- 专利标题： MANUFACTURE OF ACTIVE HIGHLY PHOSPHORYLATED HUMAN LYSOSOMAL SULFATASE ENZYMES AND USES THEREOF
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申请号： US17188994申请日： 2021-03-01
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公开(公告)号： US20210189357A1公开(公告)日： 2021-06-24
- 发明人: Erno Pungor , Charles Hague , Zhi Chen , Melita Dvorak-Ewell , Michel Claude Vellard , Vish Koppaka
- 申请人： BIOMARIN PHARMACEUTICAL INC.
- 申请人地址： US CA Novato
- 专利权人： BIOMARIN PHARMACEUTICAL INC.
- 当前专利权人： BIOMARIN PHARMACEUTICAL INC.
- 当前专利权人地址： US CA Novato
- 主分类号： C12N9/16
- IPC分类号： C12N9/16 ; C12Q1/34 ; C12Q1/44 ; G01N33/68
摘要：
This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.
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