AAVHSC17

AAVHSC17 is a recombinant adeno-associated virus (AAV) vector derived from human hematopoietic stem cells (HSCs), belonging to the Clade F family of AAVHSC vectors. Developed by Homology Medicines, Inc., it is part of a proprietary platform designed for gene therapy and gene editing applications. Preclinical research in nonhuman primates has demonstrated that AAVHSC17 possesses broad tissue tropism and the ability to cross the blood-brain barrier following intravenous administration. It effectively transduces various tissues, including the central nervous system (neurons and glial cells), peripheral nervous system, liver, skeletal muscle, heart, kidneys, and pancreatic acinar cells. Notably, AAVHSC17 shares the same amino acid sequence as AAVHSC13. It is currently being investigated for potential therapeutic use in treating genetic diseases affecting the CNS and systemic organs.