ABU DHABI: The UAE has introduced its first gene therapy treatment for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), marking a major breakthrough in healthcare innovation. The Department of Health – Abu Dhabi (DoH) has announced the availability of CASGEVY, the first CRISPR/Cas9-based gene therapy treatment in the country.
The treatment will be provided by the Abu Dhabi Stem Cells Center (ADSCC) in collaboration with DoH and Vertex Pharmaceuticals. The first patient is set to receive the revolutionary therapy at Yas Clinic Hospital in April 2025.
CRISPR/Cas9 gene editing allows precise modifications to DNA, offering long-term benefits for genetic disorders. CASGEVY is a one-time gene therapy treatment that extracts stem cells from a patient’s bone marrow, edits them in a lab, and reinfuses them to correct the genetic mutation.
In line with DoH’s mission to advance healthcare innovation, the treatment has been approved under strict regulations and is covered by insurance. Dr Noura Khamis Al Ghaithi, Undersecretary of DoH, emphasised the department’s commitment to cutting-edge treatments, positioning Abu Dhabi as a leader in life sciences.
Dr Maysoon Al Karam, Chief Medical Officer at ADSCC and Yas Clinic, highlighted the significance of partnerships with global biotechnology firms like Vertex Pharmaceuticals, ensuring that the UAE remains at the forefront of medical research.
The Emirates Drug Establishment (EDE) has granted market authorisation for CASGEVY to treat patients aged 12 and above with SCD and TDT, reinforcing the UAE’s role in pioneering medical advancements.
