Amicus Therapeutics, Inc. (FOLD)

Amicus Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of small molecule, orally-administered drugs, known as pharmacological chaperones, for the treatment of a range of human genetic diseases. The pharmacological chaperone technology involves the use of small molecules to restore biological activity in cells by binding to a misfolded protein caused by a genetic mutation. The company is targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. Its lead compound includes Amigal, which is in Phase II clinical trials is used for the treatment of Fabry disease that causes kidney failure, cardiac abnormalities, and neurological complications. The company is also developing AT2101 for the treatment of Gaucher disease, which causes an enlarged liver and spleen, low levels of red blood cells and platelets, bone pain, and fractures; and AT2220 for the treatment of Pompe disease, which causes muscle weakness primarily affecting breathing, mobility, and heart function. The company has a strategic collaboration with Shire Human Genetic Therapies, Inc. to jointly develop pharmacological chaperone compounds for lysosomal storage disorders. Amicus Therapeutics was founded in 2002 and is headquartered in Cranbury, New Jersey.