Targeted Therapeutic Lysosomal Enzyme Fusion Proteins and Uses Thereof
    2.
    发明申请
    Targeted Therapeutic Lysosomal Enzyme Fusion Proteins and Uses Thereof 有权
    靶向治疗溶酶体融合蛋白及其用途

    公开(公告)号:US20140161788A1

    公开(公告)日:2014-06-12

    申请号:US14092336

    申请日:2013-11-27

    IPC分类号: C07K14/65 C12N9/24

    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.

    摘要翻译: 本发明一般涉及可用于治疗溶酶体贮积病的治疗融合蛋白和治疗这些疾病的方法。 示例性治疗性融合蛋白包含溶酶体酶,溶酶体靶向部分,例如IGF-II肽和间隔肽。 还提供了用于治疗IIIB型粘多糖贮积症(Sanfilippo B综合征)的组合物和方法,其包含靶向治疗性融合蛋白,其包含α-N-乙酰氨基葡糖苷酶(Naglu),溶酶体靶向部分,例如IGF-II肽和间隔肽 。

    Lysosomal Targeting Peptides and Uses Thereof

    公开(公告)号:US20210069304A1

    公开(公告)日:2021-03-11

    申请号:US16869862

    申请日:2020-05-08

    IPC分类号: A61K38/47 C07K14/65 C12N9/26

    摘要: The present invention provides further improved compositions and methods for efficient lysosomal targeting based on the GILT technology. Among other things, the present invention provides methods and compositions for targeting lysosomal enzymes to lysosomes using furin-resistant lysosomal targeting peptides. The present invention also provides methods and compositions for targeting lysosomal enzymes to lysosomes using a lysosomal targeting peptide that has reduced or diminished binding affinity for the insulin receptor.