发明公开
- 专利标题: MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
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申请号: US18577374申请日: 2022-07-08
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公开(公告)号: US20240318176A1公开(公告)日: 2024-09-26
- 发明人: Cody A. Desjardins , Kim Tang , James McSwiggen , Romesh R. Subramanian , Timothy Weeden , Mohammed T. Qatanani , 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
- 国际申请: PCT/US2022/073527 2022.07.08
- 进入国家日期: 2024-01-08
- 主分类号: C12N15/113
- IPC分类号: C12N15/113 ; A61K39/00 ; A61K47/68 ; C07K16/28
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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