- 专利标题： Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
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申请号： US16378163申请日： 2019-04-08
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公开(公告)号： US11254725B2公开(公告)日： 2022-02-22
- 发明人: Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
- 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
- 代理机构： Wilson Sonsini Goodrich & Rosati
- 主分类号： C07K14/65
- IPC分类号： C07K14/65 ; C12N9/24 ; A61K38/00 ; A61K38/47
摘要：
The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide.
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