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AAV.AQP4
- Development stage
- Preclinical
- Lead developer
- University of Florida
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV.AQP4 is an experimental adeno-associated viral (AAV) gene immunotherapy designed for the treatment of neuromyelitis optica (NMO) and related neuroinflammatory disorders. Developed by researchers at the University of Florida, the therapy utilizes an AAV vector to deliver the gene encoding aquaporin-4 (AQP4), or specific immunogenic epitopes thereof, to the liver. This liver-directed expression is intended to induce peripheral immune tolerance by promoting the expansion of antigen-specific regulatory T cells (Tregs). These Tregs then suppress the autoimmune response against AQP4-expressing astrocytes in the central nervous system, thereby preventing neuroinflammation and demyelination. Preclinical studies in mouse models have shown that AAV.AQP4 can effectively prevent the development of AQP4-mediated neurological disability and focal inflammation in the spinal cord.
- Other names
- AAV-AQP4AAV-AQP-4AAV-AQP 4AAV-mediated AQP4 gene immunotherapy
02
Targets
AQP4-MHC-TCR (Aquaporin-4 (AQP4) antigenic epitopes presented on Major Histocompatibility Complex (MHC) to T-cell receptors (TCR))
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