The Department of Health – Abu Dhabi (DoH) has announced the launch of CASGEVY, marking the introduction of the first CRISPR/Cas9 gene-editing therapy in the UAE. This innovative treatment is aimed at patients suffering from sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT) and is provided by the Abu Dhabi Stem Cells Center (ADSCC) in collaboration with Vertex Pharmaceuticals, a leading biotechnology firm.

The first patient is set to begin this groundbreaking therapy at Yas Clinic Hospital in April 2025. CRISPR/Cas9 gene-editing technology allows precise genome alterations to treat specific genetic diseases, making it a significant advancement in medical treatment. CASGEVY is designed as a one-time therapy, where the patient’s own hematopoietic stem cells are extracted, genetically edited, and then re-implanted to achieve long-term health benefits.

DoH has emphasized its commitment to innovation and patient safety by establishing rigorous regulations and guidelines for managing gene therapies, adhering to international standards. The therapy has also been included in insurance coverage as per existing regulations.

Her Excellency Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, stated that the department is dedicated to enhancing community health and care quality through cutting-edge technologies. She mentioned that the goal is to create one of the world’s most efficient healthcare systems, leveraging genomic sciences to address complex health challenges and solidifying Abu Dhabi’s role as a leader in healthcare and life sciences.

Dr. Maysoon Al Karam, Chief Medical Officer at ADSCC and Yas Clinic, remarked that partnerships with biotechnology firms such as Vertex Pharmaceuticals are key milestones in ADSCC’s mission. She highlighted that this revolutionary treatment will significantly benefit patients battling genetic disorders like thalassemia and sickle cell disease, reinforcing Abu Dhabi’s status as a global hub for life sciences.

Hisham Hagar, Senior Country Manager at Vertex GCC, expressed excitement about the partnership with ADSCC, emphasizing its importance for bringing transformative medicines to patients in the UAE. He noted that this collaboration represents a major step in improving the lives of patients with serious diseases such as SCD and TDT.

The Emirates Drug Establishment (EDE) has granted market authorization for CASGEVY, allowing it to treat patients aged 12 years and older who experience recurrent vaso-occlusive crises from sickle cell disease and those with transfusion-dependent thalassemia.