The Dr. Sol J. Barer Award for
Vision, Innovation and Leadership
Loading the player ...
RareVoice Lifetime Achievement Award –
Rare Disease Congressional Caucus Briefing
Home >> About Us >> Overview
Amicus Therapeutics (Nasdaq:FOLD) is a biopharmaceutical company at the forefront
of therapies for rare and orphan diseases. The Company has a robust pipeline
of novel, first-in-class treatments in development for a broad range of human genetic diseases, with a focus on
delivering new benefits to individuals with lysosomal storage diseases (LSDs).
The Company is leveraging its Chaperone-Advanced
Replacement Therapy (CHART™) platform to develop next-generation ERTs by
identifying and co-formulating proprietary therapeutic enzymes with pharmacological
chaperones. The lead CHART programs in late preclinical development are AT-B100
co-formulated with the pharmacological chaperone migalastat HCl, a next-generation
ERT for Fabry disease; and AT-B200, co-formulated with the pharmacological chaperone
AT2220, a next-generation ERT for Pompe disease. Amicus is also evaluating a next-generation
ERT for Mucopolysaccharoidosis Type I (MPS I) in preclinical studies. Amicus retains
worldwide rights to all applications of its proprietary CHART platform.
Amicus is also developing novel small molecules as monotherapy treatments for Fabry
disease and Parkinson’s. Amicus owns global rights to its pharmacological chaperone,
migalastat HCl, as a monotherapy for Fabry disease. In addition, the Company has
entered a collaboration with Biogen Idec to discover, develop and commercialize
novel small molecules for the treatment of Parkinson's disease, for further development
by Biogen Idec.
Amicus Therapeutics is committed to improving the lives of patients and families affected by rare and orphan diseases.