Abu Dhabi, United Arab Emirates - 27 November 2025: Yas Clinic - Khalifa City, in collaboration with the Abu Dhabi Stem Cells Center (ADSCC) and Vertex Pharmaceuticals, a global leader in biotechnology and the development of innovative treatments for complex diseases, announced a landmark partnership to introduce gene therapy in the United Arab Emirates for the very first time. This includes the launch of CASGEVY, the region’s first CRISPR/Cas9-based gene-editing therapy.
This milestone marks a significant step forward for the UAE’s biotechnology and healthcare ecosystem, opening new treatment pathways for patients living with sickle cell disease and transfusion-dependent beta thalassemia. CRISPR/Cas9 gene-editing therapy is a groundbreaking technology that enables highly precise modification of disease-causing genes by targeting and correcting specific DNA sequences. The technique uses guide RNA to identify the defective gene, while the Cas9 enzyme acts as molecular scissors to cut the DNA at the targeted location, allowing correction of the underlying genetic mutation. The UAE Medicines Agency has granted marketing authorization for CASGEVY, enabling its use in patients aged 12 years and older with sickle cell disease experiencing recurrent vaso-occlusive crises, or in those with transfusion-dependent beta thalassemia. Eligible patients will also have access to the therapy through insurance agreements and reimbursement frameworks under the Abu Dhabi Department of Health. In a historic moment for the nation’s medical landscape, the first patient is expected to receive this groundbreaking gene therapy injection at Yas Clinic - Khalifa City during the first week of December this year, marking the beginning of a new era of advanced, life-changing genetic treatments in the UAE. Starting the treatment on UAE National Day carries special significance, symbolizing the UAE’s commitment to progress, resilience, and improving people’s lives. Gene-edited therapy has the potential to address genetic disorders at their source by modifying defective gene sequences within a patient’s own stem cells. Dr. Maysoon Al Karam, Chief Medical Officer at Yas Clinic, emphasized the importance of this milestone, stating that starting the UAE’s first gene-edited therapy during National Day reflects the country’s ambition and dedication to excellence. She noted that this step will enable potentially life-changing solutions for patients and reinforce Abu Dhabi’s growing role as a global center for advanced therapies and life sciences. Hisham Hagar, Executive Country Manager at Vertex GCC, is pleased to be collaborating with Yas Clinic- Khalifa City to bring the therapy to the UAE, highlighting that patients who have faced limited treatment options for many years can now benefit from a potential functional cure. The treatment process involves collecting a patient’s stem cells, editing them outside the body using the CRISPR/Cas9 modality, and reinfusing the modified cells to enable blood production. This rigorous and highly specialized journey includes cell mobilization, comprehensive quality checks, infusion, and long-term clinical monitoring to ensure safety and effectiveness. As the UAE’s first and most advanced stem cell transplant and cellular therapy center, ADSCC and Yas Clinic- Khalifa city continues to support the nation’s vision by expanding access to cutting-edge medical technologies, reducing the need for overseas treatments, and improving long-term outcomes for patients with complex and chronic genetic diseases. The center’s integration of research, clinical care, manufacturing capabilities, GMP laboratories, and FACT-accredited processing facilities further strengthens its position as a leader in regenerative medicine and precision therapies.
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