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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.
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.
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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：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：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
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公开(公告)号：EP4396351A1
公开(公告)日：2024-07-10
申请号：EP22865773.0
申请日：2022-08-31
发明人： RIGO, Frank , SWAYZE, Eric, E. , JAFAR-NEJAD, Paymaan , OESTERGAARD, Michael , LING, Kar, Yun Karen , FREIER, Susan, M. , BUI, Huynh-Hoa
IPC分类号： C12N15/113 , A61K31/7125 , A61K9/00
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公开(公告)号：EP4206213A1
公开(公告)日：2023-07-05
申请号：EP22206076.6
申请日：2017-07-17
摘要： Disclosed herein are compounds, compositions and compounds for use in modulating splicing of SMN2, said compounds consisting in oligomers comprising a modified oligonucleotide consisting of 14-25 linked nucleosides, wherein the modified oligonucleotide is complementary to an SMN2 pre-mRNA; and wherein at least one nucleoside of the modified oligonucleotide comprises a 2'-O(N-alkyl acetamide) modified sugar moiety. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of spinal muscular atrophy.
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公开(公告)号：EP3766974A1
公开(公告)日：2021-01-20
申请号：EP20181501.6
申请日：2014-03-14
发明人： RIGO, Frank , BENNETT, C., Frank
IPC分类号： C12N15/113 , C12Q1/68
摘要： Disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds. Also disclosed herein are methods for reducing expression of Tau mRNA and protein in an animal with Tau antisense compounds. Such compounds and methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration Tau antisense oligonucleotides include Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy, Chronic Traumatic Encephalopathy, Epilepsy, and Dravet's Syndrome.
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公开(公告)号：EP3389670A1
公开(公告)日：2018-10-24
申请号：EP16871732.0
申请日：2016-12-05
发明人： SPECTOR, David, L. , DIERMEIER, Sarah, Daniela , RIGO, Frank , BENNETT, C., Frank , FREIER, Susan, M. , ARUN, Gayatri , CHANG, Kung-chi
IPC分类号： A61K31/7088 , A61P35/00 , C12N15/11 , C12N15/113
CPC分类号： A61K31/713 , A61K45/06 , C12N15/113 , C12N2310/113 , C12N2310/315 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , A61K2300/00 , C12N2310/321 , C12N2310/3525
摘要： Provided herein are methods, compounds, and compositions for reducing expression of a MaTAR in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate breast cancer in an individual in need.
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公开(公告)号：EP3353303A1
公开(公告)日：2018-08-01
申请号：EP16849742.8
申请日：2016-09-23
发明人： RIGO, Frank , PRAKASH, Thazha, P. , VAN ROON-MOM, Wilhelmina Maria, Clasina , TOONEN, Lodewijk Julius, Anton
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/712 , C12N2310/11 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/3525
摘要： Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.
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