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1. 发明申请

WO2015051366A3 NOVEL FORMATS FOR ORGANIC COMPOUNDS FOR USE IN RNA INTERFERENCE 审中-公开
标题翻译：用于RNA干扰的有机化合物的新格式
公开(公告)号：WO2015051366A3
公开(公告)日：2015-07-02
申请号：PCT/US2014059301
申请日：2014-10-06
申请人： NOVARTIS AG , BARYZA JEREMY LEE , BLOMMERS MARCEL , CHUTKOW WILLIAM , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG
发明人： BARYZA JEREMY LEE , BLOMMERS MARCEL , CHUTKOW WILLIAM , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/341 , C12N2310/351 , C12N2310/3515 , C12N2310/3521 , C12N2310/3525 , C12N2310/3533
摘要： The disclosure relates to compositions comprising a RNAi agent having a novel format including a spacer subunit. The disclosure relates to compositions comprising a RNAi agent having a novel format: an 18-mer format with at least one internal spacer. These RNAi agents comprise a first and a second 18-mer strand, wherein the first strand is 18 ribonucleotides or 18 total ribonucleotides and spacer subunit(s), and the second strand is 18 total ribonucleotides and spacer subunit(s), wherein: each spacer subunit consists of: (a) a phosphate or modified internucleoside linker and (b) a spacer; a spacer subunit can be at any position in the strand; the two strands form a duplex with at least one blunt end; and the 3 end of one or both strands terminates in a phosphate or modified internucleoside linker and further comprises, in 5 to 3 order: a second spacer; a second phosphate or modified internucleoside linker, and a 3 end cap. In various embodiments, the RNAi agents comprise a first and a second strand, wherein each strand is a 30-mer or shorter, the first strand comprises ribonucleotides, and the second strand comprises ribonucleotides and one or more spacer subunit(s), wherein: each spacer subunit consists of: (a) a phosphate or modified internucleoside linker and (b) a spacer; a spacer subunit can be at any position in the strand. In some embodiments, the 3 end of both strands further comprise, in 5 to 3 order: a second spacer; a second phosphate or modified internucleoside linker; and a 3 end cap. The two strands can have the same or different spacers, phosphates or modified internucleoside linkers, and/or 3 end caps. The strands can be ribonucleotides, or, optionally, one or more nucleotide can be modified or substituted. Optionally, at least one nucleotide comprises a modified internucleoside linker. Optionally, the first two base-pairing nucleotides on the 3 end of the one or both strand are 2-MOE. Optionally, the RNAi agent can be modified on one or both 5 end. Optionally, the first or second strand is the sense strand, and the sense strand can comprise a 5 end cap which reduces the amount of the RNA interference mediated by this strand. Optionally, the RNAi agent is attached to a ligand. This format can be used to devise RNAi agents to a variety of different targets and sequences. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference.
摘要翻译：本公开涉及包含具有包括间隔子亚单位的新格式的RNAi剂的组合物。本公开涉及包含具有新格式的RNAi剂的组合物：具有至少一个内部间隔物的18聚体形式。这些RNAi剂包含第一和第二18聚体链，其中第一链是18个核糖核苷酸或18个总核糖核苷酸和间隔子亚单位，第二个链是18个总核糖核苷酸和间隔子亚基，其中：间隔子亚基由以下组成：（a）磷酸酯或修饰的核苷间连接体和（b）间隔子; 间隔子亚基可以在链中的任何位置; 两条链与至少一个钝端形成双链体; 并且一条或两条链的3端终止于磷酸酯或修饰的核苷间连接体，并且还包含5至3个顺序：第二间隔物; 第二磷酸酯或修饰的核苷间连接体，和3端帽。在各种实施方案中，RNAi试剂包含第一和第二链，其中每条链均为30聚体或更短，第一链包含核糖核苷酸，第二链包含核糖核苷酸和一个或多个间隔子亚单位，其中：每个间隔子亚基由以下组成：（a）磷酸酯或修饰的核苷间连接体和（b）间隔子; 间隔子亚基可以在链中的任何位置。在一些实施方案中，两条链的3端还包含5至3个顺序：第二间隔物; 第二磷酸酯或修饰的核苷间连接体; 和3个端盖。两条链可以具有相同或不同的间隔物，磷酸酯或修饰的核苷间连接体和/或3个末端帽。链可以是核糖核苷酸，或者任选地，一个或多个核苷酸可以被修饰或取代。任选地，至少一个核苷酸包含修饰的核苷间连接体。任选地，一条或两条链的3端上的前两个碱基配对核苷酸是2-MOE。任选地，RNAi剂可在一端或两端进行修饰。任选地，第一链或第二链是有义链，有义链可以包含5端帽，其减少由该链介导的RNA干扰的量。任选地，RNAi试剂连接到配体上。这种格式可用于将RNAi试剂设计成各种不同的靶标和序列。本公开还涉及制备这种组合物的方法，以及这种组合物的方法和用途，例如介导RNA干扰。

