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Drug intelligence / Profile preview
AAV-BEC-mAbcc12
- Development stage
- Preclinical
- Lead developer
- Cincinnati Children's Hospital Medical Center
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV-BEC-mAbcc12 is a preclinical adeno-associated virus (AAV) gene therapy designed to target biliary epithelial cells (BECs) and deliver a functional copy of the ABCC12 gene (encoding multidrug resistance-associated protein 9, or MRP9). Developed by researchers at Cincinnati Children's Hospital Medical Center and the University of California, San Francisco (UCSF), the therapy utilizes a novel engineered AAV capsid (AAV-BEC) with high tropism for cholangiocytes. In preclinical models of ABCC12 deficiency and sclerosing cholangitis, AAV-BEC-mAbcc12 restores mitochondrial function, reduces cholangiocyte apoptosis and ferroptosis, lowers serum alkaline phosphatase (ALP) levels, and promotes the formation of well-formed bile ducts. It is being investigated for the treatment of inherited cholangiopathies and cholestatic liver diseases.
- Other names
- AAV-BEC-ABCC12AAV-BEC-ABCC-12AAV-BEC-ABCC 12
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