Amicus Therapeutics Designs Clinical Programs That Focuses On Genetic Diseases

Amicus Therapeutics is a biopharmaceutical company based in New Jersey. They are a business led, a science-driven company that delivers meaningful benefits to patients. Their product development is largely based on a Chaperone-Advanced Replacement Therapy platform. The product is concentrated on the development of enzyme replacement therapies. Amicus Therapeutics doesn’t have manufacturing capabilities of its own. They rely on contract manufacturing. The company was founded in 2002, however, they went public in 2007. They opened a second site that is research only in 2008. This site is located in San Diego, California (


In 2010 Amicus Therapeutics received grants from the Michael J Fox Foundation and The Alzheimer’s Drug Discovery Foundation. In 2013 obtained Callidus Biopharma, which consisted of obtaining proprietary materials and intellectual properties for enzyme replacement. In the early months of 2014 Amicus Therapeutics didn’t have any marketed products. They obtained the company Scioderm in 2015. their Chief Executive Officer John F (Indeed). Crowley became the Chairman of the Board in 2010. The company that has a robust pipeline of treatments for a range of genetic diseases, has an intern program.


Amicus Therapeutics has an advanced pipeline and platform of technologies that put them at the forefront of developing therapies. The company has 4 different programs in place. Those programs consist of 3 clinical programs and a pre-clinical program. One of the clinical programs is the Fabry Disease Program. The program is developing an oral pharmacological chaperone for the treatment.


Another program is Epidermolysis Bullosa. This program develops the topical therapy SD-101 for the treatment of skin blistering and lesions associated with EB. The third clinical program is Pompe Disease. This program leverages biologics and Chaperone-Advanced Replacement therapy, also known as CHART, to develop ATB200/AT2221. The pre-clinical programs give the company the ability to leverage biologics capabilities to expand. Some of the preclinical programs are Lysosomal Storage Disorders and CDKL5 Deficiencies.

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