About Amicus Therapeutics
Amicus Therapeutics is a biopharmaceutical company developing novel, oral therapeutics known as pharmacological chaperones for the treatment of a range of human genetic diseases. Pharmacological chaperone technology involves the use of small molecules to restore or improve biological activity in cells by selectively binding to a misfolded protein caused by a genetic mutation. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. Amicus is currently conducting Phase 2 clinical trials for its two lead compounds, Amigal for Fabry disease, and AT2220 for Gaucher disease. The company is currently conducting Phase 1 trials with AT2220 for the treatment of Pompe disease.
Source: Amicus Therapeutics