| 发明名称 |
Methods and compositions for CNS delivery of iduronate-2-sulfatase |
| 摘要 |
The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome. |
| 申请公布号 |
US9220677(B2) |
申请公布日期 |
2015.12.29 |
| 申请号 |
US201313974457 |
申请日期 |
2013.08.23 |
| 申请人 |
Shire Human Genetic Therapies, Inc. |
发明人 |
Zhu Gaozhong;Lowe Kris;Shahrokh Zahra;Christian James;Fahrner Rick;Pan Jing;Wright Teresa Leah;Calias Pericles |
| 分类号 |
A61K38/54;A61K9/00;A61K9/19;A61K38/46;A61K38/47;C07K14/65;C12N9/42;C12N9/24 |
主分类号 |
A61K38/54 |
| 代理机构 |
|
代理人 |
Chen Fangli;Sahr Robert N.;Nguyen Suzanne |
| 主权项 |
1. A method of treating Hunter Syndrome comprising a step of
administering intrathecally to a human subject in need of treatment a formulation comprising iduronate-2-sulfatase (I2S) at a concentration of at least 10 mg/ml and no greater than 5 mM phosphate, and wherein the I2S is administered at a dose amount of at least 10 mg. |
| 地址 |
Lexington MA US |
