专利检索 ap:("Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center" OR "Phoenix Nest Inc.") AND inv:"LE, Steven" 第 1 页

1.

发明公开
PREPARATION OF ENZYME REPLACEMENT THERAPY FOR MUCOPOLYSACCHARIDOSIS IIID 审中-公开

公开(公告)号：EP3505181A1

公开(公告)日：2019-07-03

申请号：EP18248241.4

申请日：2018-12-28

申请人： Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center , Phoenix Nest Inc.

发明人： DICKSON, Patricia , CHOU, Tsui-Fen , EKINS, Sean , KAN, Shih-Hsin , LE, Steven , MOEN, Derek R.

IPC分类号： A61K38/46 , C12N9/16

摘要： The present disclosure relates to compositions for use in a method of treating Sanfilippo syndrome (also known as Sanfilippo disease type D, Sanfilippo D, mucopolysaccharidosis type IIID, MPS IIID). The method can entail injecting to the spinal fluid of a MPS IIID patient an effective amount of a composition comprising a recombinant human acetylglucosamine-6-sulfatase (GNS) protein comprising the amino acid sequence of SEQ ID NO: 1 or an amino acid sequence having at least 90% sequence identity to SEQ ID NO: 1 and having the enzymatic activity of the human GNS protein. The composition can be tested in a testing buffer comprising less than 500 mM sodium, less than 100 mM citrate, less than 100 mM phosphate, less than 100 mM sulfate, and at least 0.005% Triton X-100 at a pH of about 5.6.