- 专利标题: MUSCLE-TARGETING COMPLEXES AND USES THEREOF
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申请号: US17791667申请日: 2021-01-08
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公开(公告)号: US20230226212A1公开(公告)日: 2023-07-20
- 发明人: Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden , Cody A. Desjardins , Brendan Quinn , Jason P. Rhodes
- 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
- 国际申请: PCT/US2021/012764 2021.01.08
- 进入国家日期: 2022-07-08
- 主分类号: A61K47/68
- IPC分类号: A61K47/68 ; A61P21/00
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 activity of a disease allele associated with muscle disease. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
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