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21.发明申请公开(公告)号：US20120095076A1
公开(公告)日：2012-04-19
申请号：US13252917
申请日：2011-10-04
申请人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
发明人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
摘要翻译： 本发明涉及用于抑制亨廷顿蛋白基因（HD基因）表达的双链核糖核酸（dsRNA），其包含长度小于25个核苷酸的核苷酸序列的反义链，并且至少与至少 HD基因的一部分。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由HD基因的表达引起的疾病或其突变形式的方法; 以及抑制细胞中亨廷顿蛋白基因表达的方法。
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公开(公告)号：US20110245320A1
公开(公告)日：2011-10-06
申请号：US13038672
申请日：2011-03-02
申请人： Hans-Peter VORNLOCHER , Ingo ROEHL , Philipp HADWIGER , Tracy Stage ZIMMERMANN , Muthiah MANOHARAN , Kallanthottathil G. RAJEEV , Akin AKINC
发明人： Hans-Peter VORNLOCHER , Ingo ROEHL , Philipp HADWIGER , Tracy Stage ZIMMERMANN , Muthiah MANOHARAN , Kallanthottathil G. RAJEEV , Akin AKINC
IPC分类号： A61K31/713 , C07H21/02 , A61P43/00
CPC分类号： C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/53 , C12N2320/51 , C12N2310/3521 , C12N2310/3525 , C12N2310/3527
摘要： This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.
摘要翻译： 本发明涉及在细胞和生物流体中具有改善的稳定性的修饰的双链寡核糖核酸（dsRNA），以及制备和鉴定具有改善的稳定性的dsRNA的方法，以及使用dsRNA抑制靶基因的表达或功能。
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公开(公告)号：US20110201006A1
公开(公告)日：2011-08-18
申请号：US13124411
申请日：2009-10-06
申请人： Ingo Roehl , Markus Schuster , Stephan Seiffert
发明人： Ingo Roehl , Markus Schuster , Stephan Seiffert
摘要： The invention relates to a method for the detection of oligonucleotides using anion exchange high performance liquid chromatography. Fluorescently labelled peptide nucleic acid oligomers, complementary to the oligonucleotide are hybridized to the oligonucleotides. Anion exchange high performance liquid chromatography is then performed and the hybridized moieties detected and quantitated. The invention also relates to a method for the simultaneous detection of both strands of an oligonucleotide in parallel from one sample, and a kit for use in qualitative and quantitative detection of one or two strands of an oligonucleotide
摘要翻译： 本发明涉及使用阴离子交换高效液相色谱检测寡核苷酸的方法。 与寡核苷酸互补的荧光标记的肽核酸低聚物与寡核苷酸杂交。 然后进行阴离子交换高效液相色谱，检测和定量杂交部分。 本发明还涉及从一个样品同时检测寡核苷酸两条链的方法，以及用于定性和定量检测寡核苷酸的一条或两条链的试剂盒
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24.发明授权公开(公告)号：US07749978B2
公开(公告)日：2010-07-06
申请号：US11944961
申请日：2007-11-26
申请人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
发明人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
摘要翻译： 本发明涉及用于抑制亨廷顿蛋白基因（HD基因）表达的双链核糖核酸（dsRNA），其包含长度小于25个核苷酸的核苷酸序列的反义链，并且至少与至少 HD基因的一部分。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由HD基因的表达引起的疾病或其突变形式的方法; 以及抑制细胞中亨廷顿蛋白基因表达的方法。
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公开(公告)号：US20060094678A1
公开(公告)日：2006-05-04
申请号：US11139089
申请日：2005-05-27
申请人： Hans-Peter Vornlocher , Ingo Roehl , Philipp Hadwiger , Tracy Zimmermann , Muthiah Manoharan , Kallanthottathil Rajeev , Akin Akinc
发明人： Hans-Peter Vornlocher , Ingo Roehl , Philipp Hadwiger , Tracy Zimmermann , Muthiah Manoharan , Kallanthottathil Rajeev , Akin Akinc
CPC分类号： C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/53 , C12N2320/51 , C12N2310/3521 , C12N2310/3525 , C12N2310/3527
摘要： This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.
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26.发明授权Galactose cluster-pharmacokinetic modulator targeting moiety for siRNA 有权用于siRNA的半乳糖簇 - 药代动力学调节剂靶向部分公开(公告)号：US09249179B2
公开(公告)日：2016-02-02
申请号：US13327271
申请日：2011-12-15
申请人： Philipp Hadwiger , Torsten Hoffmann , Eric A. Kitas , Peter Mohr , Ingo Roehl , Linda Valis , David B. Rozema , David L. Lewis , Darren H. Wakefield
发明人： Philipp Hadwiger , Torsten Hoffmann , Eric A. Kitas , Peter Mohr , Ingo Roehl , Linda Valis , David B. Rozema , David L. Lewis , Darren H. Wakefield
IPC分类号： A61K31/7088 , C07H21/02
CPC分类号： C07K5/1021 , C07H21/02
摘要： The present invention is directed compositions for targeted delivery of RNA interference (RNAi) polynucleotides to cell in vivo. The pharmacokinetic modulator improve in vivo targeting compared to the targeting ligand alone. Targeting ligand-pharmacokinetic modulator targeting moiety targeted RNAi polynucleotides can be administered in vivo alone or together with co-targeted delivery polymers.
