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公开(公告)号:US09988627B2
公开(公告)日:2018-06-05
申请号:US15025826
申请日:2014-10-06
申请人: Jeremy Lee Baryza , Marcel Blommers , William Chutkow , Cesar Fernandez , Erin Geno , Alvar Gossert , Paulette Greenidge , Dieter Huesken , Juerg Hunziker , Francois Jean-Charles Natt , Anup Patnaik , Andrew Patterson , Jean-Michel Rene Rondeau , Jan Weiler , Meicheng Zhu
发明人: Jeremy Lee Baryza , Marcel Blommers , William Chutkow , Cesar Fernandez , Erin Geno , Alvar Gossert , Paulette Greenidge , Dieter Huesken , Juerg Hunziker , Francois Jean-Charles Natt , Anup Patnaik , Andrew Patterson , Jean-Michel Rene Rondeau , Jan Weiler , Meicheng Zhu
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.
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公开(公告)号:US20160244756A1
公开(公告)日:2016-08-25
申请号:US15025826
申请日:2014-10-06
申请人: Jeremy Lee BARYZA , Marcel BLOMMERS , William CHUTKOW , Cesar FERNANDEZ , Erin GENO , Alvar GOSSERT , Paulette GREENIDGE , Dieter HUESKEN , Juerg HUNZIKER , Francois Jean-Charles NATT , Anup PATNAIK , Andrew PATTERSON , Jean-Michel Rene RONDEAU , Jan WEILER , Meicheng ZHU
发明人: Jeremy Lee Baryza , Marcel Blommers , William Chutkow , Cesar Femandez , Erin Geno , Alvar Gossert , Paulette Greenridge , Dieter Huesken , Juerg Hunziker , Francois Jean-Charles Natt , Anup Patnaik , Andrew Patterson , Jean-Michel Rene Rondeau , Jan Weiler , Meicheng Zhu
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.
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3.
公开(公告)号:US20150259675A1
公开(公告)日:2015-09-17
申请号:US14177640
申请日:2014-02-11
IPC分类号: C12N15/113
CPC分类号: C12N15/113 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/332 , C12N2310/346 , C12N2310/3529 , C12N2310/50 , C12N2310/3521 , C12N2310/3533 , C12N2310/3525
摘要: Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.
摘要翻译: 用于口服给药的短干扰核糖核酸(siRNA),所述siRNA包含在至少15个核苷酸上彼此互补的两个单独的RNA链,其中每条链是49个核苷酸或更少,并且其中至少一个链含至少 一种化学改性。
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公开(公告)号:US20160304863A1
公开(公告)日:2016-10-20
申请号:US15025779
申请日:2014-10-01
申请人: Jeremy Lee BARYZA , Marcel BLOMMERS , Cesar FERNANDEZ , Erin GENO , Alvar GOSSERT , Paulette GREENIDGE , Dieter HUESKEN , Juerg HUNZIKER , Francois Jean-Charles NATT , Anup PATNAIK , Andrew PATTERSON , Jean-Michel Rene RONDEAU , Jan WEILER , Meicheng ZHU
发明人: Jeremy Lee Baryza , Marcel Blommers , Cesar Fernandez , Erin Geno , Alvar Gossert , Paulette Greenidge , Dieter Huesken , Juerg Hunziker , Francois Jean-Charles Natt , Anup Patnaik , Andrew Patterson , Jean-Michel Rene Rondeau , Jan Weiler , Meicheng Zhu
IPC分类号: C12N15/11 , C07C233/01 , C07D217/18 , C07D215/14 , C07D217/16
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。
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公开(公告)号:US08945904B2
公开(公告)日:2015-02-03
申请号:US13699301
申请日:2011-05-20
申请人: Heidi Trusheim , Peter Mason , Michael Franti , Bjoern Keiner , Melissa Sackal , Juerg Hunziker , Francois Natt , David Morrissey
发明人: Heidi Trusheim , Peter Mason , Michael Franti , Bjoern Keiner , Melissa Sackal , Juerg Hunziker , Francois Natt , David Morrissey
IPC分类号: C12N7/02 , A61K39/145 , C12N7/00 , C12N15/113 , A61K39/00
CPC分类号: C12N7/00 , A61K39/12 , A61K39/145 , A61K2039/525 , C12N15/1131 , C12N2310/14 , C12N2320/00 , C12N2760/16134 , C12N2760/16151 , C12N2760/16152
摘要: Methods for producing reassortant viruses are provided wherein the transcription and/or translation of the hemagglutinin and/or neuraminidase genes are suppressed.
