发明公开
- 专利标题: MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
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申请号: US18592695申请日: 2024-03-01
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公开(公告)号: US20240197905A1公开(公告)日: 2024-06-20
- 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 ; 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 promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.
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