公开(公告)号：EP2906696B1

公开(公告)日：2019-10-09

申请号：EP13846313.8

申请日：2013-10-15

申请人： Ionis Pharmaceuticals, Inc. ,  Ludwig Institute for Cancer Research ,  The Regents of the University of California

发明人： RIGO, Frank ,  CLEVELAND, Don W. ,  LAGIER-TOURENNE, Clotilde ,  RAVITS, John M. ,  BENNETT, C. Frank ,  FREIER, SUSAN M. ,  BAUGHN, Michael W.

IPC分类号： C12N15/113 ,  A61P25/28

公开(公告)号：EP3058069B1

公开(公告)日：2019-07-24

申请号：EP14854442.2

申请日：2014-10-14

申请人： Ionis Pharmaceuticals, Inc. ,  The Regents of the University of California ,  Ludwig Institute For Cancer Research

发明人： BENNETT, C., Frank ,  RIGO, Frank ,  CLEVELAND, Don, W. ,  LAGIER-TOURENNE, Clotilde ,  RAVITS, John, M. ,  BAUGHN, Michael, W.

IPC分类号： C12N15/113

公开(公告)号：EP3058069A1

公开(公告)日：2016-08-24

申请号：EP14854442.2

申请日：2014-10-14

申请人： Ionis Pharmaceuticals, Inc. ,  The Regents of the University of California ,  Ludwig Institute For Cancer Research Limited

发明人： BENNETT, C., Frank ,  RIGO, Frank ,  CLEVELAND, Don, W. ,  LAGIER-TOURENNE, Clotilde ,  RAVITS, John, M. ,  BAUGHN, Michael, W.

IPC分类号： C12N15/11 ,  C07H21/04 ,  A61K38/00

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525

摘要： Disclosed herein are methods for reducing expression of C90RF72 antisense transcript in an animal with C90RF72 antisense transcript specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 antisense transcript specific inhibitors include antisense compounds.

公开(公告)号：EP2906696A1

公开(公告)日：2015-08-19

申请号：EP13846313.8

申请日：2013-10-15

申请人： Isis Pharmaceuticals, Inc. ,  Ludwig Institute For Cancer Research Limited ,  The Regents of the University of California

发明人： RIGO, Frank ,  CLEVELAND, Don W. ,  LAGIER-TOURENNE, Clotilde ,  RAVITS, John M. ,  BENNETT, C. Frank ,  FREIER, SUSAN M. ,  BAUGHN, Michael W.

IPC分类号： C12N15/11 ,  A61K48/00

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/3181 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/321 ,  C12N2310/3525

摘要： Disclosed herein are methods for reducing expression of C90RF72 mRNA and protein in an animal with C90RF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C90RF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).