2. 发明申请

WO2015051045A2 3'END CAPS FOR RNAi AGENTS FOR USE IN RNA INTERFERENCE 审中-公开
标题翻译：用于RNA干扰的RNAi试剂的3'端
公开(公告)号：WO2015051045A2
公开(公告)日：2015-04-09
申请号：PCT/US2014058705
申请日：2014-10-01
申请人： NOVARTIS AG , BARYZA JEREMY LEE , BLOMMERS MARCEL , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG
发明人： BARYZA JEREMY LEE , BLOMMERS MARCEL , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG
IPC分类号： C07C43/178
CPC分类号： C12N15/111 , C07C43/23 , C07C69/40 , C07C233/01 , C07D213/30 , C07D215/12 , C07D215/14 , C07D217/14 , C07D217/16 , C07D217/18 , C07D311/80 , C07D401/04 , C12N2310/14 , C12N2310/344 , C12N2310/351 , C12N2320/51
摘要： The disclosure relates to novel compounds and compositions comprising a RNAi agent comprising a novel compound as a 3' end cap. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference.
摘要翻译：本公开涉及包含含有作为3'端帽的新化合物的RNAi剂的新化合物和组合物。本公开还涉及制备这种组合物的方法，以及这种组合物的方法和用途，例如介导RNA干扰。

3. 发明申请

WO2015050871A2 ORGANIC COMPOUNDS TO TREAT HEPATITIS B VIRUS 审中-公开
标题翻译：有机化合物治疗乙型肝炎病毒
公开(公告)号：WO2015050871A2
公开(公告)日：2015-04-09
申请号：PCT/US2014058314
申请日：2014-09-30
申请人： NOVARTIS AG , BARYZA JEREMY LEE , BLOMMERS MARCEL , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG , HOLDORF MEGHAN
发明人： BARYZA JEREMY LEE , BLOMMERS MARCEL , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG , HOLDORF MEGHAN
IPC分类号： A61K48/00 , C07F9/6512 , C07H21/02
CPC分类号： C12N15/1131 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/351 , C12N2310/3515 , C12N2320/30 , C12N2310/3521 , C12N2310/3525 , C12N2310/3533
摘要： The disclosure relates to compositions comprising a HBV RNAi agent. In some embodiments, the HBV RNAi agent comprises a sense and an anti-sense strand, each strand being an 18-mer and the strands together forming a blunt-ended duplex, wherein the 3' end of at least one strand terminates in a phosphate or modified internucleoside linker and further comprises, in 5' to 3' order: a spacer; a second phosphate or modified internucleoside linker; and a 3' end cap. In some embodiments, the 3' end of both the sense and anti-sense strand further comprise, in 5' to 3' order: a spacer; a second phosphate or modified internucleoside linker; and a 3' end cap. The two strands can have the same or different spacers, phosphates or modified internucleoside linkers, and/or 3' end caps. The strands can be ribonucleotides, or, optionally, one or more nucleotide can be modified or substituted. Optionally, at least one nucleotide comprises a modified internucleoside linker. Optionally, the RNAi agent can be modified on one or both 5' end. Optionally, the sense strand can comprise a 5' end cap which reduces the amount of the RNA interference mediated by this strand. Optionally, the RNAi agent is attached to a ligand. This format can be used to devise RNAi agents to a variety of different targets and sequences. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference. The disclosure also pertains to methods of treating, ameliorating and preventing HBV in a patient involving the step of administering to the patient a therapeutic amount of a HBV RNAi agent.
摘要翻译：本公开涉及包含HBV RNAi剂的组合物。在一些实施方案中，HBV RNAi剂包含有义链和反义链，每条链是18聚体，并且所述链一起形成平端双链体，其中至少一条链的3'末端终止于磷酸酯或修饰的核苷间连接体，并且还包含以5'至3'的顺序：间隔物; 第二磷酸酯或修饰的核苷间连接体; 和3'端盖。在一些实施方案中，有义和反义链两者的3'末端还包含5'至3'的顺序：间隔基; 第二磷酸酯或修饰的核苷间连接体; 和3'端盖。两条链可以具有相同或不同的间隔物，磷酸酯或修饰的核苷间接头，和/或3'端帽。链可以是核糖核苷酸，或者任选地，一个或多个核苷酸可以被修饰或取代。任选地，至少一个核苷酸包含修饰的核苷间连接体。任选地，RNAi剂可以在一个或两个5'端修饰。任选地，有义链可以包含5'端帽，其减少由该链介导的RNA干扰的量。任选地，RNAi试剂连接到配体上。这种格式可用于将RNAi试剂设计成各种不同的靶标和序列。本公开还涉及制备这种组合物的方法，以及这种组合物的方法和用途，例如介导RNA干扰。本公开还涉及在患者中治疗，改善和预防HBV的方法，其涉及向患者施用治疗量的HBV RNAi剂的步骤。

