UAE administers first CRISPR gene therapy, marking major leap in blood disorder treatment

Dubai: The breakthrough treatment was delivered at Yas Clinic – Khalifa City, in collaboration with the Abu Dhabi Stem Cells Centre (ADSCC) and Vertex Pharmaceuticals, under the support and oversight of the Department of Health – Abu Dhabi. The therapy uses CASGEVY, a cutting-edge gene-editing treatment based on CRISPR-Cas9 technology.

CRISPR-Cas9 is a revolutionary medical technique that allows scientists to precisely modify disease-causing genes. Using guide RNA to locate defective DNA sequences, the Cas9 enzyme acts like molecular scissors, cutting the DNA at targeted sites and enabling correction of the underlying genetic mutation responsible for disease.

CASGEVY is approved for patients aged 12 years and above who suffer from sickle cell disease with recurrent vaso-occlusive crises, as well as those with transfusion-dependent beta thalassemia, conditions that have long required lifelong management and limited treatment options.

Commenting on the milestone, Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, said the achievement reflects Abu Dhabi’s commitment to building one of the world’s most advanced and intelligent healthcare systems through the adoption of cutting-edge, gene-based therapies. She added that such innovations open new pathways to recovery and significantly improve patients’ quality of life.

Dr. Maysoon Al Karam, Chief Medical Officer at Yas Clinic, described the development as a potentially life-changing step for patients, while also strengthening Abu Dhabi’s growing reputation as a global centre for advanced therapies and life sciences.

Hisham Hagar, Executive Country Manager at Vertex GCC, underscored the importance of collaboration in bringing the therapy to the UAE, noting that patients who have endured limited treatment choices for decades can now access a potential functional cure through this groundbreaking gene therapy.