Pharmacological chaperones, Chemical chaperones , Enzyme e n- hancement therapy, GM1, Gangliosidosis, Morqui B, Lysosomal Storage Disease, Lysosomal Storage Disorders

Most Pharmacological chaperones (PC’s) described until now are substrate analogues which bind to the active site of the target protein. C ons e- quently, such PC’s also inhibit the target protein at higher concentrations thus rendering a narrow therapeutic window and have poor drug-like properties. Through our proprietary technology platform SEE-Tx™, we identify a new generation of non-substrate competitive pharmacological chaperones which p o- tentially offer a much broader therapeutic window. What’s more, such co m- pounds are not substrate analogues, thus presenting much better drug-like pro p- erties, particularly for indications with CNS involvement. Here we present our methodology to identify non-competitive pharmacological chaperones applied to the enzyme beta-galactosidase, whose deficiency is related with GM1 Gangliosidosis and Morquio B.