4. 发明申请

WO2015051135A3 ORGANIC COMPOSITIONS TO TREAT HEPCIDIN-RELATED DISEASES 审中-公开
标题翻译：治疗肝素相关疾病的有机组合物
公开(公告)号：WO2015051135A3
公开(公告)日：2015-09-03
申请号：PCT/US2014058851
申请日：2014-10-02
申请人： NOVARTIS AG , BARYZA JEREMY LEE , BLOMMERS MARCEL , BORLAND TODD , FERNANDEZ CESAR , GEBUHR TOM , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , SOLOMON JONATHAN , WEILER JAN , ZHU MEICHENG
发明人： BARYZA JEREMY LEE , BLOMMERS MARCEL , BORLAND TODD , FERNANDEZ CESAR , GEBUHR TOM , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , SOLOMON JONATHAN , WEILER JAN , ZHU MEICHENG
IPC分类号： C12N15/113 , A61K31/713 , A61P7/06
CPC分类号： C12N15/113 , C12N15/1136 , C12N2310/14 , C12N2310/321 , C12N2310/344 , C12N2310/351 , C12N2320/51
摘要： The present disclosure relates to methods of treating Hepcidin-related diseases such as anemia, iron-deficient erythropoiesis, hypoferremia, impaired dietary iron uptake, iron sequestration, anemia of inflammation (AI) (sometimes designated anemia of chronic disease or ACD), atherosclerosis, diabetes, and multiple neurodegenerative disorders such as Alzheimers disease, Parkinsons disease and Friedrichs ataxia, heart failure, chronic kidney disease, cardiorenal-anemia syndrome, infection, blood loss, hemolysis, vitamin B12 or folate deficiency, hyperparathyroidism, hemoglobinopathies and malignancies, cancer, AIDS, surgery, runted growth, and hair loss, using a therapeutically effective amount of a RNAi agent to Hepcidin.
摘要翻译：本发明涉及治疗铁调素相关疾病例如贫血，铁缺乏性红细胞生成，低铁血症，饮食铁摄取受损，铁螯合，炎症贫血（AI）（有时称为慢性疾病或ACD贫血），动脉粥样硬化，糖尿病和多种神经退行性疾病如阿尔茨海默病，帕金森病和弗里德里希共济失调，心力衰竭，慢性肾病，心肾综合征，感染，失血，溶血，维生素B12或叶酸缺乏症，甲状旁腺功能亢进症，血红蛋白病和恶性肿瘤，艾滋病，手术，运行的生长和脱发，使用治疗有效量的RNAi剂对Hepcidin。

