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CRANBURY, N.J., March 4, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), today introduced
Therapy (CHART™) as the brand name for its technology platform that combines unique pharmacological chaperones with enzyme replacement therapy (ERT) for lysosomal storage diseases.
John F. Crowley, Chairman and Chief Executive Officer of Amicus stated, "We are pleased to introduce CHART as the brand name and designation for our chaperone-ERT combination platform. CHART reflects the breadth of the opportunities at Amicus to develop chaperones co-administered with marketed ERTs, or co-formulated with proprietary enzymes as next-generation ERTs. It is a platform that has already demonstrated proof of concept in the clinic and in multiple pre-clinical studies. The direct co-formulation of our proprietary enzymes with specific small molecule chaperone stabilizers may improve treatment outcomes, reduce immunological responses and potentially enable more convenient delivery routes with these next-generation ERTs. Our strategic vision is to build out this CHART platform to deliver new benefits for patients and create multiple sources of value for our shareholders. This platform will indeed chart a course to a very bright future for Amicus."
Chaperone- Advanced Replacement Therapy (CHART)
Amicus is leveraging the CHART platform to improve currently marketed ERTs through co-administration of a pharmacological chaperone prior to ERT infusion, and to develop next-generation ERTs that consist of proprietary lysosomal enzyme therapies co-formulated with pharmacological chaperones.
ERTs are the standard of care for many lysosomal storage diseases. A recombinant or gene-activated human enzyme is manufactured for intravenous infusion into the circulation for transport to the lysosome. Once in lysosomes, ERT can perform the function of a person's own missing or deficient enzyme. However, these therapeutic enzymes may unfold and lose activity at any stage in the process, from the infusion bag to the bloodstream to the eventual uptake into cells and tissue. This instability, unfolding, and loss of enzyme activity may impact treatment outcomes with ERT.