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This cutting-edge approach represents a major advancement in how doctors can treat genetic diseases by directly modifying the faulty genes responsible for these conditions.

UAE—The United Arab Emirates has achieved a significant breakthrough in advanced medical treatment with the successful administration of the country’s first gene-therapy injection for blood disorders.
Yas Clinic in Khalifa City led this groundbreaking procedure in partnership with the Abu Dhabi Stem Cells Centre and Vertex Pharmaceuticals, working under the guidance and supervision of the Department of Health in Abu Dhabi.
The treatment involves CASGEVY, a revolutionary therapy that uses CRISPR-Cas9 gene-editing technology to address hemoglobinopathies, a group of inherited blood disorders.
This cutting-edge approach represents a major advancement in how doctors can treat genetic diseases by directly modifying the faulty genes responsible for these conditions.
The CRISPR-Cas9 technology works through a remarkably precise process that targets and corrects specific DNA sequences causing disease.
The system employs guide RNA, which functions like a genetic GPS to locate the defective gene within a patient’s DNA.
Once the problematic section is identified, the Cas9 enzyme steps in to perform its role as molecular scissors, cutting the DNA at exactly the right location.
This precise cut allows medical professionals to correct the underlying genetic mutation that causes the blood disorder.
CASGEVY offers hope specifically for patients aged 12 years and older who struggle with sickle cell disease and experience recurrent vaso-occlusive crises, which are painful episodes caused by blocked blood flow.
The therapy also benefits those living with transfusion-dependent beta thalassemia, a condition that requires patients to receive regular blood transfusions throughout their lives.
Dr. Noura Khamis Al Ghaithi, who serves as undersecretary of the Department of Health in Abu Dhabi, expressed pride in the emirate’s continued efforts to develop one of the most advanced, intelligent, and efficient healthcare systems worldwide.
The achievement reflects Abu Dhabi’s commitment to adopting cutting-edge technologies and implementing the latest gene-based therapies for its population.
These medical innovations create new pathways for patient recovery and provide individuals suffering from genetic conditions with renewed hope for an improved quality of life.
Dr. Maysoon Al Karam, serving as Chief Medical Officer at Yas Clinic, stressed the profound importance of this achievement for both patients and the medical community.
She noted that this development enables access to potentially life-changing solutions for individuals who have long struggled with limited treatment options.
Beyond its impact on patients, this milestone reinforces Abu Dhabi’s emerging position as a global hub for advanced therapies and life sciences research.
Hisham Hagar, who holds the position of Executive Country Manager at Vertex GCC, emphasized the critical nature of the collaboration between his company and Yas Clinic in Khalifa City.
This partnership has successfully brought an innovative therapy to the UAE that offers patients a potential functional cure.
For individuals who have endured years with few effective treatment alternatives, this advancement represents a transformative opportunity to manage their condition in ways previously unavailable in the region.
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