Abu Dhabi Launches Advanced Gene-Editing Therapy For Sickle Cell Disease And Thalassemia Patients

In a pioneering move, Abu Dhabi has become the first emirate in the UAE to offer CASGEVY, a groundbreaking CRISPR/Cas9 gene-editing therapy. This innovative treatment marks a significant milestone in the healthcare sector, particularly for individuals afflicted with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).

The therapy, developed through a collaboration between Abu Dhabi Stem Cells Center (ADSCC) and Vertex Pharmaceuticals, signifies a leap in medical innovation. Scheduled to begin at Yas Clinic Hospital in April, the treatment utilises a revolutionary approach by modifying the patient's stem cells to achieve long-lasting benefits.

CRISPR/Cas9 technology stands at the forefront of genetic modification, offering precise editing of the genome to correct mutations responsible for certain diseases. CASGEVY's one-time application involves extracting, editing, and reinserting the patient's stem cells, setting a new standard in therapeutic interventions.

The Department of Health – Abu Dhabi (DoH) has put forth regulations and guidelines, aligned with the highest international standards, to manage gene therapies effectively. This ensures the safety and efficacy of such treatments, with insurance coverage also being included as per the established norms.

Dr. Noura Khamis Al Ghaithi, the Undersecretary of the DoH, emphasized the emirate's commitment to improving healthcare quality by integrating the latest innovations and technologies. "The Department of Health – Abu Dhabi remains dedicated to safeguarding the health and well-being of our community while continuously enhancing the quality of care. Our goal is to establish the world's most efficient healthcare system through the adoption of the latest innovations and promising technologies. By harnessing genomic sciences, we aim to develop cutting-edge therapeutic solutions to complex health challenges, solidifying Abu Dhabi's position as a global leader in healthcare, life sciences, and innovation."

Echoing this sentiment, Dr. Maysoon Al Karam of ADSCC highlighted the significance of the partnership with Vertex Pharmaceuticals. "Partnerships with leading biotechnology companies such as Vertex Pharmaceuticals represent a significant milestone in ADSCC's global mission, aligning with the UAE's vision and strategic health priorities. Patients with genetic disorders such as thalassemia and sickle cell disease will greatly benefit from this revolutionary treatment, which we aim to offer as one of the first advanced gene therapies in the country.

"As the UAE's leading cellular research institution and the UAE's first FACT-accredited centre, we strive through such partnerships to drive medical innovation, developing treatments for a range of critical diseases, and reinforcing Abu Dhabi's position as a global hub for life sciences, in line with the UAE's leadership vision."

The Emirates Drug Establishment (EDE) has also played a crucial role by authorising CASGEVY for market release. This approval allows the treatment of patients aged 12 and over who suffer from sickle cell disease with recurrent vaso-occlusive crises and those with transfusion-dependent thalassemia, further expanding the therapy's reach and impact.