Source : WAM
Yas Clinic – Khalifa City, in partnership with the Abu Dhabi Stem Cells Centre (ADSCC) and Vertex Pharmaceuticals, and under the support and supervision of the Department of Health – Abu Dhabi, announced the successful administration of the UAE’s first gene-therapy injection for hemoglobinopathies using CASGEVY, a CRISPR-Cas9 gene-editing therapy.
CRISPR/Cas9 gene-editing is a revolutionary technology that allows precise modification of disease-causing genes by targeting and correcting specific DNA sequences.
The method uses guide RNA to locate the defective gene, while the Cas9 enzyme acts as molecular scissors to cut the DNA at the targeted site, enabling correction of the underlying genetic mutation.
CASGEVY therapy is indicated for patients aged 12 and above 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, commented, “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.”
Dr. Maysoon Al Karam, Chief Medical Officer at Yas Clinic, emphasised the importance of this milestone, noting 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, highlighted the importance of collaboration with Yas Clinic – Khalifa City in bringing the therapy to the UAE, noting that patients who have faced limited treatment options for many years can now benefit from a potential functional cure.
