Definitions for amicus therapeutics
am·i·cus ther·a·peu·tics

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1. Amicus Therapeutics

   Amicus Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of a new class of small molecule, orally-administered drugs to treat a range of human genetic diseases. Amicus’ innovative therapies apply to conditions in which crucial proteins are defective as a result of improper folding. Instead of trying to replace these complex proteins, Amicus’ approach uses pharmacological chaperones to selectively bind to the misfolded target protein, increase stability, and help the protein fold into its proper three-dimensional shape. This allows the protein to be trafficked to the specific location in the cell, where it performs its intended biological function. Pharmacological chaperone technology represents a novel, next-generation approach to the management of human genetic diseases and offers the potential to improve treatment options for patients. Founded in 2002, Amicus has assembled an outstanding leadership team that includes scientific, medical, regulatory, and business professionals with successful track records of developing and bringing to market drugs for rare genetic diseases. Amicus continues to develop its pharmacological chaperone technology and has built a significant intellectual property portfolio, as well as a robust clinical and preclinical pipeline of small molecule, orally-administered agents. Amicus’ most advanced product candidate, AT1001, is currently in Phase 2 clinical trials for Fabry disease. In Phase 1 studies in healthy volunteers, AT1001 was well tolerated, even at the highest doses, without any drug-related adverse events. In addition, AT1001 was shown to have high oral bioavailability and good pharmacokinetics. Finally, studies showed a statistically significant and dose-related increase in the level of the target enzyme in healthy volunteers. The company’s second most advanced clinical product candidate, AT2101, is in Phase 2 clinical trials for Gaucher disease. Animal studies have demonstrated that administration of AT2101 causes a dose-dependent increase in the level of the enzyme known to be deficient in Gaucher disease. Amicus’ third most advanced product candidate is AT2220 for the treatment of Pompe disease. Preclinical studies have demonstrated that administration of AT2220 causes an increase in the level of the enzyme known to be deficient in Pompe disease. Phase 1 trials are ongoing. Additionally, Amicus’ biologists and medicinal chemists are leveraging the company’s breakthrough scientific platform to conduct targeted drug development, allowing for the rapid identification and optimization of lead compounds across a variety of disease areas, including Parkinson's Disease. Amicus has broad, issued intellectual property around the methods, use, and composition of pharmacological chaperones to treat genetic diseases. In addition to the currently issued patents, Amicus has several patent applications pending. Amicus has attracted support from a number of leading venture firms and in 2006 completed a $60 million Series D financing. Amicus Therapeutics' headquarters and laboratories are located in Cranbury, NJ.

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1. Amicus Therapeutics

   Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront of developing therapies for rare and orphan diseases. The Company has a robust pipeline of novel, first-in-class, small molecules called pharmacological chaperones for the treatment of lysosomal storage diseases (LSDs). These chaperones may offer a dual-treatment approach for Fabry, Pompe, Gaucher and other LSDs. As orally administered monotherapy agents, pharmacological chaperones are designed to bind to, stabilize and increase the activity of a patient's own misfolded enzyme. In combination with enzyme replacement therapy (ERT), pharmacological chaperones may improve the uptake of the infused enzyme and potentially improve ERT outcomes.

How to pronounce amicus therapeutics?

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Numerology

1. Chaldean Numerology

   The numerical value of amicus therapeutics in Chaldean Numerology is: 2

2. Pythagorean Numerology

   The numerical value of amicus therapeutics in Pythagorean Numerology is: 4

References

1. ^ Wikidata
   https://www.wikidata.org/w/index.php?search=amicus therapeutics
2. ^ CrunchBase
   https://www.crunchbase.com/organization/amicus-therapeutics