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).

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：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：EP2831232A1

公开(公告)日：2015-02-04

申请号：EP13770075.3

申请日：2013-03-14

申请人： Washington University ,  Isis Pharmaceuticals, Inc.

发明人： MILLER, Timothy M. ,  DEVOS, Sarah ,  BENNETT, C. Frank ,  RIGO, Frank

IPC分类号： C12N15/11 ,  C07H21/00

CPC分类号： C12N15/113 ,  C07H21/02 ,  C07H21/04 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525

摘要： Disclosed herein are methods for reducing expression of Tau mRNA and protein in an animal with Tau antisense compounds. Also disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds. Such 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.

摘要翻译： 本文公开了用Tau反义化合物降低动物中Tau mRNA和蛋白质表达的方法。 还公开了用Tau反义化合物调节动物中Tau mRNA的剪接的方法。 此类方法可用于治疗，预防或改善有需要的个体中的神经变性疾病。 可以通过施用Tau反义寡核苷酸治疗，预防和改善的神经变性疾病的实例包括阿尔茨海默氏病，额颞叶痴呆（FTD），FTDP-17，进行性核上性麻痹，慢性创伤性脑病，癫痫和Dravet综合征。

公开(公告)号：EP2971142A1

公开(公告)日：2016-01-20

申请号：EP14767904.7

申请日：2014-03-14

申请人： Isis Pharmaceuticals, Inc.

发明人： RIGO, Frank ,  BENNETT, C., Frank

IPC分类号： 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.

公开(公告)号：EP2943225A1

公开(公告)日：2015-11-18

申请号：EP14737605.7

申请日：2014-01-09

申请人： Isis Pharmaceuticals, Inc.

发明人： BENNETT, C., Frank ,  HUNG, Gene ,  RIGO, Frank

IPC分类号： A61K48/00 ,  A61P21/00

CPC分类号： C12N15/113 ,  A61B5/04012 ,  A61B5/0488 ,  A61K9/0019 ,  A61K9/0085 ,  A61K31/185 ,  A61K31/7088 ,  A61K35/545 ,  A61K38/30 ,  A61K45/06 ,  A61K47/46 ,  A61K48/00 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/351 ,  C12Q1/6883 ,  C12Q2600/156 ,  G01N33/5091

摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.

摘要翻译： 在是这样的化合物，组合物和本发明公开了在受试者中调节SMN2 mRNA的剪接方法。 所以本发明提供了在用于治疗疾病和病症，包括脊髓性肌萎缩的药物中的用途游离缺失盘化合物和组合物的用途。

公开(公告)号：EP2906258A2

公开(公告)日：2015-08-19

申请号：EP13847957.1

申请日：2013-10-15

申请人： Isis Pharmaceuticals, Inc.

发明人： BENNETT, Frank, C. ,  FREIER, Susan, M. ,  SWAYZE, Eric, E. ,  RIGO, Frank

IPC分类号： A61K48/00

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/313 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3233 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525 ,  C12N2310/3533

摘要： Disclosed herein are compositions and 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).

公开(公告)号：EP2802674A1

公开(公告)日：2014-11-19

申请号：EP13735797.6

申请日：2013-01-11

申请人： Isis Pharmaceuticals, Inc. ,  Cold Spring Harbor Laboratory

发明人： BENNETT, C., Frank ,  RIGO, Frank ,  KRAINER, Adrian, R. ,  SINHA, Rahul

IPC分类号： C12Q1/68

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3233 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/33

摘要： The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IΚΑΡ protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IΚΑΡ protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.

公开(公告)号：EP2864479A2

公开(公告)日：2015-04-29

申请号：EP13809694.6

申请日：2013-06-25

申请人： Isis Pharmaceuticals, Inc. ,  Baylor College Of Medicine

发明人： RIGO, Frank ,  WARD, Amanda ,  BEAUDET, Arthur, L. ,  MENG, Linyan

IPC分类号： C12N15/00 ,  A61K31/7088

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2320/30 ,  C12N2330/10 ,  C12N2310/3525

摘要： Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for inducing expression of paternal UBE3A in cells and animals.