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1.发明申请
公开(公告)号：WO2015017675A3
公开(公告)日：2015-04-02
申请号：PCT/US2014049197
申请日：2014-07-31
发明人： PRAKASH THAZHA P , HU JIAXIN , LIU JING , YU DONGBO , COREY DAVID , SWAYZE ERIC E
IPC分类号： C07D471/04
CPC分类号： C07H21/02 , C12N15/113 , C12N2310/11 , C12N2310/32 , C12N2320/34
摘要： Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules. In certain embodiments, compounds and methods useful for the modulation of ATXN-3 pre-mRNA are described. In certain embodiments, compounds and methods useful for the modulation of ATN-1 mRNA are described.
摘要翻译： 描述了用于治疗和研究与包含扩增重复的RNA分子相关的疾病和病症的化合物和方法。 在某些实施方案中，描述了用于调节ATXN-3前体mRNA的化合物和方法。 在某些实施方案中，描述了用于调节ATN-1mRNA的化合物和方法。
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2.发明申请COMPOSITIONS AND METHODS FOR MODULATING GROWTH HORMONE RECEPTOR EXPRESSION 审中-公开用于调节生长激素受体表达的组合物和方法
公开(公告)号：WO2015168618A3
公开(公告)日：2015-12-23
申请号：PCT/US2015028887
申请日：2015-05-01
发明人： PRAKASH THAZHA P , SETH PUNIT P , SWAYZE ERIC E , BHANOT SANJAY , FREIER SUSAN M , BUI HUYNH-HOA
CPC分类号： C12N15/1138 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3525
摘要： The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating a disease associated with excess growth hormone using antisense compounds oligonucleotides targeted to growth hormone receptor (GHR).
摘要翻译： 本实施方案提供了使用针对生长激素受体（GHR）的反义寡核苷酸治疗，预防，改善与过量生长激素相关疾病的方法，化合物和组合物。
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3.发明申请
公开(公告)号：WO2015168589A2
公开(公告)日：2015-11-05
申请号：PCT/US2015028837
申请日：2015-05-01
发明人： PRAKASH THAZHA P , SETH PUNIT P , SWAYZE ERIC E , FREIER SUSAN M , GRAHAM MARK J , CROOKE ROSANNE M
CPC分类号： C07H19/20 , C07H15/04 , C07H21/02 , C07H21/04 , C12N15/1136 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2320/32 , C12N2310/3525 , C12N2310/3231
摘要： Provided herein are methods, compounds, and compositions for reducing expression of an ANGPTL3 mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for reducing lipids and/or glucose in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate any one or more of cardiovascular disease and/or metabolic disease, or a symptom thereof, in an individual in need thereof.
摘要翻译： 本文提供了用于降低动物中ANGPTL3mRNA和蛋白质表达的方法，化合物和组合物。 本文还提供了用于减少动物中的脂质和/或葡萄糖的方法，化合物和组合物。 此类方法，化合物和组合物可用于在有需要的个体中治疗，预防，延缓或改善心血管疾病和/或代谢疾病或其症状中的任何一种或多种。
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4.发明申请MODIFIED 5' DIPHOSPHATE NUCLEOSIDES AND OLIGOMERIC COMPOUNDS PREPARED THEREFROM 审中-公开改良的5'二磷酸核苷和其制备的低聚物
公开(公告)号：WO2011139695A3
公开(公告)日：2013-05-16
申请号：PCT/US2011033955
申请日：2011-04-26
申请人： ISIS PHARMACEUTICALS INC , ALNYLAM PHARMACEUTICALS INC , PRAKASH THAZHA P , SETH PUNIT P , MANORHARAN MUTHIAH , SWAYZE ERIC E , RAJEEV KALLANTHOTTATHIL G , ZLATEV IVAN
发明人： PRAKASH THAZHA P , SETH PUNIT P , MANORHARAN MUTHIAH , SWAYZE ERIC E , RAJEEV KALLANTHOTTATHIL G , ZLATEV IVAN
IPC分类号： C07H19/10
CPC分类号： C07H19/10 , C12N15/113
摘要： Provided herein are modified 5' diphosphate nucleosides and oligomeric compounds prepared therefrom. More particularly, modified 5' diphosphate nucleosides are provided that can be further modified at the 2' and 5' positions. In some embodiments, the oligomeric compounds provided herein are expected to hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The oligomeric compounds are also expected to be useful as primers and probes in diagnostic applications.
