公开(公告)号：US20160002627A1

公开(公告)日：2016-01-07

申请号：US14760171

申请日：2014-01-09

申请人： ISIS PHARMACEUTICALS, INC.

发明人： C. Frank Bennett ,  Gene Hung ,  Frank Rigo

IPC分类号： C12N15/113 ,  A61K9/00 ,  A61K31/7088 ,  A61K31/185 ,  A61K35/545 ,  A61B5/04 ,  A61K48/00 ,  A61K45/06 ,  C12Q1/68 ,  G01N33/50 ,  A61B5/0488 ,  A61K47/46 ,  A61K38/30

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： 本文公开了用于调节受试者中SMN2 mRNA的剪接的化合物，组合物和方法。 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（包括脊髓性肌肉萎缩）的药物中的用途。

公开(公告)号：US09273315B2

公开(公告)日：2016-03-01

申请号：US14528656

申请日：2014-10-30

申请人： Isis 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： C07H21/04 ,  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.

摘要翻译： 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：US20150284725A1

公开(公告)日：2015-10-08

申请号：US14434693

申请日：2013-10-11

申请人： ISIS PHARMACEUTICALS, INC.

发明人： Gene Hung

IPC分类号： C12N15/113

CPC分类号： C12N15/1138 ,  A61K31/713 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2320/30

摘要： Certain embodiments are directed to methods of ameliorating, treating, or preventing Kennedy's Disease in a subject carrying a mutation in the Androgen Receptor (AR) gene, such as expansion of a CAG trinucleotide repeat, which is associated with Kennedy's Disease, by administering an antisense compound targeted to AR. Several embodiments provided herein relate to the discovery that antisense compounds targeting Androgen Receptor can ameliorate, treat, or prevent Kennedy's Disease in a subject carrying a mutation in the Androgen Receptor gene, such as expansion of a CAG trinucleotide repeat, which is associated with Kennedy's Disease.

摘要翻译： 某些实施方案涉及在携带雄激素受体（AR）基因中的突变的受试者中改善，治疗或预防肯尼迪氏病的方法，例如与肯尼迪氏病相关的CAG三核苷酸重复的扩增，通过施用反义 化合物靶向AR。 本文提供的几个实施方案涉及以下发现：靶向雄激素受体的反义化合物可改善，治疗或预防在雄激素受体基因中携带突变的受试者中的肯尼迪氏病，例如与肯尼迪氏病相关的CAG三核苷酸重复的扩增 。

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：US20150159155A1

公开(公告)日：2015-06-11

申请号：US14528656

申请日：2014-10-30

申请人： Isis 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.

摘要翻译： 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：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。