Amicus Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of a new class of small molecule, orally-administered drugs to treat a range of human genetic diseases.
Amicus’ innovative therapies apply to conditions in which crucial proteins are defective as a result of improper folding.
Instead of trying to replace these complex proteins, Amicus’ approach uses pharmacological chaperones to selectively bind to the misfolded target protein, increase stability, and help the protein fold into its proper three-dimensional shape. This allows the protein to be trafficked to the specific location in the cell, where it performs its intended biological function. Pharmacological chaperone technology represents a novel, next-generation approach to the management of human genetic diseases and offers the potential to improve treatment options for patients.