- 专利标题: MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
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申请号: US18329781申请日: 2023-06-06
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公开(公告)号: US20230321264A1公开(公告)日: 2023-10-12
- 发明人: Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , John Najim
- 申请人: Dyne Therapeutics, Inc.
- 申请人地址: US MA Waltham
- 专利权人: Dyne Therapeutics, Inc.
- 当前专利权人: Dyne Therapeutics, Inc.
- 当前专利权人地址: US MA Waltham
- 主分类号: A61K47/68
- IPC分类号: A61K47/68 ; C07K14/47 ; C07K16/28 ; C12N15/113
摘要:
Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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