific DNA sequences. CASGEVY is a one-time treatment tailored for each patient by extracting the patient’s own hematopoietic stem cells, genetically editing them in the lab, and then re-implanting them into the patient's body to provide long-term positive effects.

In line with DoH’s commitment to fostering innovation and ensuring the health and safety of patients in the emirate, the department has established regulations and guidelines for the management of gene therapies, according to the highest international standards. The treatment has been included in insurance coverage in accordance with established regulations and standards.

Her Excellency Dr Noura Khamis Al Ghaithi, Undersecretary, DoH, said: “The Department of Health – Abu Dhabi remains dedicated to safeguarding the health and well-being of our community while continuously enhancing the quality of care. Our goal is to establish the world’s most efficient healthcare system through the adoption of the latest innovations and promising technologies. By harnessing genomic sciences, we aim to develop cutting-edge therapeutic solutions to complex health challenges, solidifying Abu Dhabi’s position as a global leader in healthcare, life sciences, and innovation.”

Dr Maysoon Al Karam, Chief Medical Officer at ADSCC and Yas Clinic, said: "Partnerships with leading biotechnology companies such as Vertex Pharmaceuticals represent a significant milestone in ADSCC's global mission, aligning with the UAE's vision and strategic health priorities. Patients with genetic disorders such as thalassemia and sickle cell disease will greatly benefit from this revolutionary treatment, which we aim to offer as one of the first advanced gene therapies in the country. As the UAE’s leading cellular research institution and the UAE's first FACT-accredited center, we strive through such partnerships to drive medical innovation, developing treatments for a range of critical diseases, and reinforcing Abu Dhabi's position as a global hub for life sciences, in line with the UAE's leadership vision.

Hisham Hagar, Senior Country Manager at Vertex GCC, said: “We are delighted to partner with the Abu Dhabi Stem Cells Center, the UAE’s leading cellular research institution, to bring the world’s first CRISPR/Cas9 gene-editing medicine to patients in the UAE. This partnership represents a significant step toward bringing transformative medicines to patients in the UAE for serious diseases like SCD and TDT, ultimately advancing our mission to improve patients’ lives in the UAE.”

The Emirates Drug Establishment (EDE) in the UAE, has granted a market authorisation for CASGEVY to treat patients aged 12 years and older who suffer from sickle cell disease with recurrent vaso-occlusive crises, as well as transfusion-dependent thalassemia.