Abu Dhabi Builds Advanced Gene-Therapy Ecosystem, Brings International Practices To UAE

For decades, patients with certain inherited blood disorders had few options beyond lifelong treatment, repeated hospital visits and, in some cases, the search for a suitable stem-cell donor. That is beginning to change in Abu Dhabi, where gene and cell therapies are moving from the realm of scientific promise into clinical practice.

At Yas Clinics, doctors are now treating patients using advanced cellular and gene-based approaches, as part of a broader effort to establish the infrastructure and expertise needed to deliver some of medicine's most complex treatments locally.

The programme sits within a wider ecosystem that includes bone-marrow transplantation, CAR-T cell therapy and gene therapy.

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Yas Clinics Chief Executive Officer Mubasher Maqbool said the centre has performed more than 260 bone-marrow transplant cases, including autologous and matched-unrelated donor transplants. He said around 92 per cent of patients who would previously have travelled to the United States for treatment are now being treated locally.

The clinic has also collected cells from around 29 patients for CAR-T treatment, while the number of gene-therapy patients remains smaller as the service is newer.

But the significance of gene therapy goes beyond the number of patients treated.

Unlike conventional medicines, some of these therapies involve taking a patient's own cells, modifying them in a laboratory and returning them to the body in the hope of correcting the underlying biological problem.

Dr Mansi Sachdev, consultant paediatric haematology and bone marrow transplant, described gene therapy as a particularly important development for inherited blood disorders such as transfusion-dependent thalassemia and sickle cell disease.

For many patients with thalassemia who require a transplant, finding a suitable donor can be a major obstacle. A fully matched family donor is not available for many patients.

Gene therapy offers another route: the patient's own stem cells can be collected, genetically modified and then returned to the patient.

The same broader revolution is visible in CAR-T therapy, in which a patient's immune cells can be genetically engineered to recognise and attack cancer cells.

Dr Sachdev pointed to the case of a child with blood cancer who had been diagnosed at the age of four and had undergone treatment after treatment for years without eliminating the disease. After his cells were engineered for CAR-T therapy and returned to him, she said, it was the first time his leukemia went into remission.

The therapies, however, are far from simple.

During Abu Dhabi's Future Health Summit, Dr Fatima Al Kaabi stressed that gene editing is not simply a matter of giving a patient an injection.

The process can involve assessment, preparation and mobilisation of stem cells, collection, gene editing and cell processing, extensive quality checks, chemotherapy conditioning, infusion of the modified cells, engraftment and long-term follow-up.

One of the patients whose story she shared was Fahd Al Numani, a 29-year-old patient whose case illustrated the journey involved in gene-editing treatment.

Al Kaabi described the treatment as an extraordinary medical journey rather than a single procedure, requiring an entire healthcare ecosystem to support it.

That ecosystem is now producing early results.

According to figures presented by Al Kaabi, 29 patients had been approved for treatment, with 19 having undergone cell collection and 18 having received their modified cells. Of those collected, 11 had thalassemia and eight had sickle cell disease.

She reported 100 per cent results for several early measures, including engraftment and the haematological outcomes presented at the event, while stressing that these were early indicators and that long-term monitoring remains essential.

One particularly notable aspect of Abu Dhabi's experience is the age of some of the patients.

Al Kaabi said 42 per cent of the collected patients were older than 35, even though the original pivotal study had included patients aged between 12 and 35. The Abu Dhabi experience is therefore also contributing real-world evidence on the treatment's use in an older population.

The development comes with a significant financial challenge.

Maqbool estimated the cost of a gene-therapy treatment at around $300,000 per patient and said the treatment is not generally covered by insurance for expatriate patients, leaving affordability as a major barrier to access.

That raises a central question for the next phase of gene therapy in the UAE: not simply whether the technology can be delivered, but how widely it can eventually be made available.

The clinic's leadership says the future extends beyond the therapies currently being offered. Areas under development include regenerative medicine, longevity and healthier ageing, alongside the expansion of gene therapies to additional diseases.

Dr Sachdev said the next generation of applications could include other single-gene disorders, including certain immunodeficiencies and neurological conditions, if appropriate gene therapies are developed and approved.

For the healthcare system, the implications extend beyond individual treatments.

The CEO said advances in pathology, radiology, artificial intelligence and big-data systems are increasingly helping clinicians make faster and more informed decisions. The larger challenge, he said, is bringing those streams of information together so doctors can understand the complete picture of a patient's health.

For Abu Dhabi, the ambition is therefore larger than offering a handful of highly specialised treatments.

It is about creating the infrastructure, expertise and evidence base needed to make advanced therapies part of the healthcare system.

Al Kaabi said the UAE is increasingly becoming a destination for patients seeking advanced medical innovation, including patients travelling from abroad.

The transformation of gene therapy from an experimental concept into a clinical service is still in its early stages. The treatments remain complex, expensive and demanding, and long-term outcomes will need to be tracked.

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