AAV8-hARSB

AAV8-hARSB is an investigational adeno-associated virus (AAV) serotype 8-based gene therapy designed for the treatment of mucopolysaccharidosis type VI (MPS VI), also known as Maroteaux-Lamy syndrome. Developed by Fondazione Telethon, the vector consists of a single-stranded AAV2 genome encoding the human arylsulfatase B (ARSB) gene under the control of the liver-specific thyroxine-binding globulin (TBG) promoter. By targeting the liver, the therapy aims to turn the organ into a factory for the production and secretion of the ARSB enzyme into the bloodstream, thereby reducing the systemic accumulation of glycosaminoglycans (GAGs) like dermatan sulfate. It is administered as a single intravenous infusion and has been evaluated in Phase I/II clinical trials to address the underlying enzyme deficiency that causes progressive organ damage in MPS VI patients.

Other names

AAV8-ARSBAAV-8-ARSBAAV 8-ARSB