ABU DHABI, 27th November, 2025 (WAM) -- Yas Clinic – Khalifa City, in collaboration with the Abu Dhabi Stem Cells Centre (ADSCC) and Vertex Pharmaceuticals, has announced a landmark partnership to introduce gene therapy in the UAE for the first time, including the launch of CASGEVY, the region’s first CRISPR/Cas9-based gene-editing therapy.
The milestone marks a major advance for the UAE’s biotechnology and healthcare landscape, opening new pathways for patients with sickle cell disease and transfusion-dependent beta thalassemia.
CRISPR/Cas9 therapy enables precise modification of disease-causing genes by correcting targeted DNA sequences using guide RNA and the Cas9 enzyme.
The UAE Medicines Agency has granted marketing authorisation for CASGEVY for patients aged 12 years and older with recurrent vaso-occlusive crises in sickle cell disease or transfusion-dependent beta thalassemia. Eligible patients will access the therapy through insurance agreements and reimbursement frameworks under the Abu Dhabi Department of Health.
The first patient is expected to receive the gene therapy injection at Yas Clinic – Khalifa City during the first week of December, marking the start of a new era of advanced, life-changing genetic treatments in the country. Beginning the treatment on UAE National Day carries symbolic significance, reflecting national commitment to progress and improved patient outcomes.
Dr. Maysoon Al Karam, Chief Medical Officer at Yas Clinic, said starting the UAE’s first gene-edited therapy during National Day highlights national ambition and Abu Dhabi’s growing role as a centre for advanced therapies and life sciences.
Hisham Hagar, Executive Country Manager at Vertex GCC, said the collaboration will provide patients long limited in treatment options with access to a potential functional cure.
The treatment process involves collecting a patient’s stem cells, editing them outside the body using CRISPR/Cas9, then reinfusing the modified cells to restore blood production. The rigorous pathway includes cell mobilisation, quality checks, infusion and long-term monitoring.
As the UAE’s first and most advanced stem cell transplant and cellular therapy centre, ADSCC and Yas Clinic – Khalifa City continue to expand access to cutting-edge medical technologies, reduce the need for overseas treatment and improve long-term outcomes for complex genetic diseases. Their integrated research, clinical care, manufacturing capabilities, GMP laboratories and FACT-accredited facilities reinforce their leadership in regenerative medicine and precision therapies.