Amicus Therapeutics, Inc. is a biopharmaceutical company based in New Jersey. It is a leading company in the field of advanced therapies and treats a wide range of rare and orphan diseases. It deals specifically with conditions known as lysosomal storage disorders. Its product development pipeline is primarily based on the Chaperone-advanced replacement Therapy (CHART) and Enzyme Replacement Therapies (ERT).
Amicus Therapeutics boasts of having the broadest range of small molecule pharmacological chaperones in the industry. Its most significant product is known as migalastat which is a personalized medicine that can treat Fabry disease based on genetic diagnosis. Another late-stage development product in Amicus Therapeutics product pipeline is SD-101 which is a potential first-to-market therapy used to treat the rare genetic connective tissue disorder Epidermolysis Bullosa.
In the recent past, the company has collaborated with GlaxoSmithKline and JCR Pharmaceutical to investigate the coformulation with recombinant alpha-galactosidase. Amicus Therapeutics does not have manufacturing capability and mostly relies on contract manufacturing. The company has been able to expand its location in New Jersey to another research site in San Diego. It has been receiving a string of grants to help it support its studies. View the market summary of Amicus Therapeutics at finance.google.com.
In 2010. It received $0.5 million grant from the Michael J. Fox-Foundation for studies that it was doing in collaboration with the David Geffen School of Medicine at UCLA. It also received additional funds from the Alzheimer Drug Discovery Foundation in support of pre-clinical work it was undertaking together with Icahn School of Medicine at Mount Sinai.
In 2013, Amicus Therapeutics was able to acquire Callidus Biopharma together with its intellectual property and proprietary materials for the enzyme replacement therapy treatment of the Pompe disease. This development was a major milestone for the company which made it stamp its authority as a leading biotechnology company in the industry. Read the company profile at Crunchbase.