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 misfolded, unstable proteins caused by genetic mutations. This technology represents a novel, next-generation approach to the management of human genetic diseases and offers the potential to improve treatment options for many patients.
Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. The company believes pharmacological chaperone technology has broad applicability and expects to expand the portfolio to include other diseases of neurodegeneration, metabolic diseases, and oncology. Founded in 2002, Amicus has assembled an outstanding leadership team that includes scientific, medical, regulatory, and business professionals with successful track records of developing and bringing to market drugs for genetic diseases. Amicus continues to develop its pharmacological chaperone technology and has built a significant intellectual property portfolio along with its robust clinical and preclinical pipeline.
Amicus has a fast-paced, high-energy, entrepreneurial team environment where employees are encouraged to be innovative and resourceful. Amicus seeks high-caliber, talented individuals with a passion to succeed.