- Patent Title: Methods and compositions for CNS delivery of iduronate-2-sulfatase
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Application No.: US16515568Application Date: 2019-07-18
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Publication No.: US11260112B2Publication Date: 2022-03-01
- Inventor: Gaozhong Zhu , Kris Lowe , Zahra Shahrokh , James Christian , Richard Fahrner , Jing Pan , Teresa Leah Wright , Pericles Calias
- Applicant: Shire Human Genetic Therapies, Inc.
- Applicant Address: US MA Lexington
- Assignee: Shire Human Genetic Therapies, Inc.
- Current Assignee: Shire Human Genetic Therapies, Inc.
- Current Assignee Address: US MA Lexington
- Agency: Proskauer Rose LLP
- Agent Fangli Chen
- Main IPC: A61K38/47
- IPC: A61K38/47 ; A61K35/76 ; A61K35/761 ; A61K9/19 ; C12N9/24 ; A61K9/00 ; A61K38/46 ; C07K14/65 ; C12N9/42 ; A61K9/08 ; A61K47/02 ; A61K47/26

Abstract:
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.
Public/Granted literature
- US20200046810A1 METHODS AND COMPOSITIONS FOR CNS DELIVERY OF IDURONATE-2-SULFATASE Public/Granted day:2020-02-13
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