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AAVKP1
- Development stage
- Preclinical
- Lead developer
- Stanford University
- Modality
- Gene Therapies
- Administration
- Intravenous, Intra-arterial, Parenteral
01
Overview
AAVKP1 (also known as AAV-KP1) is a bioengineered, chimeric adeno-associated virus (AAV) capsid developed through DNA family shuffling of multiple parental AAV serotypes (including AAV1, AAV2, AAV3b, AAV6, AAV8, AAV9, AAV-rh.10, and AAV-porcine-2). Originally selected for its high transduction efficiency in human pancreatic islet cells and embryonic stem cell-derived beta cells, AAVKP1 has also demonstrated robust transduction of human and murine hepatocytes, cochlear cells, and renal proximal tubule cells. In the kidney, local administration of AAVKP1 via the renal vein or renal pelvis enables efficient gene transfer to proximal tubules while minimizing off-target liver transduction and bypassing systemic clearance barriers. AAVKP1 binds to cell-surface heparan sulfate proteoglycans (HSPGs) for attachment and utilizes the adeno-associated virus receptor (AAVR/KIAA0319L) for functional cellular entry.
- Other names
- AAV-KP1 capsidAAV-KP-1 capsidAAV-KP 1 capsidKP1 capsidKP-1 capsidKP 1 capsid
02
Targets
HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))
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