ABU DHABI — December 29, 2025— The United Arab Emirates has solidified its position as a global pioneer in genomic medicine by becoming the first country in the world to successfully administer ITVISMA, a groundbreaking AAV-mediated gene replacement therapy, for the treatment of spinal muscular atrophy (SMA). This landmark medical achievement, carried out at Sheikh Khalifa Medical City (SKMC), marks the UAE as the second nation globally to approve this specific AAV viral vector treatment, following only the United States.
Harnessing AAV Technology for Genetic Cures
Developed by Novartis, ITVISMA (onasemnogene abeparvovec) utilizes a specialized adeno-associated virus (AAV) vector to deliver a functional copy of the SMN1 gene directly into a patient’s cells. By leveraging the precision of the AAV delivery system, the therapy addresses the root genetic cause of SMA, a condition that was previously considered untreatable. This AAV gene therapy is designed for a wide patient demographic, including adults and children aged two and older, providing a vital one-time AAV dosing option for those who were previously ineligible for earlier gene treatments.
A Precision Healthcare Milestone Driven by AAV
The Emirates Drug Establishment (EDE) granted accelerated approval for the AAV therapy on November 25, 2025, emphasizing the safety and efficacy demonstrated by the AAV viral vector in clinical trials.
“This milestone reflects Abu Dhabi’s commitment to delivering world-class care through AAV-driven precision medicine,” said Dr. Noura Khamis Al Ghaithi, Under-Secretary of the Department of Health – Abu Dhabi. “By administering this AAV gene therapy, we are reinforcing our role as a leader and accelerator in advanced healthcare.”
Expanding the AAV Therapeutic Landscape
The successful rollout of this AAV treatment is part of a broader strategy to establish the UAE as a regional hub for AAV and biotechnology innovation. The UAE’s healthcare ecosystem is now set to expand its AAV pipeline, with ongoing initiatives to integrate AAV therapies for other rare genetic diseases. Through strategic partnerships and a robust regulatory model, the UAE continues to streamline the delivery of life-changing AAV therapies, ensuring that patients across the region have immediate access to the latest breakthroughs in AAV gene-editing technology.
Source:
https://www.msn.com/en-ae/news/other/abu-dhabi-leads-world-in-breakthrough-gene-replacement-therapy-itvisma-for-spinal-muscular-atrophy/ar-AA1TagOR
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