Tau Pharmacological Chaperone

Small molecule pharmacological chaperones targeting the Tau protein

CB301 for Alzheimer’s Disease

Preventing, reducing and/or inhibiting the aggregation of Tau protein is a promising therapeutic strategy against Alzheimer's disease (AD) and Tauopathies in general. The application of small molecules to reduce and inhibit the aggregation process of Tau has high potential as a therapeutic approach through the prevention of the formation of toxic Tau oligomers and aggregates. Therapeutic targeting of the monomeric state of Tau by small molecules has to date been a major challenge because of their range of structural forms. Despite this, through the application of an innovative and unique drug discovery approach1 we have been successful in identifying novel small molecules that bind to monomeric Tau. Developed in conjunction with the University of Cambridge and the Max Planck Institute, Cantabio's Tau targeting drug candidates are small molecule pharmacological chaperones which bind to the native monomeric Tau protein and reduce its aggregation in vitro and in cells. Cantabio is currently studying and further developing these Tau targeting drug candidates in relevant AD in vivo models with the aim of producing clinical candidates for the treatment of Alzheimer’s.

Theoretical representation of the interaction between small molecule pharmacological chaperone and Tau

1 (Pickhard et. al. ‘Identification of Small Molecule Inhibitors of Tau Aggregation by Targeting Monomeric Tau As a Potential Therapeutic Approach for Tauopathies’ Current Alzheimer Research, 2015, 12, 814-828)