Amicus Therapeutics: Continual Crusade Against Rare Diseases

Amicus Therapeutics was first found on February 4, 2002, but it became public in 2007 in being traded as NASDAQ: FOLD. It is headquartered in Cranbury, New Jersey, and its key personnel includes John Crowley, CEO; Bradley Campbell, COO; and William Baird, CFO.


Its History as a Private Company


Previously funded by New Enterprise Associates, Canaan Partners, and Radius Ventures while being a private company, Amicus Therapeutics focused on orphan and rare diseases, especially lysosomal storage disorders. Their product development is largely based on Chaperone-Advanced Replacement Therapy (CHART). They also concentrate on enzyme replacement therapy development.


Their Current Status


Today, they still focus mainly on advanced therapies to treat devastatingly rare, orphan diseases. However, Amicus Therapeutics now works on a global level. They still have the main headquarters in Cranbury, New Jersey, and their international headquarters is located in the United Kingdom. Amicus Therapeutics also has offices in five other locations throughout Europe and a research site in San Diego, California. As far as knowing their current, treatment techniques, they are as follows:


  1. Migalastat– their leading medicine candidate that is in the late stage of development for treating people with Fabry disease based on their genetic diagnosis.


  1. SD-101– another medical product candidate in its late development stage that is potentially the first therapy to market for Epidermolysis Bullosa (EB), a rare disorder of the genetic connective tissue.


In other developments, Amicus Therapeutics is leveraging their platform technologies in CHART and biologics to develop their novel enzyme therapies for Pompe disease, Fabry disease, and other lysosomal storage disorders (LSDs) (YahooFinance). They have a biologics program called ATB200/AT2221. This program is geared as a ERT for the Pompe disease in which will be combined with a pharmacological chaperone for administering.

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