发明公开
EP3027617A2 METHODS AND COMPOUNDS USEFUL IN CONDITIONS RELATED TO REPEAT EXPANSION
审中-公开
在与重复扩增有关的条件中使用的方法和化合物
- 专利标题： METHODS AND COMPOUNDS USEFUL IN CONDITIONS RELATED TO REPEAT EXPANSION
- 专利标题（中）： 在与重复扩增有关的条件中使用的方法和化合物
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申请号： EP14832900.6申请日： 2014-07-31
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公开(公告)号： EP3027617A2公开(公告)日： 2016-06-08
- 发明人: PRAKASH, Thazha, P. , HU, Jiaxin , LIU, Jing , YU, Dongbo , COREY, David , SWAYZE, Eric, E.
- 申请人： Ionis Pharmaceuticals, Inc. , Board of Regents of the University of Texas System
- 申请人地址： 2855 Gazelle Court Carlsbad, CA 92010 US
- 专利权人： Ionis Pharmaceuticals, Inc.,Board of Regents of the University of Texas System
- 当前专利权人： Ionis Pharmaceuticals, Inc.,Board of Regents of the University of Texas System
- 当前专利权人地址： 2855 Gazelle Court Carlsbad, CA 92010 US
- 代理机构： Carpmaels & Ransford LLP
- 优先权： US201361860823P 20130731; US201462019820P 20140701
- 国际公布： WO2015017675 20150205
- 主分类号： C07D471/04
- IPC分类号： C07D471/04
摘要：
Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules. In certain embodiments, compounds and methods useful for the modulation of ATXN-3 pre-mRNA are described. In certain embodiments, compounds and methods useful for the modulation of ATN-1 mRNA are described.
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