Patent ID: 9567400
Date: 2017-02-14
CPC Classifications: A61K,A61P,C07K,C12N,C12Y

Claim:
1. A method of treating a central nervous system α-L-iduronidase deficiency in a subject in need thereof, comprising systemically administering to the subject in need thereof a therapeutically effective dose of a fusion antibody having α-L-iduronidase activity, wherein: (a) at least about 25,000 units of α-L-iduronidase activity are delivered to a brain tissue of the subject in need thereof; (b) the therapeutically effective dose of a fusion antibody having α-L-iduronidase activity comprises at least about 200,000 units of α-L-iduronidase activity/Kg of body weight; (c) the fusion antibody having α-L-iduronidase activity comprises a fusion protein comprising an amino acid sequence of an immunoglobulin heavy chain and an α-L-iduronidase; (d) the fusion antibody having α-L-iduronidase activity binds to an endogenous receptor of a blood brain barrier (BBB) transport system; and (e) the amino acid sequence of the α-L-iduronidase is covalently linked at its amino terminus to the carboxy terminus of the amino acid sequence of the immunoglobulin heavy chain, wherein the α-L-iduronidase retains at least 30% of its enzymatic activity compared to an unfused α-L-iduronidase.