"Yas Clinic" and"Abu Dhabi Stem Cells" They succeed in providing the first gene therapy injection for hemoglobinopathy
ABU DHABI, 5 JANUARY / WAM / Yas Clinic Hospital – Khalifa City, in cooperation with the Abu Dhabi Stem Cell Center “ADSCC” and “Vertex” Company, with the support and supervision of the Department of Health – Abu Dhabi, succeeded in providing the first injections of gene therapy for hemoglobinopathies, using the “CASGEVY” treatment based on the gene editing technology “CRISPR/Cas9 – CRISPR-Cas9”, in a medical achievement that is the first of its kind in the country.
This achievement represents a pivotal step that contributes to consolidating Abu Dhabi’s position as a leading destination for healthcare and medical innovation, and opens new treatment horizons for patients with hereditary blood disorders, especially sickle cell anemia and thalassemia that depend on blood transfusions.
The “CRISPR/Cas9” technology for gene therapy relies on the principle of precise modification of disease-causing genes, by targeting specific sequences of DNA using directed RNA, while the “9Cas” enzyme acts as a molecular scissors that cuts the DNA at the desired location, allowing the genetic defect to be corrected from its roots. This development is considered one of the most advanced technologies in the world in the field of personal and precise treatments.
“CASGEVY” gene therapy is used to treat patients aged 12 years and above who suffer from sickle cell anemia with recurrent vascular obstructive episodes, or thalassemia patients who are dependent on blood transfusions.
Eligible patients will also be able to obtain treatment based on the evaluation of the treating physician and in accordance with applicable standards.
Her Excellency Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, confirmed that Abu Dhabi is moving steadily towards establishing a smart and highly efficient health system, by adopting advanced technological technologies and modern genetic therapies, which opens new horizons for recovery and gives patients better opportunities for a stable, healthy life.
She pointed out that the success of providing the first injections of advanced gene therapy for hemoglobinopathies at Yas Clinic Hospital reflects the department’s commitment to providing the highest levels of health care to members of society, and enhancing Abu Dhabi’s position as a leading global destination in the health care sector.
For her part, Dr. Maysoon Al Karam, Executive Director of Medical Affairs at Yas Clinic Hospital, explained that the partnership with leading international companies in the field of biotechnology, such as Vertex, represents an important strategic step that is consistent with the vision of the UAE and its national priorities in developing the health sector.
She explained that this cooperation contributes to providing advanced genetic treatments for patients with genetic diseases such as thalassemia and sickle cell anemia, noting that treatment based on CRISPR/Cas9 technology is a qualitative leap in the treatment of these conditions, and reflects the hospital’s keenness to provide the latest internationally approved medical solutions to improve the quality of life of patients.
For his part, Hisham Hajar, General Manager of Vertex in the Gulf Cooperation Council countries, stressed the importance of cooperation with Yas Clinic Hospital in providing the first treatment based on CRISPR-Cas9 technology in the UAE, pointing out that this achievement came with the support and supervision of the Department of Health – Abu Dhabi.
He pointed out that this partnership represents a pivotal step towards providing innovative, transformative treatments for patients suffering from diseases such as sickle cell anemia and blood transfusion-dependent thalassemia, which supports the company’s mission to improve the lives of patients in the country and the region.
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