Invention Grant
- Patent Title: Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
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Application No.: US16378163Application Date: 2019-04-08
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Publication No.: US11254725B2Publication Date: 2022-02-22
- Inventor: Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
- Applicant: BioMarin Pharmaceutical Inc.
- Applicant Address: US CA Novato
- Assignee: BioMarin Pharmaceutical Inc.
- Current Assignee: BioMarin Pharmaceutical Inc.
- Current Assignee Address: US CA Novato
- Agency: Wilson Sonsini Goodrich & Rosati
- Main IPC: C07K14/65
- IPC: C07K14/65 ; C12N9/24 ; A61K38/00 ; A61K38/47

Abstract:
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.
Public/Granted literature
- US20190225666A1 Targeted Therapeutic Lysosomal Enzyme Fusion Proteins and Uses Thereof Public/Granted day:2019-07-25
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