摘要翻译： 本文提供修饰的5'二磷酸核苷和由其制备的低聚化合物。 更具体地，提供修饰的5'-二磷酸核苷，其可以在2'和5'位置进一步修饰。 在一些实施方案中，预期本文提供的寡聚化合物与靶RNA的一部分杂交，导致靶RNA的正常功能丧失。 还预期寡聚化合物可用作诊断应用中的引物和探针。
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公开(公告)号：WO2007136988A8
公开(公告)日：2009-09-24
申请号：PCT/US2007068410
申请日：2007-05-07
申请人： ISIS PHARMACEUTICALS INC , BHANOT SANJAY , GEARY RICHARD S , MCKAY ROBERT , MONIA BRETT P , SETH PUNIT P , SIWKOWSKI ANDREW M , SWAYZE ERIC E , WANCEWICZ EDWARD
发明人： BHANOT SANJAY , GEARY RICHARD S , MCKAY ROBERT , MONIA BRETT P , SETH PUNIT P , SIWKOWSKI ANDREW M , SWAYZE ERIC E , WANCEWICZ EDWARD
IPC分类号： A61K31/70 , C07H21/04 , C12N5/00 , C12N15/113
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3515 , C12Y203/0102 , C12N2310/3521
摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
摘要翻译： 本公开描述了短反义化合物，包括长度为化学修饰的高亲和力单体8-16单体的这类化合物。 某些这样的短反义化合物可用于在增加的效力和改善的治疗指数的细胞，组织和动物中还原靶核酸和/或蛋白质。 因此，本文提供了包含用于在体内降低靶RNA的高亲和力核苷酸修饰的短反义化合物。 这样的短反义化合物在比以前描述的反义化合物低的剂量下是有效的，允许降低毒性和治疗成本。 此外，所述短反义化合物具有更大的口服给药潜力。
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公开(公告)号：WO2009023855A2
公开(公告)日：2009-02-19
申请号：PCT/US2008073379
申请日：2008-08-15
申请人： ISIS PHARMACEUTICALS INC , ALLERSON CHARLES , BHAT BALKRISHEN , PRAKASH THAZHA P , SWAYZE ERIC E , SIWKOWSKI ANDREW M , SETH PUNIT P
发明人： ALLERSON CHARLES , BHAT BALKRISHEN , PRAKASH THAZHA P , SWAYZE ERIC E , SIWKOWSKI ANDREW M , SETH PUNIT P
IPC分类号： C12N15/11
CPC分类号： C12N15/113 , C07H19/10 , C07H19/20 , C07H21/00 , C12N15/111 , C12N2310/323 , C12N2320/51
摘要： The present disclosure describes tetrahydropyran nucleoside analogs, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, tetrahydropyran nucleoside analogs are provided, having one or more chiral substituents, that are useful for enhancing properties of oligomeric compounds including nuclease resistance and binding affinity. In some embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.
摘要翻译： 本公开描述了四氢吡喃核苷类似物，由其制备的寡聚化合物以及使用该寡聚化合物的方法。 更具体地说，提供了具有一个或多个手性取代基的四氢吡喃核苷类似物，其可用于增强寡聚化合物的性质，包括核酸酶抗性和结合亲和力。 在一些实施方案中，本文提供的寡聚化合物与靶RNA的一部分杂交，导致靶RNA的正常功能丧失。
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公开(公告)号：WO2007131237A3
公开(公告)日：2008-11-13
申请号：PCT/US2007068402
申请日：2007-05-07
申请人： ISIS PHARMACEUTICALS INC , BHANOT SANJAY , GEARY RICHARD S , MCKAY ROBERT , MONIA BRETT P , SETH PUNIT P , SIWKOWSKI ANDREW M , SWAYZE ERIC E , WANCEWICZ EDWARD
发明人： BHANOT SANJAY , GEARY RICHARD S , MCKAY ROBERT , MONIA BRETT P , SETH PUNIT P , SIWKOWSKI ANDREW M , SWAYZE ERIC E , WANCEWICZ EDWARD
IPC分类号： C12Q1/68 , C12N5/16 , C12N15/113
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3515 , C12Y203/0102 , C12N2310/3521
摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
摘要翻译： 本公开描述了短反义化合物，包括长度为化学修饰的高亲和力单体8-16单体的这类化合物。 某些这样的短反义化合物可用于在增加的效力和改善的治疗指数的细胞，组织和动物中还原靶核酸和/或蛋白质。 因此，本文提供了包含用于在体内降低靶RNA的高亲和力核苷酸修饰的短反义化合物。 这样的短反义化合物在比以前描述的反义化合物低的剂量下是有效的，允许降低毒性和治疗成本。 此外，所述短反义化合物具有更大的口服给药潜力。
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公开(公告)号：WO2008094945A3
公开(公告)日：2008-10-16
申请号：PCT/US2008052361
申请日：2008-01-29
IPC分类号： C07H21/04
CPC分类号： C07H21/00 , C12N15/113 , C12N2310/315 , C12N2310/322 , C12N2310/344 , C12N2310/346 , C12N2310/3533
摘要： The present invention includes compositions and methods useful for modulating protein expression. In certain embodiments, the present invention includes oligomeric compounds comprising modified nucleosides and modified internucleoside linkages.
