Amicus Therapeutics

Amicus Therapeutics develops drugs that treat rare genetic diseases known as lysosomal storage disorders. Unlike other treatments which replace defective enzymes, Amicus uses small molecule pharmacological "chaperones" which bind to a patient's own defective proteins and restore their functions. Its lead drug candidate, Amigal, is aimed at aiding patients with Fabry disease. Another candidate, Plicera, is in development to treat Gaucher disease, while a third candidate, AT2220 is targeting Pompe disease. The company is also researching treatments for neurodegenerative diseases including Alzheimer's and Parkinson's.