DoH introduces first CRISPR gene-editing therapy for genetic blood disorders

UAE— The Department of Health – Abu Dhabi (DoH), the healthcare regulator in the emirate, has announced the launch of CASGEVY, the UAE’s first CRISPR/Cas9 gene-editing therapy.

This groundbreaking development marks a significant milestone in the field of genetic medicine and innovation, offering advanced treatment options for patients suffering from sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).

By launching this therapy, Abu Dhabi strengthens its position as a global hub for life sciences and medical advancements.

The innovative treatment will be provided by the Abu Dhabi Stem Cells Center (ADSCC) in collaboration with Vertex Pharmaceuticals, a leading biotechnology company.

The first patient is set to begin therapy at Yas Clinic Hospital in April.

CASGEVY employs CRISPR/Cas9 technology to precisely edit specific DNA sequences, enabling targeted treatment for genetic disorders.

This one-time therapy involves extracting stem cells from the patient’s bone marrow, modifying them in a laboratory setting, and reintroducing them into the patient’s body to deliver long-term benefits.

To ensure safe and effective implementation, DoH has established comprehensive regulations and guidelines for managing gene therapies in line with international standards.

Additionally, the treatment has been included in insurance coverage to enhance accessibility for eligible patients.

Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, emphasized the department’s commitment to improving community health and advancing healthcare quality through innovation.

She highlighted that adopting cutting-edge technologies like genomic sciences helps address complex health challenges while positioning Abu Dhabi as a leader in healthcare and life sciences globally.

Adding to this, Dr. Maysoon Al Karam, Chief Medical Officer at ADSCC and Yas Clinic, noted that partnerships with leading biotechnology firms such as Vertex Pharmaceuticals represent a significant step forward for ADSCC’s mission.

She explained that these collaborations align with the UAE’s strategic health priorities and enable the development of advanced treatments for genetic disorders like SCD and TDT.

As a FACT-accredited center, ADSCC aims to drive medical innovation and reinforce Abu Dhabi’s status as a global hub for life sciences.

For his part, Hisham Hagar, Senior Country Manager at Vertex GCC, expressed enthusiasm about partnering with ADSCC to bring transformative medicines like CASGEVY to UAE patients.

He stated that this collaboration underscores Vertex’s mission to improve lives by addressing serious diseases through innovative therapies.

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

This approval paves the way for wider access to revolutionary gene-editing treatments in the UAE.

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