Amicus Therapeutics Fighting Rare and Orphan Diseases with Relentless Research

Based in Cranbury, New Jersey, Amicus Therapeutics, is a biopharmaceutical company that develops therapeutics for rare and orphan diseases that are called lysosomal storage disorders.

Their platform, CHART, focuses on developing of enzyme replacement therapies with which to treat these lysosomal disorders. They are known in the biopharmaceutical industry as having an abundance of small molecule pharmacological chaperones.

As Amicus Therapeutics does not have the capability of manufacturing their products on their own they must rely on contracting it to other manufacturers, such as JCR Pharmaceutical and GlaxoSmithKline. They worked with them from 2010 to 2013 in investigating the coformulation and recombinant alpha-galactosidase for the advancement of migalastat monotherapy.

Amicus Therapeutics expanded its research facility from New Jersey, opening a second research site in San Diego, California in 2008 (Yahoo Finance). Then in 2010, they were able to collaborate with David Geffen at the School of Medicine,UCLA due to a grant received for $500,000 from the Michael J. Fox Foundation, the same year they received another grant for $210,300 from the Alzheimer’s Drug Discovery Foundation for pre-clinical research with Ichan School of Medicine, Mount Sinai, working with the Alzheimer’s Disease Research Center.

In 2013, a competitor of Amicus, Callidus Biopharma was acquired which gained them intellectual property and proprietary access for an enzyme replacement treatment for Pompe disease, an inherited Lysosomal Storage Disorder caused by the body not producing the enzyme acid alpha-glucosidase or GAA.

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Their leading product, migalastat, which is used to treat persons with Fabry disease,a disease that can lead to progressive and irreversible organ damage. This disease will affect the nervous system as well as cardiac, renal and a multitude of other tissues. Migalastat is in the late stages of development.

Amicus has built a belief statement to cover their core values plus be a reminder for the reason they do every day what they do. Part of the belief is that they believe in the fight against these diseases, they strive to have the highest quality therapies for anyone with these diseases. They firmly believe in supporting the communities of these diseases and their families.
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