摘要翻译： 本发明包括用于调节蛋白质表达的组合物和方法。 在某些实施方案中，本发明包括包含修饰的核苷和修饰的核苷间键的寡聚化合物。
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公开(公告)号：WO2007136989A2
公开(公告)日：2007-11-29
申请号：PCT/US2007068415
申请日：2007-05-07
申请人： ISIS PHARMACEUTICALS INC , BHANOT SANJAY , GEARY RICHARD S , MCKAY ROBERT , MONIA BRETT P , SETH PUNIT P , SIWKOWSKI ANDREW M , SWAYZE ERIC E , WANCEWICZ EDWARD
发明人： BHANOT SANJAY , GEARY RICHARD S , MCKAY ROBERT , MONIA BRETT P , SETH PUNIT P , SIWKOWSKI ANDREW M , SWAYZE ERIC E , WANCEWICZ EDWARD
IPC分类号： A61K48/00 , A61P43/00 , C07H21/04 , C12N15/113
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3515 , C12Y203/0102 , C12N2310/3521
摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
摘要翻译： 本公开描述了短反义化合物，包括长度为化学修饰的高亲和力单体8-16单体的这类化合物。 某些这样的短反义化合物可用于在增加的效力和改善的治疗指数的细胞，组织和动物中还原靶核酸和/或蛋白质。 因此，本文提供了包含用于在体内降低靶RNA的高亲和力核苷酸修饰的短反义化合物。 这样的短反义化合物在比以前描述的反义化合物低的剂量下是有效的，允许降低毒性和治疗成本。 此外，所述短反义化合物具有更大的口服给药潜力。
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10.发明申请NON-PHOSPHOROUS-LINKED OLIGOMERIC COMPOUNDS AND THEIR USE IN GENE MODULATION 审中-公开非磷酸二酯化合物及其在基因调控中的应用
公开(公告)号：WO2004041924A3
公开(公告)日：2005-05-19
申请号：PCT/US0335137
申请日：2003-11-04
申请人： ISIS PHARMACEUTICALS INC , BAKER BRENDA F , ELDRUP ANNE B , MANOHARAN MUTHIAH , BHAT BALKRISHEN , GRIFFEY RICHARD , SWAYZE ERIC E , CROOKE STANLEY T , PRAKASH THAZHA P
发明人： BAKER BRENDA F , ELDRUP ANNE B , MANOHARAN MUTHIAH , BHAT BALKRISHEN , GRIFFEY RICHARD , SWAYZE ERIC E , CROOKE STANLEY T , PRAKASH THAZHA P
IPC分类号： A01N43/04 , A61K31/70 , A61K48/00 , C07H21/02 , C07H21/04 , C08L20060101 , C12N15/113 , C12Q1/68 , C12N15/87
CPC分类号： C12N15/1135 , A61K31/70 , C07H21/04 , C12N15/1137 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/3181 , C12N2310/3183 , C12N2310/322 , C12N2310/323 , C12N2310/341 , C12N2310/346 , C12N2310/53
摘要： Oligomer compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capble of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes at least two nucleoside having a modified non-phosphorous-containing internucleoside linkage. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least two nucleosides of the oligomer has a modified non-phosphorous-containing internucleoside linkage.
摘要翻译： 提供包含第一和第二寡聚物的低聚物组合物，其中至少一部分第一寡聚体能够与至少一部分第二寡聚物杂交，至少一部分第一寡聚体与所选择的寡核苷酸杂交互补和杂交 靶核酸，并且第一或第二寡聚体中的至少一个包含至少两个具有修饰的含有非磷的核苷间键的核苷。 还提供低聚物/蛋白质组合物，其包含与选定的靶核酸互补并且能够与选定的靶核酸杂交的寡聚体和至少一种包含至少一部分RNA诱导的沉默复合物（RISC）的蛋白质，其中至少两个核苷 低聚物具有经修饰的不含磷的核苷间键。
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