发明申请
WO2020028864A1 MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
审中-公开
基本信息:
- 专利标题： MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
- 申请号：PCT/US2019/044990 申请日：2019-08-02
- 公开(公告)号：WO2020028864A1 公开(公告)日：2020-02-06
- 发明人： SUBRAMANIAN, Romesh, R. , QATANANI, Mohammed, T. , WEEDEN, Timothy
- 申请人： DYNE THERAPEUTICS, INC.
- 申请人地址： 400 Technology Square, 10th Floor Cambridge, MA 02139 US
- 专利权人： DYNE THERAPEUTICS, INC.
- 当前专利权人： DYNE THERAPEUTICS, INC.
- 当前专利权人地址： 400 Technology Square, 10th Floor Cambridge, MA 02139 US
- 代理机构： YOOUNG, Daniel, W. et al.
- 优先权： US62/713,933 20180802
- 主分类号： C12N15/00
- IPC分类号： C12N15/00 ; C12N15/09 ; C12N15/63 ; A61K47/00 ; A61K47/50
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 DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.