Amicus Therapeutics – Striving to Give Solutions to Rare and Devastating Diseases

Amicus Therapeutics is New Jersey-based biotechnology company dealing with coming up with therapies to treat rare or orphan diseases. The company uses a unique combination of technologies and medications to ensure people living with rare and devastating diseases are cured. Such conditions include Pompe disease, Lysosomal Storage Disorders Fabry, and Epidermolysis Bullosa ( To treat Fabry disease, Amicus Therapeutics has come up with migalastat HCI which is a small molecule that can be used as monotherapy together with enzyme replacement therapy (ERT) for useful results. They have again come up with SD-101, an effective medicine for treating Epidermolysis Bullosa, a rare connective tissues disorder.

To come up with ERT solutions for Pompe disease, Fabry disease, and potentially other lysosomal storage disorders (LSD, Amicus Therapeutics uses Chaperone-Advanced Replacement Therapy (CHART) technological platform. Research is also underway pertaining preclinical and invention projects aimed at discovering solutions to treat other diseases that rare and devastating. One such condition is the cyclin-dependent kinase-like 5 (CDKL5) deficiency.

Their technological platform consists of CHART, Enzyme Targeting, and Pharmacological Chaperone Monotherapy. All these form Pharmacological Chaperone Technology which operates to discover solutions for human genetic diseases. Their solutions work by targeting unfolded and unstable mutated proteins in the body of the victim.

The Company’s customized medicine approach comprises of an oral small molecule pharmacological chaperone monotherapy that is made in a way to bind and an endogenous target protein in a victim (GoogleFinance). In Amicus Therapeutics CHART programs, every chaperone is designed to attach and bring stability to a specific therapeutic enzyme. The Company is also developing ERTs with higher amounts of mannose 6-phosphate (M6P) for lysosomal targeting.

Their core operations regarding innovative science, commercial organization, and clinical programs are squarely based in addressing the needs of patients suffering from rare diseases. The company’s objectives in all the levels of its operations are centered on making life simple for the patients and those around them.

Amicus Therapeutics does not manufacture the medicines alone but relies on contracts. The company has developed since its establishment in Feb 2002 where they expanded from their single operation site in New Jersey and opened a second research site in San Diego in 2008.