World’s First ITVISMA Gene Therapy Administered in Abu Dhabi

Historic First in Global Gene Therapy

Abu Dhabi has achieved a landmark breakthrough in global healthcare by becoming the first place in the world to administer ITVISMA , a one-time gene therapy for spinal muscular atrophy (SMA) . The treatment was successfully delivered at Sheikh Khalifa Medical City on December 28, 2025 , positioning the UAE at the forefront of genomics-based precision medicine.

Regulatory Approval and Global Significance

The therapy was administered under the supervision of the Department of Health, Abu Dhabi , following regulatory approval granted on November 25, 2025 . With this approval, the UAE became the second country after the United States to authorise ITVISMA for clinical use. The first successful administration underscores Abu Dhabi’s capacity for rapid translation of advanced biomedical innovation into patient care.

What Is ITVISMA Gene Therapy?

Developed by Novartis , ITVISMA is a next-generation gene therapy designed to address the root genetic cause of SMA. It works by replacing the defective SMN1 gene , enabling the body to produce the Survival Motor Neuron (SMN) protein , which is critical for motor neuron function. Unlike earlier therapies limited to infants, ITVISMA is approved for patients aged two years and above , including adults.

Understanding Spinal Muscular Atrophy

SMA is a rare inherited neuromuscular disorder characterised by progressive muscle weakness, loss of mobility, and, in severe cases, respiratory failure. It arises from mutations in the SMN1 gene, leading to degeneration of motor neurons. Gene therapy offers a potential curative, single-dose intervention , representing a paradigm shift from lifelong treatment.

Cost and Access Considerations

Although pricing has not been officially disclosed, ITVISMA is expected to be comparable to Zolgensma , which costs around $2 million per dose . This raises important debates on affordability, insurance coverage, and sustainable financing for rare disease therapies worldwide.

Important Facts for Exams

• ITVISMA is a one-time gene therapy for SMA

• It replaces the defective SMN1 gene

• First administered globally in Abu Dhabi

• Developed by Novartis

• Approved in UAE in November 2025