Amicus Therapeutics a Leading Giant in the Pharmaceutical Industry

Amicus Therapeutics Inc. is an international biotechnology company that is at the forefront of advancing therapies that aims at treating devastating rare and orphan diseases. Amicus Therapeutics became a public company in 2007 under FOLD which is a NASDAQ trading symbol. Prior to going public, the company was being funded by several venture capital firms such as Radius Ventures and Canaan Partners among others.

Amicus Therapeutics specializes in disorders collectively known as lysosomal storage disorders. The company products are largely based on the Chaperone-Advanced Replacement Therapy (CHART) platform which has concentrated on the development of enzyme replacement therapies (ERTs). Amicus in 2014 was noted as one of the companies with the broadest portfolio of small molecule also known as pharmacological chaperones in the Pharmaceutical industry.

Amicus lead product is the Migalastat which is a personalized late-stage development medicine that helps treat individuals who have Fabry disease from their genetic diagnosis. Another product of Amicus is the SD-101 which is also a late stage development product that provides therapy for Epidermolysis Bullosa (EB) which is a rare genetic connective tissue disorder ( Currently, Amicus is leveraging its biologics program and the CHART platform technologies to create a novel Enzyme Replacement Therapy ERT product to help in the diagnosis of Fabry disease, Pompe disease including other Lysosomal Storage Disorders. Amicus lead biologics program is the ATB200/AT2221. The program is a uniquely engineered Pompe disease ERT that will have to be administered together with a pharmacological chaperone.

Amicus Therapeutics has received many grants and donations to help it in conducting researches of advancing the Pharmacological Chaperone Technology. In 2010 Amicus received a grant of 500,000 dollars from The Michael J. Fox Foundation to help advance studies and research in collaboration with David Geffen School of Medicine at UCLA. Also the same year the company received another grant of over 200,000 dollars from the Alzheimers Drug Discovery Foundation. The grant was meant to support the Pre-clinical work together with the Icahn School of Medicine at Mount Sinai under the directive of Alzheimer Disease Research Center.

Amicus Therapeutics has been involved in a couple of major acquisition, and in 2013 the company acquired Callidus Biopharma Company which was one of its competitors. This was a significant move for the company as it gave them the rights to the enzyme replacement therapy treatment of Pompe disease.

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