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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
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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.
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).
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公开(公告)号：EP3626823A1
公开(公告)日：2020-03-25
申请号：EP19199744.4
申请日：2010-09-10
发明人： HUNG, Gene , BENNETT, C. Frank , FREIER, Susan M. , KORDASIEWICZ, Holly , STANEK, Lisa , CLEVELAND, Don W. , CHENG, Seng H. , SHIHABUDDIN, Lamya
IPC分类号： C12N15/113
摘要： Provided herein are methods, compounds, and compositions for reducing expression of hunting-tin mRNA and protein in an animal. Such methods, compounds and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
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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
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.
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公开(公告)号：EP3178933B1
公开(公告)日：2019-11-13
申请号：EP16197941.4
申请日：2010-09-10
发明人： HUNG, Gene , BENNETT, C. Frank , FREIER, Susan M. , KORDASIEWICZ, Holly , STANEK, Lisa , CLEVELAND, Don W. , CHENG, Seng H. , SHIHABUDDIN, Lamya
IPC分类号： C12N15/113
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公开(公告)号：EP3178933A1
公开(公告)日：2017-06-14
申请号：EP16197941.4
申请日：2010-09-10
发明人： HUNG, Gene , BENNETT, C. Frank , FREIER, Susan M. , KORDASIEWICZ, Holly , STANEK, Lisa , CLEVELAND, Don W. , CHENG, Seng H. , SHIHABUDDIN, Lamya
IPC分类号： C12N15/113
摘要： Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
摘要翻译： 本文提供了用于减少动物中亨廷丁蛋白mRNA和蛋白质表达的方法，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，延迟或改善亨廷顿病或其症状。
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公开(公告)号：EP3978610A2
公开(公告)日：2022-04-06
申请号：EP21187734.5
申请日：2015-03-19
发明人： FREIER, Susan, M. , HUNG, Gene , BENNETT, C. Frank
IPC分类号： C12N15/113
摘要： Disclosed herein are antisense compounds and methods for decreasing Ataxin 2 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotropic sclerosis (ALS), and parkinsonism.
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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
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.
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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
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
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公开(公告)号：EP3321361A1
公开(公告)日：2018-05-16
申请号：EP17206749.8
申请日：2011-02-08
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2320/34
摘要： Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).
摘要翻译： 本文公开了用于选择性减少含有单核苷酸多态性（SNP）的基因的等位基因变体的表达的反义化合物和方法。 这些方法，化合物和组合物可用于治疗，预防或改善疾病，包括神经退行性疾病，如亨廷顿病（HD）。
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