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AAV9 gene therapy vector
- Development stage
- Unknown
- Lead developer
- Novartis
- Modality
- Gene Therapies
- Administration
- Intravenous, Intrathecal, Intramuscular
01
Overview
AAV9 (Adeno-Associated Virus serotype 9) is a recombinant viral vector platform widely utilized in gene therapy for its unique ability to cross the blood-brain barrier and efficiently transduce cells within the central nervous system, including motor neurons and astrocytes. Characterized by low immunogenicity and nonpathogenicity, AAV9 serves as a delivery vehicle for therapeutic transgenes in various genetic disorders. The most prominent application is the FDA-approved Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy. Numerous other investigational programs leverage AAV9 to treat lysosomal storage disorders, neuromuscular diseases, and metabolic conditions such as GM1 gangliosidosis, propionic acidemia, and Duchenne muscular dystrophy.
- Other names
- AAV9AAV-9AAV 9Adeno-associated virus serotype 9rAAV9rAAV-9rAAV 9
02
Targets
Terminal galactose-containing cell-surface glycans
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