AM805

AM805 is an investigational AAV9-based gene therapy developed by Amlogenyx for the treatment of Alzheimer's disease. It is designed to deliver the gene encoding protective protein cathepsin A (PPCA), a lysosomal enzyme, to the central nervous system. Unlike monoclonal antibodies that bind to amyloid, AM805 uses a catalytic approach to degrade amyloid-beta both intracellularly and in extracellular plaques. Preclinical studies have demonstrated significant reductions in amyloid burden in mouse models, and the program is currently in IND-enabling stages with a planned IND submission in 2027.

Other names

AAV9-PPCAAAV-9-PPCAAAV 9-PPCA