发明公开
- 专利标题: MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING MYOTONIC DYSTROPHY
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申请号: US18416981申请日: 2024-01-19
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公开(公告)号: US20240197901A1公开(公告)日: 2024-06-20
- 发明人: Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins
- 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 ; A61K47/60 ; A61P21/00 ; 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 a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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