Amicus Therapeutics is a biopharmaceutical company developing oral therapeutics known as pharmacological chaperones for the treatment of a range of human genetic diseases. The Company is focused on discovering, developing and delivering medicines for people living with rare metabolic diseases. Its 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. It is initially targeting lysosomal storage disorders and continues to develop its pharmacological chaperone technology having built an intellectual property portfolio along with its robust clinical and preclinical pipeline.