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：US11834660B2

公开(公告)日：2023-12-05

申请号：US17393738

申请日：2021-08-04

申请人： Ionis Pharmaceuticals, Inc.

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

IPC分类号： C12N15/113 ,  A61K48/00

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/321 ,  C12N2310/3525

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.

公开(公告)号：US10006027B2

公开(公告)日：2018-06-26

申请号：US15127352

申请日：2015-03-19

申请人： Ionis Pharmaceuticals, Inc. ,  University of Utah Research Foundation

发明人： C. Frank Bennett ,  Susan M. Freier ,  Stefan M. Pulst ,  Daniel R. Scoles ,  Gene Hung

IPC分类号： C07H21/02 ,  C07H21/04 ,  A61K31/70 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525

摘要： Disclosed herein are methods for decreasing Ataxin 2 mRNA and protein expression. Such methods 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), amyotrophic lateral sclerosis (ALS), and parkinsonism. Provided are methods for reducing expression of Ataxin 2 (ATXN2) mRNA and protein in an animal. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases, including spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism by inhibiting expression of Ataxin 2 in an animal.

公开(公告)号：US20180087052A1

公开(公告)日：2018-03-29

申请号：US15596249

申请日：2017-05-16

申请人： Ionis Pharmaceuticals, Inc.

发明人： Gene Hung ,  C. Frank Bennett ,  Susan M. Freier ,  Holly Kordasiewicz ,  Lisa Stanek ,  Don W. Cleveland ,  Seng H. Cheng ,  Lamya Shihabuddin

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/3525

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.

公开(公告)号：US10533178B2

公开(公告)日：2020-01-14

申请号：US15973088

申请日：2018-05-07

申请人： Ionis Pharmaceuticals, Inc. ,  University of Utah Research Foundation

发明人： C. Frank Bennett ,  Susan M. Freier ,  Stefan M. Pulst ,  Daniel R. Scoles ,  Gene Hung

IPC分类号： C12N15/113 ,  A61K48/00 ,  C12N5/00

摘要： Disclosed herein are methods for decreasing Ataxin 2 mRNA and protein expression. Such methods 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), amyotrophic lateral sclerosis (ALS), and parkinsonism.