摘要翻译： 本发明涉及用于靶向将RNA干扰（RNAi）多核苷酸递送至细胞体内的组合物。 与单独的靶向配体相比，药代动力学调节剂改善体内靶向。 靶向配体 - 药代动力学调节剂靶向部分靶向的RNAi多核苷酸可以单独施用或与共靶向递送聚合物一起施用。
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27.发明申请公开(公告)号：US20130190484A1
公开(公告)日：2013-07-25
申请号：US13833814
申请日：2013-03-15
申请人： David Rozema , David Lewis , Darren Wakefield , Eric Kitas , Philipp Hadwiger , Jon Wolff , Ingo Roehl , Peter Mohr , Torsten Hoffmann , Kerstin Jahn-Hofmann , Hans Martin Mueller , Guenther Ott , Andrei Blokhin , Jeffrey Carlson , Jonathan Benson
发明人： David Rozema , David Lewis , Darren Wakefield , Eric Kitas , Philipp Hadwiger , Jon Wolff , Ingo Roehl , Peter Mohr , Torsten Hoffmann , Kerstin Jahn-Hofmann , Hans Martin Mueller , Guenther Ott , Andrei Blokhin , Jeffrey Carlson , Jonathan Benson
IPC分类号： C12N15/113 , C07C271/20 , C07C69/96
CPC分类号： A61K38/00 , A61K31/7088 , A61K47/60 , A61K47/65 , A61K48/00 , C07C69/96 , C07C271/20 , C07H15/04 , C07H15/20 , C07H15/26 , C07K7/02 , C07K2317/77 , C12N15/1138 , C12N15/87 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/351 , C12N2320/30
摘要： The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.
摘要翻译： 本发明涉及用于将RNA干扰（RNAi）多核苷酸递送至细胞体内的组合物。 组合物包含用酶切割二肽 - 氨基苄基 - 碳酸酯掩蔽剂可逆地修饰的两亲膜活性多胺。 改性掩盖聚合物的膜活性，而可逆性提供生理反应性。 可逆修饰的多胺（动态多偶联物或DPC）进一步共价连接到RNAi多核苷酸或与靶向RNAi多核苷酸靶向分子缀合物共同施用。
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公开(公告)号：US20120165393A1
公开(公告)日：2012-06-28
申请号：US13326433
申请日：2011-12-15
申请人： David B. Rozema , Darren H. Wakefield , David L. Lewis , Jon A. Wolff , Andrei V. Blokhin , Jonathan D. Benson , Jeffrey C. Carlson , Philipp Hadwiger , Eric A. Kitas , Torsten Hoffman , Kerstin Jahn-Hofmann , Peter Mohr , Hans Martin Mueller , Guenther Ott , Ingo Roehl
发明人： David B. Rozema , Darren H. Wakefield , David L. Lewis , Jon A. Wolff , Andrei V. Blokhin , Jonathan D. Benson , Jeffrey C. Carlson , Philipp Hadwiger , Eric A. Kitas , Torsten Hoffman , Kerstin Jahn-Hofmann , Peter Mohr , Hans Martin Mueller , Guenther Ott , Ingo Roehl
CPC分类号： A61K47/48261 , A61K47/549 , A61K47/64 , A61K47/6415 , C12N15/111 , C12N2310/14 , C12N2310/351 , C12N2310/3515 , C12N2320/32
摘要： The present invention is directed compositions for targeted delivery of RNA interference (RNAi) polynucleotides to hepatocytes in vivo. Targeted RNAi polynucleotides are administered together with co-targeted melittin delivery peptides. Delivery peptides provide membrane penetration function for movement of the RNAi polynucleotides from outside the cell to inside the cell. Reversible modification provides physiological responsiveness to the delivery peptides.
摘要翻译： 本发明涉及用于将体内RNA干扰（RNAi）多核苷酸靶向递送至肝细胞的组合物。 靶向的RNAi多核苷酸与共靶向的蜂毒肽递送肽一起施用。 递送肽提供膜穿透功能，用于将RNAi多核苷酸从细胞外部移动到细胞内。 可逆修饰提供对递送肽的生理反应性。
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公开(公告)号：US07928217B2
公开(公告)日：2011-04-19
申请号：US11139089
申请日：2005-05-27
申请人： Hans-Peter Vornlocher , Ingo Roehl , Philipp Hadwiger , Tracy Stage Zimmermann , Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc
发明人： Hans-Peter Vornlocher , Ingo Roehl , Philipp Hadwiger , Tracy Stage Zimmermann , Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc
CPC分类号： C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/53 , C12N2320/51 , C12N2310/3521 , C12N2310/3525 , C12N2310/3527
摘要： This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.
摘要翻译： 本发明涉及在细胞和生物流体中具有改善的稳定性的修饰的双链寡核糖核酸（dsRNA），以及制备和鉴定具有改善的稳定性的dsRNA的方法，以及使用dsRNA抑制靶基因的表达或功能。
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公开(公告)号：US07902168B2
公开(公告)日：2011-03-08
申请号：US12487605
申请日：2009-06-18
申请人： Dinah Wen-Yee Sah , Maria Frank-Kamenetsky , Anke Geick , Philipp Hadwiger , Ingo Roehl , Pamela Tan , Hans-Peter Vornlocher
发明人： Dinah Wen-Yee Sah , Maria Frank-Kamenetsky , Anke Geick , Philipp Hadwiger , Ingo Roehl , Pamela Tan , Hans-Peter Vornlocher
CPC分类号： C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Nav1.8 gene (Nav1.8 gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Nav1.8 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the Nav1.8 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Nav1.8 gene gene in a cell.
摘要翻译： 本发明涉及用于抑制Nav1.8基因（Nav1.8基因）表达的双链核糖核酸（dsRNA），其包含具有长度小于25个核苷酸的核苷酸序列的反义链，其基本上 与Nav1.8基因的至少一部分互补。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 用于治疗由使用该药物组合物的Nav1.8基因表达引起的疾病的方法; 以及抑制细胞中Nav1.8基因表达的方法。
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