公开(公告)号：US09175291B2

公开(公告)日：2015-11-03

申请号：US14050574

申请日：2013-10-10

Applicant: ISIS Pharmaceuticals Inc.

Inventor： Robert A. Macleod ,  Youngsoo Kim ,  Tianyuan Zhou ,  Susan M. Freier ,  Brett Monia

IPC: C12N15/11 ,  C12N15/113 ,  A61K31/7088 ,  A61K31/4166

CPC classification number: C12N15/1138 ,  A61K31/4166 ,  A61K31/7088 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/30 ,  C12N2310/3525

Abstract: Certain embodiments are directed to compounds and compositions targeted to human androgen receptor (AR) for inhibiting androgen receptor levels in a cell, which can be useful for methods of treating cancer and inhibiting cancer cell growth or proliferation.

Abstract translation: 某些实施方案涉及靶向人雄激素受体（AR）的化合物和组合物，用于抑制细胞中的雄激素受体水平，其可用于治疗癌症和抑制癌细胞生长或增殖的方法。

公开(公告)号：US20150176005A1

公开(公告)日：2015-06-25

申请号：US14403103

申请日：2013-05-22

Applicant: Christopher K. GLASS ,  Michael G. ROSENFELD ,  Wenbo LI ,  Michael t. LAM ,  Isis Pharmaceuticals, Inc. ,  The Regents of the University of California

Inventor： Susan M. Freier ,  Christopher K. Glass ,  Michael G. Rosenfeld ,  Wenbo Li ,  Michael T. Lam

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N15/85 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/321 ,  C12N2310/3525

Abstract: Disclosed herein are methods and compounds for inhibiting gene expression by inhibiting enhancer RNAs (eRNAs). Such methods and compounds are useful for reducing expression of certain genes, many of which are associated with a variety of diseases and disorders.

Abstract translation: 本文公开了通过抑制增强子RNA（eRNA）抑制基因表达的方法和化合物。 这些方法和化合物可用于减少某些基因的表达，其中许多基因与多种疾病和病症有关。

公开(公告)号：US08952145B2

公开(公告)日：2015-02-10

申请号：US13789368

申请日：2013-03-07

Applicant: Isis Pharmaceuticals, Inc.

Inventor： Susan M. Freier

IPC: C07H21/04 ,  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 (HD) progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntington's Disease (HD) in an individual susceptible to Huntington's Disease (HD). 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缓或预防亨廷顿病（HD）进展的方法。 另外提供的是延迟或预防亨廷顿舞蹈病（HD）易感人群亨廷顿病（HD）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：US20140309282A1

公开(公告)日：2014-10-16

申请号：US14315186

申请日：2014-06-25

Applicant: Isis Pharmaceuticals, Inc.

Inventor： C. Frank Bennett ,  Kenneth W. Dobie ,  Susan M. Freier ,  Stanley T. Crooke

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2320/33

Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds.

Abstract translation: 本文公开了用于调节细胞，组织或动物中LMNA表达的化合物，组合物和方法。 还提供了目标验证的方法。 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。 还提供了使用所公开的化合物鉴定所选mRNA的顺式剪接调节元件的方法。

公开(公告)号：US20130190384A1

公开(公告)日：2013-07-25

申请号：US13693939

申请日：2012-12-04

Applicant: Isis Pharmaceuticals, Inc.

Inventor： Susan M. Freier ,  Brett P. Monia ,  Hong Zhang ,  Chenguang Zhao ,  Jeffrey R. Crosby ,  Andrew M. Siwkowski

IPC: C12N15/113

CPC classification number: A61K31/7125 ,  A61K31/366 ,  A61K31/4365 ,  A61K31/4545 ,  A61K31/713 ,  A61K31/727 ,  A61K31/737 ,  C07H21/00 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12Y304/21027

Abstract: Disclosed herein are antisense compounds and methods for decreasing Factor 11 and treating or preventing thromboembolic complications in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to Factor 11 include thrombosis, embolism, and thromboembolism, such as, deep vein thrombosis, pulmonary embolism, myocardial infarction, and stroke. Antisense compounds targeting Factor 11 can also be used as a prophylactic treatment to prevent individuals at risk for thrombosis and embolism.

Abstract translation: 本文公开了用于减少因子11并在需要其的个体中治疗或预防血栓栓塞并发症的反义化合物和方法。 通过施用靶向因子11的反义化合物可以改善的疾病状况的实例包括血栓形成，栓塞和血栓栓塞，例如深静脉血栓形成，肺栓塞，心肌梗塞和中风。 靶向因子11的反义化合物也可用作预防性治疗，以预防患有血栓形成和栓塞风险的个体。

公开(公告)号：US20130143943A1

公开(公告)日：2013-06-06

申请号：US13650801

申请日：2012-10-12

Applicant: Isis Pharmaceuticals, Inc.

Inventor： Sanjay Bhanot ,  Susan M. Freier ,  Kenneth W. Dobie

IPC: C07H21/04

CPC classification number: C07H21/04 ,  A61K38/00 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12Q1/6883 ,  C12Q2600/136 ,  C12Q2600/158 ,  C12N2310/3525

Abstract: Compounds, compositions and methods are provided for modulating the expression of glucocorticoid receptor. The compositions comprise oligonucleotides, targeted to nucleic acid encoding glucocorticoid receptor. Methods of using these compounds for modulation of glucocorticoid receptor expression and for diagnosis and treatment of diseases and conditions associated with expression of glucocorticoid receptor are provided.

Abstract translation: 提供化合物，组合物和方法用于调节糖皮质激素受体的表达。 组合物包含靶向编码糖皮质激素受体的核酸的寡核苷酸。 提供了使用这些化合物调节糖皮质激素受体表达和诊断和治疗与糖皮质激素受体表达相关的疾病和病症的方法。

公开(公告)号：US20130116301A1

公开(公告)日：2013-05-09

申请号：US13623337

申请日：2012-09-20

Applicant: Isis Pharmaceuticals, Inc.

Inventor： Susan M. Freier ,  Sanjay Bhanot

IPC: C12N15/113

CPC classification number: C12N15/1138 ,  A61K31/711 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/3525

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of GCGR mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate metabolic disease, for example, diabetes, or a symptom thereof.

公开(公告)号：US10479993B2

公开(公告)日：2019-11-19

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: C07H21/00 ,  C12N15/113 ,  C07H21/04 ,  C12N15/00 ,  C12N15/11

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.

公开(公告)号：US09765338B2

公开(公告)日：2017-09-19

申请号：US14814174

申请日：2015-07-30

Applicant: Isis Pharmaceuticals, Inc. ,  University Of Rochester

Inventor： C. Frank Bennett ,  Susan M. Freier ,  Robert A. MacLeod ,  Sanjay K. Pandey ,  Charles A. Thornton ,  Thurman Wheeler ,  Seng H. Cheng ,  Andrew Leger ,  Bruce M. Wentworth

IPC: C12N15/11 ,  C12N15/113

CPC classification number: C12N15/1137 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525 ,  C12Y207/11 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/3527

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.

公开(公告)号：US09359608B2

公开(公告)日：2016-06-07

申请号：US14338880

申请日：2014-07-23

Applicant: ISIS PHARMACEUTICALS, INC.

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

IPC: C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  C07H21/00 ,  C12N15/00 ,  C12N15/11

CPC classification number: C12N15/113 ,  C07H21/00 ,  C07H21/04 ,  C12N15/00 ,  C12N15/11 ,  C12N15/1135 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/30 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/3525

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。