发明申请
- 专利标题: MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING CENTRONUCLEAR MYOPATHY
-
申请号: US17264966申请日: 2019-08-02
-
公开(公告)号: US20210322562A1公开(公告)日: 2021-10-21
- 发明人: Romesh R. Subramanian , Mohammed T Qatanani , Timothy Weeden
- 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/US2019/044963 WO 20190802
- 主分类号: A61K47/68
- IPC分类号: A61K47/68 ; C07K16/28 ; C12N15/113 ; C12N9/10 ; C12N9/14
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 DNM2. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
信息查询
IPC分类: