Khalifa city: In collaboration with the Abu Dhabi Stem Cells Centre (ADSCC) and Vertex Pharmaceuticals, and with the support and oversight of the Department of Health- Abu Dhabi, announced the successful administration of the first gene-therapy injection for hemoglobinopathies in the UAE, using CASGEVY, a CRISPR-Cas9 gene-editing-based therapy. According to Emirates News Agency, CRISPR/Cas9 gene-editing therapy is a technology that 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, allowing correction of the underlying genetic mutation. The CASGEVY gene therapy is used in patients aged 12 years and older with sickle cell disease experiencing recurrent vaso-occlusive crises, or in those with transfusion-dependent beta thalassemia. Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health- Ab u Dhabi, stated that Abu Dhabi is building one of the world's most advanced healthcare systems by adopting cutting-edge technologies and the latest gene-based therapies. These innovations are seen as opening new doors for recovery and offering patients hope for a better quality of life. Dr. Maysoon Al Karam, Chief Medical Officer at Yas Clinic, noted the importance of this milestone, suggesting that this step will enable potentially life-changing solutions for patients and reinforce Abu Dhabi's role as a global centre for advanced therapies and life sciences. Hisham Hagar, Executive Country Manager at Vertex GCC, emphasized the importance of collaboration with Yas Clinic - Khalifa City in bringing 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.
