| 发明名称 |
METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE |
| 摘要 |
PROBLEM TO BE SOLVED: To provide methods and compositions for CNS delivery of heparan n-sulfatase.SOLUTION: The present invention provides compositions and methods for CNS delivery of lysosomal enzymes, particularly for an effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.SELECTED DRAWING: None |
| 申请公布号 |
JP2016040335(A) |
申请公布日期 |
2016.03.24 |
| 申请号 |
JP20150246258 |
申请日期 |
2015.12.17 |
| 申请人 |
SHIRE HUMAN GENETIC THERAPIES INC |
发明人 |
FARAH NATOLI;ZHU GAOZHONG;JENNIFER TEREW;JIANG YUAN;JAMIE TSUNG;SHAHROKH ZAHRA;BRIAN VERNAGLIA;PAN JING;RICHARD PFEIFER;PERICLES CALIAS |
| 分类号 |
A61K38/00;A61K9/08;A61K9/19;A61K47/02;A61K47/10;A61K47/18;A61K47/24;A61K47/26;A61K47/34;A61P25/00;A61P25/14;A61P25/20;A61P25/28;A61P27/16;A61P43/00 |
主分类号 |
A61K38/00 |
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