Amicus Therapeutics is a biotechnology company focused on discovering, developing, and delivering medicines for diseases. Co.'s gene therapy portfolio pipeline include Pompe disease, Fabry disease, CDKL5 deficiency disorder, ceroid lipofuscinosis, neuronal, Mucopolysaccharidosis Type IIIB, as well as a program in Mucopolysaccharidosis Type IIIA. Co.'s collaboration with University of Pennsylvania also provides Co. with disease-specific access and option rights to develop new gene therapy platform technologies and programs for most lysosomal disorders and a portfolio of other rare diseases.