公开(公告)号：US09057066B2

公开(公告)日：2015-06-16

申请号：US13708839

申请日：2012-12-07

Applicant: Isis Pharmaceuticals, Inc. ,  CHDI Foundation Inc.

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

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

CPC classification number: 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

Abstract: 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.

Abstract translation: 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

Applicant: ISIS PHARMACEUTICALS, INC. ,  CHDI FOUNDATION INC.

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

IPC: C12N15/113

CPC classification number: 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

Abstract: 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

Applicant: Isis Pharmaceuticals, Inc. ,  CHDI Foundation Inc.

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

IPC: C12N15/113

CPC classification number: 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

Abstract: 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.

Abstract translation: 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：US10738307B2

公开(公告)日：2020-08-11

申请号：US15725933

申请日：2017-10-05

Applicant: Ionis Pharmaceuticals, Inc. ,  CHDI Foundation, Inc.

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

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

Abstract: 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

Applicant: Ionis Pharmaceuticals, Inc. ,  CHDI Foundation, Inc.

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

IPC: C12N15/113

Abstract: 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：US20210017522A1

公开(公告)日：2021-01-21

申请号：US16988862

申请日：2020-08-10

Applicant: IONIS PHARMACEUTICALS, Inc. ,  CHDI Foundation, Inc.

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

IPC: C12N15/113

Abstract: 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：US20160348104A1

公开(公告)日：2016-12-01

申请号：US15149642

申请日：2016-05-09

Applicant: ISIS PHARMACEUTICALS, INC.

Inventor： Eric E. Swayze ,  Susan M. Freier ,  Robert A. MacLeod ,  Youngsoo Kim

IPC: C12N15/113

Abstract: Disclosed herein are antisense compounds and methods for decreasing STAT3 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate hyperproliferative diseases.

Abstract translation: 本文公开了用于降低STAT3 mRNA和蛋白质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，预防或改善过度增殖性疾病。

公开(公告)号：US09273315B2

公开(公告)日：2016-03-01

申请号：US14528656

申请日：2014-10-30

Applicant: Isis Pharmaceuticals, Inc.

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

IPC: C07H21/04 ,  C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/3525

Abstract: 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.

Abstract translation: 本文提供了用于降低动物中亨廷丁蛋白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。

公开(公告)号：US20160024496A1

公开(公告)日：2016-01-28

申请号：US14436039

申请日：2013-10-15

Applicant: ISIS PHARMACEUTICALS, INC. ,  THE JOHNS HOPKINS UNIVERSITY

Inventor： C. Frank Bennett ,  Susan M. Freier ,  Jeffrey D. Rothstein ,  Christopher Donnelly ,  Rita G. Sattler

IPC: C12N15/113 ,  A61K31/7088

CPC classification number: C12N15/113 ,  A61K31/7088 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12Q1/6883 ,  C12Q2600/156 ,  C12Q2600/158 ,  C12N2310/3521

Abstract: Disclosed herein are methods for monitoring expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Such C9ORF72 specific inhibitors include antisense compounds.

Abstract translation: 本文公开了用C9ORF72特异性抑制剂监测动物中C9ORF72 mRNA和蛋白质的表达的方法。 这种C9ORF72特异性抑制剂包括反义化合物。

公开(公告)号：US20150291955A1

公开(公告)日：2015-10-15

申请号：US14403953

申请日：2013-06-03

Applicant: Isis Pharmaceuticals, Inc. ,  South West Thames Institute for Renal Research

Inventor： Susan M. Freier ,  Frank Rigo ,  Mark Dockrell ,  Felicia Heidibrecht

IPC: C12N15/113 ,  A61K31/7105

CPC classification number: C12N15/113 ,  A61K31/7105 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/3341 ,  C12N2310/343 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2310/321 ,  C12N2310/3525

Abstract: Provided are compounds comprising oligonucleotides complementary to a fibronectin transcript. Certain such compounds are useful for hybridizing to a fibronectin transcript, including but not limited to a fibronectin transcript in a cell. Such hybridization results in modulation of splicing of the fibronectin transcript. Such compounds are used to treat one or more symptoms associated with fibrosis. Such compounds are used to treat one or more symptoms associated with renal fibrosis.

Abstract translation: 提供了包含与纤连蛋白转录物互补的寡核苷酸的化合物。 某些这样的化合物可用于与纤连蛋白转录物杂交，包括但不限于细胞中的纤连蛋白转录物。 这种杂交导致纤连蛋白转录物剪接的调节。 这些化合物用于治疗与纤维化相关的一种或多种症状。 这些化合物用于治疗与肾纤维化相关的一种或多种症状。