Yas Clinic – Khalifa City, in collaboration with the Abu Dhabi Stem Cells Centre (ADSCC) and Vertex Pharmaceuticals, announced the successful administration of the first gene therapy injection for hemoglobinopathies in the UAE. The procedure was carried out with the support and oversight of the Department of Health ,Abu Dhabi.

The therapy, CASGEVY, is based on CRISPR-Cas9 gene-editing technology. It enables 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. This allows correction of the underlying genetic mutation.

CASGEVY is intended for patients aged 12 years and older with sickle cell disease experiencing recurrent vaso-occlusive crises or those with transfusion-dependent beta thalassemia.

Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health –Abu Dhabi, stated, “In Abu Dhabi, we continue to build an advanced healthcare system by adopting cutting-edge technologies and gene-based therapies. These innovations open new doors for recovery and give patients renewed hope for a better quality of life.”

Dr. Maysoon Al Karam, Chief Medical Officer at Yas Clinic, highlighted that this milestone could provide potentially life-changing solutions for patients and strengthen Abu Dhabi’s role as a centre for advanced therapies and life sciences.

Hisham Hagar, Executive Country Manager at Vertex GCC, noted that collaboration with Yas Clinic – Khalifa City brings this therapy to the UAE. He added that patients who have faced limited treatment options for many years can now benefit from a potential functional cure.

Key Points:

• First CRISPR-based gene therapy administered for hemoglobinopathies in UAE.
• CASGEVY targets sickle cell disease and transfusion-dependent beta thalassemia.
• Therapy offers precise gene correction and potential functional cure.

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