Invention Grant
US09320711B2 Methods and compositions for CNS delivery of heparan N-sulfatase
有权
CNS递送肝素N-硫酸酯酶的方法和组成
- Patent Title: Methods and compositions for CNS delivery of heparan N-sulfatase
- Patent Title (中): CNS递送肝素N-硫酸酯酶的方法和组成
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Application No.: US13168957Application Date: 2011-06-25
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Publication No.: US09320711B2Publication Date: 2016-04-26
- Inventor: Farah Natoli , Gaozhong Zhu , Jennifer Terew , Yuan Jiang , Jamie Tsung , Zahra Shahrokh , Brian Vernaglia , Jing Pan , Richard Pfeifer , Pericles Calias
- Applicant: Farah Natoli , Gaozhong Zhu , Jennifer Terew , Yuan Jiang , Jamie Tsung , Zahra Shahrokh , Brian Vernaglia , Jing Pan , Richard Pfeifer , Pericles Calias
- 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: Choate, Hall & Stewart LLP
- Main IPC: A61K38/46
- IPC: A61K38/46 ; C12N9/14 ; A61P25/00 ; C12Q1/34 ; A61K9/00 ; A61K9/19 ; C12N9/24 ; 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 a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.
Public/Granted literature
- US20120014936A1 METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE Public/Granted day:2012-01-19
Information query
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