- 专利标题： Oligomeric compounds for reducing DMPK expression
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申请号： US17823854申请日： 2022-08-31
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公开(公告)号： US11833221B2公开(公告)日： 2023-12-05
- 发明人: Frank Rigo , Chrissa A. Dwyer
- 申请人： Ionis Pharmaceuticals, Inc.
- 申请人地址： US CA Carlsbad
- 专利权人： Ionis Pharmaceuticals, Inc.
- 当前专利权人： Ionis Pharmaceuticals, Inc.
- 当前专利权人地址： US CA Carlsbad
- 代理机构： McNeill Baur PLLC
- 主分类号： A61K47/68
- IPC分类号： A61K47/68 ; A61K47/54 ; C12N15/113
摘要：
Provided are oligomeric compounds, methods, and pharmaceutical compositions for DMPK the amount or activity of DMPK RNA in a cell or animal, and in certain instances reducing the amount of DMPK protein in a cell or animal. Such oligomeric compounds, methods, and pharmaceutical compositions are useful to treat type 1 myotonic dystrophy.
公开/授权文献
- US20230114429A1 COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION 公开/授权日：2023-04-13
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