Amicus Therapeutics Continues With Its Mission To Treat Rare Orphan Diseases

Amicus Therapeutics is a global biotechnology company, with headquarters in New Jersey, that is passionate about what it does. It is positioned as a leader in its industry and works to create therapies that help treat rare orphan diseases that can be very destructive in nature. Their leading product, which is in the later stages of its development, is called Migalastat, and it is a personalized treatment that helps to heal people who have Fabry disease (Crunchbase). Another treatment in late-stage development is SD-101, which looks to treat the rare genetic connective tissue disorder Epidermolysis Bullosa (EB). The company creates unique enzyme replacement therapies by using a platform of tech known as Chaperone-Advanced Replacement Therapy; otherwise known as CHARTâ„¢.


Amicus Therapeutics was founded in 2008 and while it began with one research site in New Jersey, it quickly grew and opened up the second one in San Diego. During the 2009 calendar year, Amicus had some financial issues that began with the end of a collaborative partnership with The Shire that ended after years of cooperation. By 2010, the company recovered and picked up a grant from the Michael J. Fox Foundation worth $500,000, which was granted to help its partnership with the David Geffen School of Medicine at UCLA. During the same year, a $210,300 grant was issued from the Alzheimer’s Drug Discovery Foundation that was given to help with a joined project between Amicus and the Icahn School of Medicine at Mount Sinai. During 2013, Amicus purchased Callidus Biopharma, which was a competitor, at the time, and in turn became the owner of a Pompe disease enzyme replacement therapy treatment (ForexWizard).


To this day, Amicus Therapeutics has not released any marketable products and relies on contract manufacturing as it has no manufacturing capabilities, itself. This has not stopped the company from continuing its mission to create treatments for some of the rarest kinds of diseases in existence, today.


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