ITVISMA Gene Therapy

News: Abu Dhabi’s Sheikh Khalifa Medical City became the first hospital to administer ITVISMA after UAE regulatory approval in November 2025.

About ITVISMA Gene Therapy

• “Itvisma” is a brand name for the gene therapy drug onasemnogene abeparvovec-brve.
• It  is a one-time gene therapy approved in late 2025 for the treatment of spinal muscular atrophy (SMA) in adults and children aged 2 years and older.
• The therapy addresses the genetic cause of the disease rather than only managing symptoms.
• Developed by: ITVISMA was developed by Novartis.
• Aim: The treatment aims to improve motor function and reduce long-term treatment dependence.
• Mechanism
  • SMN1 Gene: This is the gene that is mutated or missing in people with spinal muscular atrophy (SMA), the condition Itvisma treats.
  • The gene’s role is to produce the essential SMN protein needed for motor neuron function, muscle movement, breathing, and swallowing.
  • ITVISMA’s role: It is an adeno-associated virus (AAV) vector-based therapy designed to deliver a new, working copy of the SMN1 gene directly to the motor neurons in the spinal cord via a one-time intrathecal injection.
  • Result: The new gene enables continuous and sustained production of the SMN protein, addressing the genetic root cause of SMA to improve or stabilize motor function.

• Risk of Therapy
  • Common Side Effects: Common side effects include fever, vomiting, headache, and upper respiratory tract infections.
  • Serious Risks: Serious risks include liver toxicity, heart-related effects, and reduced platelet counts.
• Significance : ITVISMA can deliver sustained motor-function improvement with a single dose, reduce lifelong treatment dependence, and expand access beyond infant-only therapies.