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AAV-KP1-CAG-human HNF1B
- Development stage
- Preclinical
- Lead developer
- Boston Children's Hospital
- Modality
- Gene Therapies
- Administration
- Parenteral
01
Overview
AAV-KP1-CAG-human HNF1B is an adeno-associated virus (AAV)-based gene replacement therapy designed to deliver a wild-type human HNF1B transgene under the control of a CAG promoter. It utilizes the engineered AAV-KP1 capsid, which exhibits high tropism for kidney tubular epithelial cells. Developed by researchers at Boston Children's Hospital and Harvard Medical School, the therapy is being investigated for the treatment of HNF1B-associated diseases, including cystic kidney disease and congenital anomalies of the kidneys and urinary tract (CAKUT). In preclinical mouse models of Hnf1b knockout, direct medullary injection of the vector resulted in efficient transduction of renal epithelial cells and a significant reduction in cystic burden.
- Other names
- AAV-KP1-CAG-humanHNF1BAAV-KP-1-CAG-humanHNF1BAAV-KP 1-CAG-humanHNF1B
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