公开(公告)号：US20210017522A1

公开(公告)日：2021-01-21

申请号：US16988862

申请日：2020-08-10

申请人： IONIS PHARMACEUTICALS, Inc. ,  CHDI Foundation, Inc.

发明人： Gene Hung ,  C. Frank Bennett ,  Janet M. Leeds ,  Susan M. Freier

IPC分类号： C12N15/113

摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

公开(公告)号：US10738307B2

公开(公告)日：2020-08-11

申请号：US15725933

申请日：2017-10-05

申请人： Ionis Pharmaceuticals, Inc. ,  CHDI Foundation, Inc.

发明人： Gene Hung ,  C. Frank Bennett ,  Janet M. Leeds ,  Susan M. Freier

IPC分类号： C07H21/02 ,  A01N43/04 ,  C12N15/113

摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

公开(公告)号：US20180273945A1

公开(公告)日：2018-09-27

申请号：US15725933

申请日：2017-10-05

申请人： Ionis Pharmaceuticals, Inc. ,  CHDI Foundation, Inc.

发明人： Gene Hung ,  C. Frank Bennett ,  Janet M. Leeds ,  Susan M. Freier

IPC分类号： C12N15/113

摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

公开(公告)号：US09057066B2

公开(公告)日：2015-06-16

申请号：US13708839

申请日：2012-12-07

申请人： Isis Pharmaceuticals, Inc. ,  CHDI Foundation Inc.

发明人： Gene Hung ,  Janet M. Leeds ,  C. Frank Bennett ,  Susan M. Freier

IPC分类号： C07H21/02 ,  A01N43/04 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/33 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/30 ,  C12N2310/3525

摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

摘要翻译： 本文公开了用于调节细胞，组织或动物中亨廷丁氏蛋白的表达的化合物，组合物和方法。 还提供了使用靶向亨廷顿汀的反义化合物减慢或预防亨廷顿病进展的方法。 另外提供的方法是延缓或预防在亨廷顿病易感的个体中亨廷顿病发病的方法。 还提供了所公开的化合物和组合物在制备用于治疗疾病和病症的药物中的用途。

公开(公告)号：US20160053256A1

公开(公告)日：2016-02-25

申请号：US14708511

申请日：2015-05-11

申请人： ISIS PHARMACEUTICALS, INC. ,  CHDI FOUNDATION INC.

发明人： Gene Hung ,  C. Frank Bennett ,  Janet M. Leeds ,  Susan M. Freier

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/33 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/30 ,  C12N2310/3525

摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

公开(公告)号：US20130189782A1

公开(公告)日：2013-07-25

申请号：US13708839

申请日：2012-12-07

申请人： Isis Pharmaceuticals, Inc. ,  CHDI Foundation Inc.

发明人： Gene Hung ,  Janet M. Leeds ,  C. Frank Bennett ,  Susan M. Freier

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/33 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/30 ,  C12N2310/3525

摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

摘要翻译： 本文公开了用于调节细胞，组织或动物中亨廷丁氏蛋白的表达的化合物，组合物和方法。 还提供了使用靶向亨廷顿汀的反义化合物减慢或预防亨廷顿病进展的方法。 另外提供的方法是延缓或预防在亨廷顿病易感的个体中亨廷顿病发病的方法。 还提供了所公开的化合物和组合物在制备用于治疗疾病和病症的药物中的用途。

公开(公告)号：US20240327838A1

公开(公告)日：2024-10-03

申请号：US18492683

申请日：2023-10-23

申请人： IONIS PHARMACEUTICALS, INC.

发明人： Jeffrey R. Crosby ,  Shuling Guo ,  Huynh-Hoa Bui ,  Andrew T. Watt ,  Susan M. Freier

IPC分类号： C12N15/113 ,  A61K9/00 ,  A61K31/404 ,  A61K31/47 ,  A61K31/7105 ,  A61K31/713 ,  A61M11/00 ,  A61P11/00

CPC分类号： C12N15/113 ,  A61K9/0019 ,  A61K9/0075 ,  A61K9/0078 ,  A61K31/404 ,  A61K31/47 ,  A61K31/7105 ,  A61K31/713 ,  A61P11/00 ,  A61M11/005 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2320/35

摘要： The present embodiments provide methods, compounds, and compositions useful for inhibiting ENaC expression, which may be useful for treating, preventing, or ameliorating a disease associated with ENaC.

公开(公告)号：US20240318183A1

公开(公告)日：2024-09-26

申请号：US18489316

申请日：2023-10-18

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K38/36 ,  C07H21/04

CPC分类号： C12N15/1137 ,  A61K31/7088 ,  A61K38/36 ,  C07H21/04 ,  C12N15/113 ,  C12Y304/21034 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3525 ,  C12N2320/31 ,  C12N2320/35

摘要： Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

公开(公告)号：US20240309452A1

公开(公告)日：2024-09-19

申请号：US18487664

申请日：2023-10-16

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

发明人： C. Frank Bennett ,  Michael Hayden ,  Susan M. Freier ,  Sarah Greenlee ,  Jeffrey Carroll ,  Simon Warby ,  Eric E. Swayze

IPC分类号： C12Q1/6883 ,  C12N15/113 ,  C12Q1/6811 ,  C12Q1/6897

CPC分类号： C12Q1/6883 ,  C12N15/113 ,  C12Q1/6811 ,  C12Q1/6897 ,  C12N2310/11 ,  C12N2320/34 ,  C12Q2539/107 ,  C12Q2600/136 ,  C12Q2600/156 ,  C12Q2600/158

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：US20240200067A1

公开(公告)日：2024-06-20

申请号：US18330084

申请日：2023-06-06

申请人： Ionis Pharmaceuticals, Inc.

发明人： C. Frank Bennett ,  Susan M. Freier ,  Frank Rigo ,  Eric E. Swayze

IPC分类号： C12N15/113

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

摘要： Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C9ORF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).