World’s First ITVISMA Gene Therapy Given in Abu Dhabi

Abu Dhabi has achieved a historic milestone in global healthcare. For the first time in the world, ITVISMA gene therapy has been administered to a patient suffering from spinal muscular atrophy (SMA). The treatment marks a major leap in precision medicine and genomics-based healthcare.

What Is the News About?

• On December 28, 2025, Sheikh Khalifa Medical City successfully administered ITVISMA, a one-time gene therapy for SMA.
• The therapy was delivered under the supervision of the Department of Health, Abu Dhabi, following regulatory approval granted on November 25, 2025.
• With this step, the UAE became the second country after the United States to approve ITVISMA for clinical use.

About ITVISMA Gene Therapy

• ITVISMA is a next-generation gene therapy developed by Novartis.
• It directly targets the genetic cause of spinal muscular atrophy.

Key Features

• One time single dose gene therapy
• Replaces the defective SMN1 gene
• Enables production of Survival Motor Neuron (SMN) protein
• Approved for patients aged 2 years and above, including adults
• Expands access beyond infant-only SMA treatments

Understanding Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy is a rare genetic neuromuscular disorder.

It leads to,

• Progressive muscle weakness
• Loss of motor function
• Difficulty in breathing and swallowing
• Life-threatening complications in severe cases

SMA is caused by mutations in the SMN1 gene, which is essential for motor neuron survival.

Policy and Global Health Significance

• Positions Abu Dhabi as a leader in genomics and precision medicine
• Strengthens the UAE’s role in advanced rare disease care
• Demonstrates rapid regulatory and clinical capability

According to Dr. Noura Khamis Al Ghaithi, Undersecretary of the Department of Health, Abu Dhabi, the achievement reflects the emirate’s commitment to world-class healthcare delivery.

Cost and Accessibility Concerns

• The exact cost of ITVISMA has not been officially disclosed.
• However, it is expected to be comparable to Zolgensma, another SMA gene therapy priced at around $2 million per dose.

This highlights,

• Ongoing debates around affordability of rare disease therapies
• Need for innovative health financing models
• Importance of insurance and government support systems

Static Background: Gene Therapy in Medicine

Gene therapy involves inserting, altering, or replacing genes to treat disease.

It is increasingly used in,

• Rare genetic disorders
• Certain cancers
• Neuromuscular conditions

Gene therapies are considered a frontier area of modern medicine due to their potential for curative outcomes with a single intervention.

Key Data at a Glance

• Therapy Name: ITVISMA
• Disease targeted: Spinal Muscular Atrophy
• Developer: Novartis
• First administration: December 28, 2025
• First hospital globally: Sheikh Khalifa Medical City, Abu Dhabi
• UAE approval date: November 25, 2025
• Patient eligibility: 2 years and above

Question

Q. ITVISMA gene therapy targets which genetic defect?

A. DMD gene
B. BRCA gene
C. SMN1 gene
D. CFTR gene