With the support and oversight of the Department of Health- Abu Dhabi: “Yas Clinic – Khalifa City and Abu Dhabi Stem Cells Center successfully administer the first gene-therapy in

Abu Dhabi, United Arab Emirates - 5 January 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 the successful administration of the first gene-therapy injection for hemoglobinopathies in the United Arab Emirates, using CASGEVY, a CRISPR-Cas9 gene-editing-based therapy.

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 authorisation 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 infusion 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.

In this context, Her Excellency Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health, Abu Dhabi, said: “In Abu Dhabi, we continue to build one of the world’s most advanced, intelligent, and efficient healthcare systems by adopting cutting-edge technologies and the latest gene-based therapies. These innovations open new doors for recovery and give patients renewed hope for a better quality of life. We are proud of this pioneering milestone for Abu Dhabi’s healthcare sector, as Yas Clinic-Khalifa City delivers the first successful gene-therapy injection for hemoglobinopathies. This achievement reflects our continuous commitment to providing exceptional care for our community and strengthening Abu Dhabi’s position as a global leader in healthcare sector.”

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, emphasised 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 centre 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 centre, ADSCC and Yas Clinic - Khalifa City continue 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 centre’s integration of research, clinical care, manufacturing capabilities, GMP laboratories and FACT-accredited processing facilities further strengthens its role in advancing regenerative medicine and precision therapies.