Amicus Therapeutics is developing a diverse pipeline of novel, small molecule, orally-administered pharmacological chaperones to treat a range of human genetic diseases. Amicus is currently conducting multiple Phase 2 studies of its lead product, AT1001, for Fabry disease. Amicus begins Phase 2 clinical studies of AT2101 for Gaucher disease in Spring, 2007. Amicus is leveraging its core pharmacological chaperone platform to actively pursue therapies in other genetic diseases, including Parkinson's disease. Phase 1 clinical studies of AT2220 in Pompe disease are ongoing.


