公开(公告)号：EP3031920A1

公开(公告)日：2016-06-15

申请号：EP16153949.9

申请日：2011-07-19

申请人： Ionis Pharmaceuticals, Inc. ,  University of Rochester

发明人： BENNETT, C. Frank ,  FREIER, Susan M. ,  MACLEOD, Robert A. ,  PANDEY, Sanjey K. ,  THORNTON, Charles A. ,  WHEELER, Thurman ,  CHENG, Seng H. ,  LEGER, Andrew ,  WENTWORTH, Bruce M.

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61P21/00

CPC分类号： C12N15/1137 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525 ,  C12Y207/11 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/3527

摘要： Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.

摘要翻译： 本文提供的是减少动物体内DMPK mRNA和蛋白质表达的方法，化合物和组合物。 本文还提供了用于优先减少CUGexp DMPK RNA，减少肌动蛋白或减少动物中的剪接病变的方法，化合物和组合物。 这些方法，化合物和组合物可用于治疗，预防，延迟或改善1型肌强直营养不良症或其症状。

公开(公告)号：EP3633038A3

公开(公告)日：2020-07-29

申请号：EP19191940.6

申请日：2011-07-19

申请人： Ionis Pharmaceuticals, Inc. ,  University of Rochester

发明人： BENNETT, C. Frank ,  FREIER, Susan M. ,  MACLEOD, Robert A. ,  PANDEY, Sanjey K. ,  THORNTON, Charles A. ,  WHEELER, Thurman ,  CHENG, Seng H. ,  LEGER, Andrew ,  WENTWORTH, Bruce M.

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61P21/00

摘要： Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.

公开(公告)号：EP3031920B1

公开(公告)日：2019-08-21

申请号：EP16153949.9

申请日：2011-07-19

申请人： Ionis Pharmaceuticals, Inc. ,  University of Rochester

发明人： BENNETT, C. Frank ,  FREIER, Susan M. ,  MACLEOD, Robert A. ,  PANDEY, Sanjey K. ,  THORNTON, Charles A. ,  WHEELER, Thurman ,  CHENG, Seng H. ,  LEGER, Andrew ,  WENTWORTH, Bruce M.

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61P21/00

公开(公告)号：EP3633038A2

公开(公告)日：2020-04-08

申请号：EP19191940.6

申请日：2011-07-19

申请人： Ionis Pharmaceuticals, Inc. ,  University of Rochester

发明人： BENNETT, C. Frank ,  FREIER, Susan M. ,  MACLEOD, Robert A. ,  PANDEY, Sanjey K. ,  THORNTON, Charles A. ,  WHEELER, Thurman ,  CHENG, Seng H. ,  LEGER, Andrew ,  WENTWORTH, Bruce M.

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61P21/00

摘要： Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.