YM-01 is an allosteric inhibitor of Hsp70 that binds within its nucleotide-binding domain, adjacent to the ATP/ADP pocket. It disrupts Hsc70-Bag1 interactions and enhances Hsp70 binding to misfolded proteins. In neuroscience, YM-01 has shown efficacy in reducing tau protein levels in cellular and animal models of tauopathy, including Alzheimer's disease. It restores long-term potentiation in brain slices from tau-overexpressing mice, indicating potential for cognitive recovery. YM-01's dual activity in protein quality control and synaptic function makes it a valuable tool for studying and potentially treating tau-related neurodegenerative disorders.
