Founded in 2002, Amicus Therapeutics is a public biopharmaceutical company that handles devastating rare and orphan illnesses. The firm focuses on disorders collectively referred to as Lysosomal Storage Disorders (LSDs). They include Pompe disease, Fabry disease, CDKL5, and rare genetic connective skin disorders called Epidermolysis Bullosa (EB).
Based in Cranbury, New Jersey, Amicus Therapeutics goal involves making the lives of the patients meaningful. The company collaborates with patients, health practitioners, and the caregivers to assist the rare disease community. They have utilized the aspect of science to invent and develop effective Enzyme Replacement Therapies (ERT). The company’s product is founded in a big way on the Chaperone-Advanced Replacement Therapy (CHART).
Amicus Therapeutics Chairman and CEO, John F. Crowley has been involved with biotechnology stems since 1998 after losing his two children with Pompe disease. John Crowley has dedicated his life to developing remedies for Pompe disease as well as other rare disorders. He has enabled the company to develop a technology called pharmacological chaperone that treat specific LSDs. This LSDs have traits of unfolded, unstable or misfolded mutated proteins. The technology is designed with tiny molecules which selectively target enzyme with the aim of stabilizing and turning it into the required three-dimensional shape.
The company’s leading product, though in the last stage of development, is referred to as Migalastat (https://finance.yahoo.com/quote/FOLD?ltr=1). The personalized medicine is meant to treat patients with Fabry disorder on the ground of individual genetic diagnosis. For the Epidermolysis Bullosa, Amicus Therapeutics is developing a first to market therapy called SD-101. Its topping biological program is the ATB200/AT2221, a different Pompe disease ERT that will be combined with the pharmacological chaperone.
Amicus Therapeutics in its development has collaborated with other companies as well acquiring others. In 2010, it worked with JCR pharmaceutical and GlaxoSmithKline to research on alpha-galactosidase. In 2013, it acquired a competitor, Callidus Biopharma as well as Scioderm in 2015.