AAVHSC16

AAVHSC16 is a naturally occurring adeno-associated virus (AAV) capsid belonging to clade F, originally isolated from human hematopoietic stem cells (HSCs). Developed by Homology Medicines, it serves as a gene therapy vector designed for systemic delivery with a distinct biodistribution profile. Unlike many other AAV vectors, AAVHSC16 exhibits significantly reduced liver tropism, which is linked to unique amino acid residues at positions 501I and 706C and a lack of binding to terminal galactose. Preclinical data in non-human primates indicate that intravenous administration of AAVHSC16 leads to robust transduction of the central nervous system (CNS), heart, and skeletal muscle without the liver enzyme elevations typically associated with high-dose AAV administration. This makes it a specialized tool for treating genetic diseases affecting the brain and peripheral organs while minimizing off-target hepatic effects.