Broadening applications for intrathecal gene therapy: a case for lysosomal storage diseases
Abstract
On the Nov 24, 2025, the USA Food and Drug Administration (FDA) approved Itvisma, supported by a pair of clinical trials published in Nature Medicine on Dec 8, 2025 (STEER and STRENGTH). Itvisma joins Zolgensma (previously labelled as the world's most expensive medicine) as a gene therapy for patients with spinal muscular atrophy, a progressive neuromuscular disease caused by mutations in a single gene (SMN1). Both Itvisma and Zolgensma comprise an adeno-associated virus serotype 9 (AAV9) capsid containing a corrected copy of the SMN1 gene, but differ primarily in the route of administration.
Fields of Study & Subjects
Virus-based gene therapy research
Lysosomal Storage Disorders Research
Neurogenetic and Muscular Disorders Research
Publication details
- Journal / source
- EBioMedicine
- Volume
- 123
- Pages
- 106129–106129
- Publication date
- 1 January 2026
- Language
- EN