Gene therapy in the UAE treated 370 patients worldwide with rare diseases over six years
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Khaleej Times
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Gene therapy in the UAE treated 370 patients worldwide with rare diseases over six years

Over 370 international patients with advanced gene therapy treatments were provided in Abu Dhabi between 2020 and 2026. It is estimated that the cost of these medications for international patients reached 3.3 billion dirhams.

These data, revealed for the first time, were presented on October 20 at the Abu Dhabi Health Future Summit. The presentation was given by Dr. Fatima Al Kaabi, Executive Director of the Abu Dhabi Bone Marrow Transplant Program at ADSCC and Yas Clinic.

She reported that over a six-year period, approximately 400 patients received advanced gene therapy in the UAE. Among the conditions treated were sickle cell anemia, thalassemia, spinal muscular atrophy, and Duchenne muscular dystrophy. Global partners such as Roche and Novartis participated in the project.

Dr. Al Kaabi noted that previously, patients were forced to travel the world in search of hope, but today they are coming to the UAE to find it. One factor contributing to this shift is the CRISPR gene editing program for hereditary blood disorders. This therapy involves collecting the patient's own stem cells, correcting the defective gene in the laboratory, and returning the repaired cells to the body.

Al Kaabi emphasized that the process is more complex than it seems, calling it an 'exceptional medical journey,' which includes assessment, stem cell mobilization, gene editing, conditioning chemotherapy, implantation, recovery, and long-term monitoring. She added that a complete healthcare ecosystem is required for safe gene therapy, which they are creating in Abu Dhabi.

Currently, 18 patients aged 12 to 43 have been treated under this program, and 29 patients have been approved and are awaiting treatment. All patients achieved stable blood cell production within 100 days, and none required blood transfusions. Notably, 42 percent of those treated were over 35 years old, exceeding the age range studied in the initial clinical trials.

Dr. Al Kaabi also shared the story of Fahad, a 29-year-old UAE national who spent his life moving between hospitals and receiving blood transfusions after birth due to sickle cell anemia and G6PD deficiency. She urged people to remember families, not dirhams.

The Minister of the State Council, Sheikh Shahbout bin Nahyan Al Nahyan, stated in his keynote address that the UAE Genome Program has collected nearly one million genetic samples linked to over 3,000 medical institutions and more than 3.5 million clinical records. He noted that the question before them is not whether the innovation works, but whether they can ensure its scaling for everyone, especially for those most vulnerable.

The Abu Dhabi Health Future Summit continues until Thursday, October 22, under the patronage of the Crown Prince of Abu Dhabi, Sheikh Khalid bin Mohammed bin Zayed Al Nahyan, with scheduled sessions on cancer treatment, digital health, and healthy living.

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