Abu Dhabi Introduces CASGEVY: First Gene-Editing Therapy

Post by: Bandan Preet

Photo: WAM

The Department of Health – Abu Dhabi (DoH) has introduced a groundbreaking treatment called CASGEVY, the first CRISPR/Cas9 gene-editing therapy in the UAE. This advanced treatment offers new hope for people suffering from sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). With this major step, Abu Dhabi is strengthening its position as a leader in medical innovation and life sciences.

Collaboration and Treatment Details

The Abu Dhabi Stem Cells Center (ADSCC) will provide this advanced treatment in partnership with DoH and Vertex Pharmaceuticals, a well-known biotechnology company. The first patient will receive the therapy at Yas Clinic Hospital in April.

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How the CRISPR/Cas9 Gene-Editing Therapy Works

The CRISPR/Cas9 technology is a cutting-edge method that allows scientists to edit genes by changing specific parts of DNA. This therapy is a one-time treatment that is specially designed for each patient. Here’s how it works:

• Stem cells are taken from the patient’s bone marrow.

• These cells are genetically edited in a lab to fix the faulty DNA.

• The edited cells are put back into the patient’s body.

• Over time, the patient may experience long-term improvements in their condition.

Ensuring Safety and Accessibility

The Department of Health has created strict guidelines and regulations to ensure the treatment is safe and effective. The UAE government has also made sure that insurance will cover the therapy, making it affordable for eligible patients.

Statements from Key Experts

• Dr. Noura Khamis Al Ghaithi, Undersecretary of DoH, said this treatment is part of Abu Dhabi’s mission to improve healthcare quality and make the city a global leader in medical innovation.

• Dr. Maysoon Al Karam, Chief Medical Officer at ADSCC and Yas Clinic, highlighted the importance of collaborating with top biotech companies like Vertex Pharmaceuticals. She believes this partnership will help patients with genetic disorders such as sickle cell disease and thalassemia.

• Hisham Hagar, Senior Country Manager at Vertex GCC, is excited about bringing this new therapy to the UAE. He says this step will improve patients' lives and accelerate medical progress.

Approval and Age Requirements

The Emirates Drug Establishment (EDE) has officially approved CASGEVY for use in the UAE. The therapy is available for patients aged 12 years and older who have:

• Sickle cell disease with repeated pain episodes caused by blocked blood flow (vaso-occlusive crises).

• Transfusion-dependent beta-thalassemia, where patients rely on regular blood transfusions.

This new therapy is a huge leap forward in healthcare, giving patients with serious genetic blood disorders a chance at a healthier and better life.

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