5. 发明申请

WO2015051044A3 NOVEL FORMATS FOR ORGANIC COMPOUNDS FOR USE IN RNA INTERFERENCE 审中-公开
标题翻译：用于RNA干扰的有机化合物的新形式
公开(公告)号：WO2015051044A3
公开(公告)日：2015-07-02
申请号：PCT/US2014058703
申请日：2014-10-01
申请人： NOVARTIS AG , BARYZA JEREMY LEE , BLOMMERS MARCEL , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCIOS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG
发明人： BARYZA JEREMY LEE , BLOMMERS MARCEL , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCIOS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/332 , C12N2310/3515 , C12N2310/3521 , C12N2310/3525 , C12N2310/3533
摘要： The disclosure relates to compositions comprising a RNAi agent having a novel format. These RNAi agents comprise a sense and an anti-sense strand, each strand being an 18-mer and the strands together forming a blunt-ended duplex, wherein the 3' end of at least one strand terminates in a phosphate or modified internucleoside linker and further comprises, in 5' to 3' order: a spacer; a second phosphate or modified internucleoside linker; and a 3' end cap. In some embodiments, the 3' end of both the sense and anti-sense strand further comprise, in 5' to 3' order: a spacer; a second phosphate or modified internucleoside linker; and a 3' end cap. The two strands can have the same or different spacers, phosphates or modified internucleoside linkers, and/or 3' end caps. The strands can be ribonucleotides, or, optionally, one or more nucleotide can be modified or substituted. Optionally, at least one nucleotide comprises a modified internucleoside linker. Optionally, the first two base-pairing nucleotides on the 3' end of the sense and/or anti-sense strand are 2'-MOE. Optionally, the RNAi agent can be modified on one or both 5' end. Optionally, the sense strand can comprise a 5' end cap which reduces the amount of the RNA interference mediated by this strand. Optionally, the RNAi agent is attached to a ligand. This format can be used to devise RNAi agents to a variety of different targets and sequences. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference.
摘要翻译：本公开涉及包含具有新颖形式的RNAi剂的组合物。这些RNAi剂包含有义链和反义链，每条链都是18聚体，并且链一起形成平头双链体，其中至少一条链的3'末端终止于磷酸盐或修饰的核苷间连接体并且进一步包括按照5'至3'的顺序：间隔物; 第二磷酸盐或修饰的核苷间连接体; 和3'端盖。在一些实施方案中，有义链和反义链两者的3'末端还以5'至3'的顺序包含：间隔子; 第二磷酸盐或修饰的核苷间连接体; 和3'端盖。两条链可以具有相同或不同的间隔区，磷酸盐或修饰的核苷间接头，和/或3'端帽。链可以是核糖核苷酸，或者任选地，一个或多个核苷酸可以被修饰或取代。任选地，至少一个核苷酸包含修饰的核苷间连接体。任选地，有义链和/或反义链的3'末端上的前两个碱基配对核苷酸是2'-MOE。任选地，RNAi剂可以在一个或两个5'末端被修饰。任选地，有义链可以包含5'端帽，其减少由该链介导的RNA干扰的量。任选地，RNAi剂附着于配体。该格式可用于将RNAi试剂设计为多种不同的靶标和序列。本公开还涉及制备这种组合物的方法，以及这些组合物的方法和用途，例如介导RNA干扰。

6. 发明专利

CA2925129A1 3'END CAPS FOR RNAI AGENTS FOR USE IN RNA INTERFERENCE 未知
公开(公告)号：CA2925129A1
公开(公告)日：2015-04-09
申请号：CA2925129
申请日：2014-10-01
申请人： NOVARTIS AG
发明人： BARYZA JEREMY LEE , BLOMMERS MARCEL , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG
IPC分类号： C07C43/178 , C07C69/40 , C07D213/30 , C07D215/12 , C07D215/14 , C07D217/14 , C07D311/80 , C07D401/04 , C12N15/11
摘要： The disclosure relates to novel compounds and compositions comprising a RNAi agent comprising a novel compound as a 3' end cap. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference.

7. 发明专利

AU2014329560B2 3'end caps for RNAi agents for use in RNA interference 未知
公开(公告)号：AU2014329560B2
公开(公告)日：2017-03-02
申请号：AU2014329560
申请日：2014-10-01
申请人： NOVARTIS AG
发明人： BARYZA JEREMY LEE , BLOMMERS MARCEL , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG
IPC分类号： C07C43/178 , C07C69/40 , C07D213/30 , C07D215/12 , C07D215/14 , C07D217/14 , C07D311/80 , C07D401/04 , C12N15/11
摘要： The disclosure relates to novel compounds and compositions comprising a RNAi agent comprising a novel compound as a 3' end cap. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference.

8. 发明专利

AU2014329560A1 3'end caps for RNAi agents for use in RNA interference 未知
公开(公告)号：AU2014329560A1
公开(公告)日：2016-04-07
申请号：AU2014329560
申请日：2014-10-01
申请人： NOVARTIS AG
发明人： BARYZA JEREMY LEE , BLOMMERS MARCEL , FERNANDEZ CESAR , GENO ERIN , GOSSERT ALVAR , GREENIDGE PAULETTE , HUESKEN DIETER , HUNZIKER JUERG , NATT FRANCOIS JEAN-CHARLES , PATNAIK ANUP , PATTERSON ANDREW , RONDEAU JEAN-MICHEL RENE , WEILER JAN , ZHU MEICHENG
IPC分类号： C07C43/178 , C07C69/40 , C07D213/30 , C07D215/12 , C07D215/14 , C07D217/14 , C07D311/80 , C07D401/04 , C12N15/11
摘要： The disclosure relates to novel compounds and compositions comprising a RNAi agent comprising a novel compound as a 3' end cap. The disclosure also relates to processes for making such compositions, and methods and uses of such compositions, e.g., to mediate RNA interference.

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