发明名称 |
Targeted Therapeutic Lysosomal Enzyme Fusion Proteins and Uses Thereof |
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. |
申请公布号 |
US2016031965(A1) |
申请公布日期 |
2016.02.04 |
申请号 |
US201514883203 |
申请日期 |
2015.10.14 |
申请人 |
BioMarin Pharmaceutical Inc. |
发明人 |
Aoyagi-Scharber Mika;Christianson Teresa Margaret;Dvorak-Ewell Melita;Wendt Daniel J.;Long Shinong;LeBowitz Jonathan;Gold Daniel Solomon |
分类号 |
C07K14/65;C12N9/24 |
主分类号 |
C07K14/65 |
代理机构 |
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代理人 |
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主权项 |
1. A targeted therapeutic fusion protein comprising (a) a lysosomal enzyme, (b) a peptide tag having an amino acid sequence at least 70% identical to amino acids 8-67 of mature human IGF-II and (c) a spacer peptide between the lysosomal enzyme and the IGF-II peptide tag, wherein the spacer peptide comprises the amino acid sequence set out in SEQ ID NO: 55. |
地址 |
Novato CA US |