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1. (WO2018144441) TREATMENT OF MUCOPOLYSACCHARIDOSIS I WITH FULLY-HUMAN GLYCOSYLATED HUMAN ALPHA-L-IDURONIDASE (IDUA)

Pub. No.:    WO/2018/144441    International Application No.:    PCT/US2018/015910
Publication Date: Fri Aug 10 01:59:59 CEST 2018 International Filing Date: Wed Jan 31 00:59:59 CET 2018
IPC: A61K 38/00
A61K 38/43
A61K 48/00
A61P 25/00
A61P 25/28
C12N 15/09
Applicants: REGENXBIO INC.
Inventors: YOO, Stephen
REINHARDT, Rickey Robert
SIMPSON, Curran Matthew
WU, Zhuchun
Title: TREATMENT OF MUCOPOLYSACCHARIDOSIS I WITH FULLY-HUMAN GLYCOSYLATED HUMAN ALPHA-L-IDURONIDASE (IDUA)
Abstract:
Compositions and methods are described for the delivery of a fully human-glycosylated (HuGly) α-L-iduronidase (IDUA) to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis I (MPS I).