Amicus Therapeutics, Inc. (Nasdaq:FOLD) is a global biotechnology company at the forefront of advanced therapies to treat a range of devastating rare and orphan diseases. We have a robust development pipeline of treatments for a range of human genetic diseases.
Our lead product candidate, migalastat, is a personalized medicine in late-stage development to treat individuals with Fabry disease on the basis of their genetic diagnosis. SD-101, a product candidate in late-stage development, is a potential first-to-market therapy for the rare genetic connective tissue disorder Epidermolysis Bullosa (EB). We are leveraging our biologics and Chaperone-Advanced Replacement Therapy (CHART™)platform technologies to develop novel enzyme replacement therapy (ERT) products for Fabry disease, Pompe disease, and other Lysosomal Storage Disorders (LSDs). Our lead biologics program is ATB200/AT2221, a uniquely engineered Pompe disease ERT that will be administered in combination with a pharmacological chaperone.