摘要翻译: 提供了产生重配病毒的方法,其中抑制了血凝素和/或神经氨酸酶基因的转录和/或翻译。
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公开(公告)号:US20130216573A1
公开(公告)日:2013-08-22
申请号:US13699301
申请日:2011-05-20
申请人: Heidi Trusheim , Peter Mason , Michael Franti , Bjoern Keiner , Melissa Sackal , Juerg Hunziker , Francois Natt , David Morrissey
发明人: Heidi Trusheim , Peter Mason , Michael Franti , Bjoern Keiner , Melissa Sackal , Juerg Hunziker , Francois Natt , David Morrissey
IPC分类号: A61K39/145
CPC分类号: C12N7/00 , A61K39/12 , A61K39/145 , A61K2039/525 , C12N15/1131 , C12N2310/14 , C12N2320/00 , C12N2760/16134 , C12N2760/16151 , C12N2760/16152
摘要: Methods for producing reassortant viruses are provided wherein the transcription and/or translation of the hemagglutinin and/or neuraminidase genes are suppressed.
摘要翻译: 提供了产生重配病毒的方法,其中抑制了血凝素和/或神经氨酸酶基因的转录和/或翻译。
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公开(公告)号:US08404831B2
公开(公告)日:2013-03-26
申请号:US13218835
申请日:2011-08-26
IPC分类号: C07H21/04
CPC分类号: C12N15/113 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/332 , C12N2310/346 , C12N2310/3529 , C12N2310/50 , C12N2310/3521 , C12N2310/3533 , C12N2310/3525
摘要: Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.
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公开(公告)号:US20120029053A1
公开(公告)日:2012-02-02
申请号:US13218941
申请日:2011-08-26
IPC分类号: A61K31/7088 , A61P35/00 , A61P29/00 , A61P9/10 , A61P19/02 , A61P11/06 , C07H21/02 , A61P35/04
CPC分类号: C12N15/113 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/332 , C12N2310/346 , C12N2310/3529 , C12N2310/50 , C12N2310/3521 , C12N2310/3533 , C12N2310/3525
摘要: Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.
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公开(公告)号:US10519446B2
公开(公告)日:2019-12-31
申请号:US15025852
申请日:2014-09-30
申请人: Novartis AG , Jeremy Lee Baryza , Marcel Blommers , Cesar Fernandez , Erin Geno , Alvar Gossert , Paulette Greenidge , Dieter Huesken , Juerg Hunziker , Francois Jean-Charles Natt , Anup Patnaik , Andrew Patterson , Jean-Michel Rene Rondeau , Jan Weiler , Meicheng Zhu , Meghan Holdorf
发明人: Jeremy Lee Baryza , Marcel Blommers , Cesar Fernandez , Erin Geno , Alvar Gossert , Paulette Greenidge , Dieter Huesken , Juerg Hunziker , Francois Jean-Charles Natt , Anup Patnaik , Andrew Patterson , Jean-Michel Rene Rondeau , Jan Weiler , Meicheng Zhu , Meghan Holdorf
IPC分类号: C12N15/113
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.
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公开(公告)号:US08404832B2
公开(公告)日:2013-03-26
申请号:US13218880
申请日:2011-08-26
IPC分类号: C07H21/04
CPC分类号: C12N15/113 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/332 , C12N2310/346 , C12N2310/3529 , C12N2310/50 , C12N2310/3521 , C12N2310/3533 , C12N2310/3525
摘要: Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.
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