DJ-1 Pharmacological Chaperones
Small molecule pharmacological chaperones targeting the DJ-1 protein
CB101 for Parkinson’s Disease
Using our pharmacological chaperone drug discovery strategy, dozens of novel small molecule scaffolds have been identified that bind to DJ-1. One example of our novel small molecule binder to DJ-1 protein is illustrated below:
From among our novel DJ-1 binding compounds, selected compounds from our CB101 program were shown to have a therapeutic effect in primary neuronal models of and in vivo models of PD.
Cantabio’s pharmacological chaperones from the CB101 program may mitigate the harmful effects of superfluous oxidation via thermodynamic stabilization of the DJ-1 homodimer. Moreover, they may directly interfere with DJ-1 superfluous oxidation by preventing over-oxidation of Cys106 a critical amino acid required for DJ-1 native and therapeutic function.