CHAPERONE - Allosteric chaperones to treat rare lysosomal storage disorders

The consortium will deliver proof-of-concept (PoC) data for a novel class of therapeutic molecules to treat rare lysosomal storage disorders (LSD), for which no cure exists. A computational platform that identifies unique allosteric enzyme binding sites has generated novel compounds to restore the function of LSD-causing defects in enzymes. During the project the efficacy to restore enzyme function will be validated resulting in a data package including efficacy and mode-of-action.