Glucocerebrosidase Non-inhibitory Chaperones for the Treatment of Gaucher Disease, Parkinson's Disease, and Other Proteinopathies
- Early-stage
- In vitro data available
This technology describes a collection of salicylic acid-derived small molecules that act as chaperones to activate proper GCase folding and subsequent transport from the endoplasmic reticulum into the lysosome. Unlike many other small molecule chaperones, these salicylic acid derivatives do not inhibit the activity of the GCase enzyme. These small molecules have been tested for the ability to activate GCase in vitro and show chaperone activity in a patient-derived fibroblast translocation assay.
- Treatment of Gaucher disease
- Treatment of Parkinson’s disease
- Treatment of other lysosomal storage diseases
- The compounds are novel small molecules that enhance proper GCase folding and transport without inhibiting enzyme activity in the lysosome.