公开(公告)号：EP2534262A2

公开(公告)日：2012-12-19

申请号：EP11740543.1

申请日：2011-02-08

申请人： Isis Pharmaceuticals, Inc. ,  The University Of British Columbia

发明人： BENNETT, C., Frank ,  HAYDEN, Michael ,  FREIER, Susan, M. ,  GREENLEE, Sarah ,  CARROLL, Jeffrey ,  WARBY, Simon ,  SWAYZE, Eric, E.

IPC分类号： C12Q1/68 ,  C12N15/11 ,  C07H21/04

CPC分类号： C12Q1/6883 ,  C12N15/113 ,  C12N2310/11 ,  C12N2320/34 ,  C12Q1/6811 ,  C12Q1/6897 ,  C12Q2600/136 ,  C12Q2600/156 ,  C12Q2600/158 ,  C12Q2525/207 ,  C12Q2535/131 ,  C12Q2539/107 ,  C12Q2521/327 ,  C12Q2525/121 ,  C12Q2525/185

摘要： Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).

公开(公告)号：EP2534262B1

公开(公告)日：2016-12-14

申请号：EP11740543.1

申请日：2011-02-08

申请人： Ionis Pharmaceuticals, Inc. ,  The University Of British Columbia

发明人： BENNETT, C., Frank ,  HAYDEN, Michael ,  FREIER, Susan, M. ,  GREENLEE, Sarah ,  CARROLL, Jeffrey ,  WARBY, Simon ,  SWAYZE, Eric, E.

IPC分类号： C12Q1/68 ,  C12N15/11 ,  C07H21/04

CPC分类号： C12Q1/6883 ,  C12N15/113 ,  C12N2310/11 ,  C12N2320/34 ,  C12Q1/6811 ,  C12Q1/6897 ,  C12Q2600/136 ,  C12Q2600/156 ,  C12Q2600/158 ,  C12Q2525/207 ,  C12Q2535/131 ,  C12Q2539/107 ,  C12Q2521/327 ,  C12Q2525/121 ,  C12Q2525/185

摘要： Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).

公开(公告)号：EP2297341A1

公开(公告)日：2011-03-23

申请号：EP09741640.8

申请日：2009-05-08

申请人： The University of British Columbia

发明人： HAYDEN, Michael ,  CARROLL, Jeffrey ,  WARBY, Simon

IPC分类号： C12Q1/68 ,  A61K31/7088 ,  A61K31/713 ,  A61P25/14 ,  C12N15/63 ,  C12N15/85 ,  G01N33/53 ,  G01N33/68 ,  C07H21/00 ,  C12N15/11

CPC分类号： A61K31/713 ,  A61K48/00 ,  C12N15/113 ,  C12N2310/11

摘要： Methods and compositions for reducing expression of a mutant huntingtin (mHTT) protein in a cell are provided. Such methods include contacting the cell with an effective amount of a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding the mHTT.

公开(公告)号：EP3208347B1

公开(公告)日：2019-08-14

申请号：EP16203872.3

申请日：2011-02-08

申请人： Ionis Pharmaceuticals, Inc. ,  The University Of British Columbia

发明人： BENNETT, C., Frank ,  HAYDEN, Michael ,  FREIER, Susan, M. ,  GREENLEE, Sarah ,  CARROLL, Jeffrey ,  WARBY, Simon ,  SWAYZE, Eric, E.

IPC分类号： C12Q1/68 ,  C12N15/11 ,  C07H21/04 ,  C12Q1/6811

公开(公告)号：EP3628750A1

公开(公告)日：2020-04-01

申请号：EP19191293.0

申请日：2011-02-08

申请人： Ionis Pharmaceuticals, Inc. ,  The University Of British Columbia

发明人： BENNETT, Frank, C. ,  HAYDEN, Michael ,  FREIER, Susan, M. ,  GREENLEE, Sarah ,  SWAYZE, Eric, E. ,  CARROLL, Jeffrey ,  WARBY, Simon

IPC分类号： C12Q1/6897

摘要： Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).