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AAV-BEC
- Development stage
- Preclinical
- Lead developer
- University of California, San Francisco
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV-BEC is an engineered adeno-associated virus (AAV) capsid designed for efficient and specific gene delivery to biliary epithelial cells (BECs). Developed by researchers at the University of California, San Francisco (UCSF), AAV-BEC was identified by screening a peptide-display AAV capsid library in mouse livers. Unlike standard AAV capsids that exhibit tropism primarily for hepatocytes, AAV-BEC specifically targets naive, reactive, and malignant BECs. In preclinical models, AAV-BEC-mediated gene delivery has been utilized to restore biliary function in cholangiopathy (via ABCC12 delivery), modulate cholestasis (via CRISPR-mediated Rbpj knockout), and reduce tumor burden in cholangiocarcinoma (via diphtheria toxin-mediated cell ablation). It represents a promising platform for the development of targeted gene therapies for a broad range of biliary diseases.
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