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Company Overview: |
Amicus Therapeutics is a biopharmaceutical company that develops drugs which are used to treat rare genetic diseases called lysosomal storage disorders. Their enzyme replacement therapies have been designed to find defective proteins and restore their proper functioning. Amicus Therapeutics’ top drug candidate, Amigal is formulated to treat those with Fabry disease, an illness that is accompanied by symptoms like impaired sweating, skin rashes, as well as heart, kidney and gastrointestinal problems of different degrees of intensity.
Amicus Therapeutics is also working on special drugs candidates which are designed to provide treatment for Gaucher disease, lysosomal disorder with symptoms like anemia and skeletal lesions; and Pompe disease, an illness characterized by muscle weakness as well as impaired breathing, heart function and bodily mobility.
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Products and Services: |
Amicus Therapeutics’ drug line is centered on the full-scale development of small molecule, orally-administered known as pharmacological chaperones in order to deal with a group of human genetic diseases. The company is in the middle of conducting multiple Phase II clinical studies for Amigal while AT2101 is in Phase I clinical studies and it is preparing to file an INDA (Investigational New Drug Application) with the FDA for AT2220
Amigal (migalastat hydrochloride) is an experimental treatment of Fabry disease that is administered orally. It belongs to a group of molecules called pharmacological chaperones. The drug functions by binding only to those misfolded enzymes that are responsible for Fabry disease (In this case, it is a-GAL). The treatment is thought to increase the enzyme’s stability and allow it to fold properly, process and travel through the cell unhindered. Its final destination is the lysosome, the part of the cell’s structure where enzymes operate. Here in the lysosome, the pharmacological chaperone falls away and the enzyme can perform its normal biological function.
AT2101 is an oral therapy used to treat Gaucher disease and belongs to a group of molecules known as pharmacological chaperones. The drug works by selectively binding to the misfolded enzyme responsible for Gaucher disease (GCase). Afterwards, the drug encourages proper folding, processing, and movement of the enzyme throughout the cell to its proper place in the lysosome. Once it has arrived there, the pharmacological chaperone falls away and the enzyme can perform its normal function.
AT2220 is an experimental, oral therapy that is used to treat Pompe disease. It too is a member of a class of molecules called pharmacological chaperones. Like Amigal and AT2101, AT2220 is specifically designed to function in as the pharmacological chaperone for Gaa enzyme, which is responsible for Pompe disease, binding to it and allowing it to fold properly, operate and move through the cell infrastructure unhindered until it can reach the lysosome. Like the other drug candidates, AT2220 breaks down once it has fulfilled its purpose. Then the enzyme can do what it is supposed to do.
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Key Personnel Facts: |
Donald J. Hayden Jr. Executive Chairman, Interim President, and CEO
Matthew R. Patterson: Chief Operating Officer
Mark J. Simon: Senior Vice President, Business Development
Employees (2005): 52
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Key Financial Facts: |
Fiscal Year-End: December
Sales (2005): $0.0M
1-Year Sales Growth: N/A
Net Income (2005): $19.7M
1-Year Employee Growth: 372.7%
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Working @ Amicus Therapeutics
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Working @ Amicus Therapeutics
Share your experiences about working, interviewing or applying for a job at Amicus ...
Author: Obi Posted: 3/3/2007 4:39:36 AM |
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Amicus Therapeutics in the News
- Amicus Therapeutics Announces Second Quarter 2007 Financial Results (PR Newswire)
Published: Tuesday, August 07, 2007
- Amicus Therapeutics Files For Initial Public Offering
Published: Friday, March 30, 2007
- Amicus Therapeutics Presents Data From Preclinical and Phase 1 Studies of Amigal(TM) for Fabry ...
Published: Monday, March 19, 2007
- Amicus Therapeutics Files for Initial Public Offering
Published: Wednesday, May 17, 2006
- Amicus Therapeutics Raises $55 Million Series C
Published: Thursday, September 08, 2005
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Biotechnology
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