DoH launches CRISPR gene therapy for sickle cell and thalassemia

Sharjah 24 – WAM: The Department of Health – Abu Dhabi (DoH) has announced the introduction of CASGEVY, the first CRISPR/Cas9 gene-editing therapy in the UAE. This groundbreaking treatment offers innovative solutions for patients suffering from sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), establishing Abu Dhabi as a leading destination for life sciences and medical innovation.

Collaboration and treatment overview

The innovative treatment is being provided by Abu Dhabi Stem Cells Center (ADSCC), in coordination with DoH and in collaboration with Vertex Pharmaceuticals, a leading biotechnology company. The first patient is scheduled to begin the therapy at Yas Clinic Hospital in April.

CRISPR/Cas9 gene-editing technology

CRISPR/Cas9 gene-editing is an advanced technology that allows for precise genome editing to treat genetic diseases by altering specific DNA sequences. CASGEVY is a one-time treatment tailored to each patient, involving the extraction of stem cells from the patient's bone marrow, genetic editing in the lab, and re-implanting the edited cells into the patient's body for long-term positive effects.

DoH's commitment to innovation and safety

In line with its commitment to innovation, DoH has established guidelines and regulations for the management of gene therapies, adhering to the highest international standards. The treatment has been included in insurance coverage, ensuring accessibility for eligible patients.

Statements from key figures

Dr Noura Khamis Al Ghaithi, Undersecretary of DoH, emphasized the department's dedication to enhancing healthcare quality and solidifying Abu Dhabi's position as a global leader in life sciences and innovation.

Dr Maysoon Al Karam, Chief Medical Officer at ADSCC and Yas Clinic, highlighted the significance of partnerships with leading biotechnology companies like Vertex Pharmaceuticals, noting the positive impact the treatment will have on patients with genetic disorders such as sickle cell disease and thalassemia.

Hisham Hagar, Senior Country Manager at Vertex GCC, expressed excitement about the collaboration with ADSCC to bring the CRISPR/Cas9 gene-editing therapy to the UAE, aiming to improve patients’ lives and advance medical innovation.

Regulatory approval and market authorisation

The Emirates Drug Establishment (EDE) in the UAE has granted market authorization for CASGEVY to treat patients aged 12 years and older who suffer from sickle cell disease with recurrent vaso-occlusive crises and transfusion-dependent thalassemia.