Amicus Therapeutics Announces Chaperone-Advanced Replacement Therapy in Development for MPS I

Amicus Therapeutics FOLD today disclosed a preclinical Chaperone-Advanced Replacement Therapy (CHART™) program for Mucopolysaccharidosis Type I (MPS I), a lysosomal storage disease caused by missing or deficient alpha-L-iduronidase (IDUA) enzyme. Amicus is developing a proprietary human recombinant IDUA (rhIDUA) enzyme co-formulated with a novel pharmacological chaperone as a next-generation therapy for MPS I. The pharmacological chaperone is designed to improve tissue uptake and reduce the immunogenicity of rhIDUA by stabilizing the enzyme in its properly folded and active form. In addition, in support of its development of a proprietary rhIDUA enzyme, Amicus has received a grant of up to approximately $250,000 from a private U.S.-based donor that provides medical research grants to find better treatments and cures for rare genetic disorders, including lysosomal storage diseases.