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公开(公告)号：US07745609B2
公开(公告)日：2010-06-29
申请号：US11466369
申请日：2006-08-22
CPC分类号： C12N15/1138 , A61K38/00 , A61K48/00 , B01J2219/007 , C07K14/003 , C12N15/1048 , C12N2310/11 , C12N2310/3181 , C12N2310/321 , C12N2310/3513 , C12N2310/52 , C12N2310/3521
摘要： Antisense compounds, compositions and methods are provided for modulating the expression of CD40. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding CD40. Methods of using these compounds for modulation of CD40 expression and for treatment of diseases associated with CD40 are provided.
摘要翻译： 提供反义化合物，组合物和方法用于调节CD40的表达。 组合物包含靶向编码CD40的核酸的反义化合物，特别是反义寡核苷酸。 提供了使用这些化合物调节CD40表达和治疗与CD40相关疾病的方法。
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公开(公告)号：US20120264806A1
公开(公告)日：2012-10-18
申请号：US13148291
申请日：2010-02-05
IPC分类号： A61K31/7088 , A61K31/713
CPC分类号： C12N15/113 , A61K9/0019 , A61K31/7088 , A61K31/721 , A61K47/36 , C12N15/111 , C12N2310/11 , C12N2320/30 , C12N2320/32
摘要： The present invention provides method of optimizing the efficacy and potency of antisense compounds. In certain embodiments, the invention provides assays useful for determining favorable oligonucleotide characteristics and excipients for improved cellular uptake.
摘要翻译： 本发明提供了优化反义化合物的功效和效力的方法。 在某些实施方案中，本发明提供可用于确定有利的寡核苷酸特征的测定和用于改善细胞摄取的赋形
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公开(公告)号：US20120196921A1
公开(公告)日：2012-08-02
申请号：US13300404
申请日：2011-11-18
申请人： Susan M. Freier , Edward Wancewicz , Brett P. Monia , Andrew M. Siwkowski , Lynnetta Watts , Thomas A. Leedom
发明人： Susan M. Freier , Edward Wancewicz , Brett P. Monia , Andrew M. Siwkowski , Lynnetta Watts , Thomas A. Leedom
IPC分类号： A61K31/711 , A61P3/08 , A61K31/7105 , A61P3/10 , C07H21/04 , C07H21/02
CPC分类号： C07H21/02 , A61K31/70 , C07H21/04 , C12N15/111 , C12N15/1136 , C12N15/1138 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2320/32 , C12N2500/40 , C12Q1/6886 , C12Q2600/158 , C12N2310/3525 , C12N2310/3527
摘要： Compounds, compositions and methods are provided for modulating the expression of SGLT2. The compositions comprise oligonucleotides, targeted to nucleic acid encoding SGLT2. Methods of using these compounds for modulation of SGLT2 expression and for diagnosis and treatment of diseases and conditions associated with expression of SGLT2 are provided.
摘要翻译： 提供化合物，组合物和方法用于调节SGLT2的表达。 组合物包含靶向编码SGLT2的核酸的寡核苷酸。 提供了使用这些化合物调节SGLT2表达和用于诊断和治疗与SGLT2表达相关的疾病和病症的方法。
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公开(公告)号：US08188059B2
公开(公告)日：2012-05-29
申请号：US12299609
申请日：2007-05-07
申请人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewicz
发明人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewicz
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：US20120071645A1
公开(公告)日：2012-03-22
申请号：US13308750
申请日：2011-12-01
申请人： Eric E. Swayze , Andrew M. Siwkowski , Balkrishen Bhat , Thazha P. Prakash , Charles Allerson , Punit P. Seth
发明人： Eric E. Swayze , Andrew M. Siwkowski , Balkrishen Bhat , Thazha P. Prakash , Charles Allerson , Punit P. Seth
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，由其制备的低聚化合物和使用寡聚化合物的方法。 更具体地，提供四氢吡喃核苷类似物，其具有一个或多个手性取代基，其可用于增强低聚化合物的性质，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：US20120021515A1
公开(公告)日：2012-01-26
申请号：US13148288
申请日：2010-02-05
申请人： Eric E. Swayze , Andrew M. Siwkowski , Balkrishen Bhat , Thazha P. Prakash , Charles Allerson , Punit P. Seth
发明人： Eric E. Swayze , Andrew M. Siwkowski , Balkrishen Bhat , Thazha P. Prakash , Charles Allerson , Punit P. Seth
CPC分类号： C07H21/00
摘要： The present invention provides oligomeric compounds and uses thereof. In certain embodiments, such oligomeric compounds are useful as antisense compounds. Certain such antisense compounds are useful as RNase H antisense compounds, as RNAi compounds, and/or as modulators of splicing.
摘要翻译： 本发明提供低聚化合物及其用途。 在某些实施方案中，这些低聚化合物可用作反义化合物。 某些这样的反义化合物可用作RNase H反义化合物，作为RNAi化合物，和/或作为剪接调节剂。
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公开(公告)号：US20110065775A1
公开(公告)日：2011-03-17
申请号：US12883049
申请日：2010-09-15
申请人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewicz
发明人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewicz
IPC分类号： A61K31/7052 , C07H21/00 , A61P3/00 , A61P3/04 , A61P3/10
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：US20090326042A1
公开(公告)日：2009-12-31
申请号：US12299768
申请日：2007-05-07
申请人： Sanjay Bhanot , Richard Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewitz
发明人： Sanjay Bhanot , Richard Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric E. Swayze , Edward Wancewitz
IPC分类号： A61K31/7052 , C07H21/00 , C12N5/00 , A61P9/00
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：US20090306005A1
公开(公告)日：2009-12-10
申请号：US12299572
申请日：2007-05-07
申请人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric Swayze , Edward Wancewicz
发明人： Sanjay Bhanot , Richard S. Geary , Robert McKay , Brett P. Monia , Punit P. Seth , Andrew M. Siwkowski , Eric Swayze , Edward Wancewicz
IPC分类号： A61K31/7088 , C07H21/00 , C12N5/02
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.发明授权Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity 有权包含双环核苷并具有降低的毒性的低聚化合物公开(公告)号：US08846639B2
公开(公告)日：2014-09-30
申请号：US12936156
申请日：2009-04-03
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/3231 , C12N2310/341 , C12N2310/346 , C12N2320/53
摘要： In certain embodiments, the present invention provides oligomeric compounds having favorable toxicity profiles and therapeutic indexes. Compounds of the present invention comprise bicyclic nucleosides. Certain such bicyclic nucleosides are pyrimidines that do not include a methyl group at the 5-carbon. Oligomeric compounds comprising such nucleosides are less toxic than compounds comprising bicyclic nucleosides that do include a methyl group at the 5-carbon. In certain embodiments, the present invention provides methods of preparing and using such compounds.
摘要翻译： 在某些实施方案中，本发明提供具有良好毒性特征和治疗指标的低聚物。 本发明的化合物包含双环核苷。 某些这样的双环核苷是在5-碳不包括甲基的嘧啶。 包含这种核苷的低聚化合物比包含在5碳上包含甲基的双环核苷的化合物毒性更低。 在某些实施方案中，本发明提供了制备和使用这些化合物的方法。
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