Amicus Therapeutics is a biotechnology company with a global aim of identifying therapies for rare and devastating diseases. With John F. Crowley as the Chairman and CEO Amicus Therapeutics is the Leader in innovative technologies and medicines development for rare and devastating diseases. Including Lysosomal Storage Disorders and rare genetic connective tissue disorder Epidermolysis Bullosa (EB). The company has been in operation from 2007 when it went public under the NASDAQ trading symbol FOLD. In 2008, the company expanded to another research site in San Diego making them two.
Amicus Therapeutics has several programs managed through its development pipeline that includes Fabry Franchise (Migalastat), Epidermolysis Bullosa (SD-101), Pompe Disease (ATB200/AT2221) and other rare diseases. The first program is Fabry disease program, which has led to the development of an oral pharmacological chaperone, migalastat, for treatment of Fabry disease. Another is Epidermolysis Bullosa (EB) program that is developing SD-101 for the treatment of skin blistering and lesions linked to EB.
Thirdly, there is the Pompe Disease program to develop ATB200/AT2221, which is a scheme that aims to treat the Pompe disease. The disease arises because of a deficiency of enzyme acid alpha-glucosidase, which leads to a Lysosomal Storage Disorder (LSD) and it is inheritable. Lastly is the Preclinical programs, focusing on Lysosomal Storage Disorders a unique tool set to develop Enzyme Replacement Therapy (ERT) products for LSDs (http://alivenewspaper.com/2017/09/amicus-therapeutics-fabry-disease-treatment/). Another program within the Preclinic is the Cyclin-dependent kinase-like 5. In particular, it is a gene deficiency found on the X-chromosome that encodes the CDKL5 protein. The function of the protein is to regulate the expression of essential proteins for normal brain development.
Amicus Therapeutics does not own any manufacturing capability and so partners with other organization. The company has also received several grants from the following foundations, the Michael J. Fox Foundation and Alzheimer’s Drug Discovery Foundation to support pre-clinical work. Amicus Therapeutics believes and is committed to the needs of patients with the rare disease, and thus becoming the center of their inventive technology, marketing, and clinical programs. With their belief statement “We believe…” that encompasses around their core values and crucial in understanding the passion and commitment behind Amicus (Crunchbase).