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AVB-101
- Development stage
- Unknown
- Lead developer
- AviadoBio
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intrathalamic
01
Overview
AVB-101 is an investigational gene therapy designed to treat frontotemporal dementia (FTD) caused by mutations in the progranulin (GRN) gene. It uses a recombinant adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the human GRN gene directly into the brain via intrathalamic administration. The therapy aims to restore normal levels of progranulin in neurons, thereby addressing lysosomal dysfunction and neurodegeneration associated with FTD-GRN. Developed as a one-time treatment, AVB-101 is administered through a minimally invasive neurosurgical procedure and is currently being evaluated in Phase 1/2 clinical trials. The drug has received Fast Track and Orphan Drug designations from regulatory agencies in both the United States and Europe[1][2][3][4][5][7].
- Other names
- AAV.PGRN
02
Targets
GRN (Progranulin)
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