Patent ID: 9579366
Date: 2017-02-28
CPC Classifications: A61K,A61P,C07K,C12N,C12Y

Claim:
1. A method of treating a subject suffering from NaGlu deficiency, the method comprising intravenously administering to the subject a therapeutically effective amount of a composition comprising an isolated recombinant human N-acetyl-alpha-D-glucosaminidase (rhNaGlu) whose amino acid sequence is set forth in 24-743 of SEQ ID NO:1, wherein said rhNaGlu comprises one or more glycan structures having sufficient amount of mannose-6-phosphate (M6P) that allows for internalization of said rhNaGlu into a mammalian cell having NaGlu deficiency via M6P receptor-mediated endocytosis, such that when internalized in vivo into a subject showing a deficiency in NaGlu, said rhNaGlu restores at least 50% of NaGlu activity observed in a cell of the same type in a normal subject, and wherein the rhNaGlu contains at least 1 mole of M6P per mole of the rhNaGlu protein, with the proviso that the rhNaGlu is not administered intrathecally.