$2 Million Gene Therapy Offers Hope for Rare Blood Disorder with CRISPR Breakthrough
June 12, 2025
Khalifa Dawood, a 22-year-old Emirati man, has become one of the first patients globally to receive a groundbreaking $2 million gene therapy aimed at treating his rare blood disorder.
The innovative therapy, known as Casgevy, was administered at Cleveland Clinic Children's Hospital in Ohio, which recently secured medical approval for this treatment.
Casgevy utilizes CRISPR gene editing technology to modify the patient's blood-forming cells, offering a one-time solution that is designed to last a lifetime.
The treatment process involves collecting the patient's stem cells, editing them in a laboratory, and then reintroducing them after chemotherapy is used to eliminate unhealthy cells.
Following its FDA approval in January 2025, Casgevy has demonstrated promising outcomes, with 91% of beta thalassaemia patients not requiring blood transfusions for at least a year post-treatment.
Dr. Rabi Hanna from Cleveland Clinic emphasized that while the treatment comes at a high cost, it marks a significant advancement in medical care for patients like Dawood who endure lifelong conditions.
Despite its success, the steep price of Casgevy and similar therapies often restricts access, although some financial support options are available through insurers and charitable organizations.
Thalassaemia, the disorder affecting Dawood, is notably prevalent in the UAE, impacting an estimated 16.5% of the population and leading to serious symptoms such as bone deformities and organ damage.
This case highlights a growing trend in the Gulf region, where hospitals in the UAE and Saudi Arabia are emerging as leaders in advanced medical treatments, particularly in clinical trials for genetic disorders.
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The National • Jun 12, 2025
Emirati man with rare blood disorder benefits from new $2 million gene therapy in the US