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公开(公告)号：US11473083B2
公开(公告)日：2022-10-18
申请号：US15383452
申请日：2016-12-19
申请人： NOVARTIS AG
IPC分类号： C12N15/113 , A61K31/711 , A61K31/7115 , A61K48/00 , A61K31/7125
摘要： Provided herein are compositions and methods for decreasing tau mRNA and protein expression. These compositions and methods are useful in treating tau-related diseases and disorders.
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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：US20190390196A1
公开(公告)日：2019-12-26
申请号：US16249128
申请日：2019-01-16
申请人： Novartis AG
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 , C07C69/40 , C07C233/01 , C07C43/23 , C07D213/30 , C07D401/04 , C07D217/18 , C07D217/16 , C07D217/14 , C07D311/80 , C07D215/14 , C07D215/12
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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公开(公告)号：US20190263914A1
公开(公告)日：2019-08-29
申请号：US16210480
申请日：2018-12-05
发明人： Jennifer Brogdon , Boris Engels , David Jonathan Glass , Brian Granda , John Hastewell , Andreas Loew , Joan Mannick , Michael C. Milone , Leon Murphy , William Raj Sellers , Huijuan Song , Brian Edward Vash , Jan Weiler , Qilong Wu , Li Zhou
IPC分类号： C07K16/28 , A61K31/436 , C07K14/725 , C07K14/705 , C07K14/74 , C12N9/90 , A61K35/17 , C07K14/71 , C12N9/12
摘要： Compositions and methods relating to regulatable chimeric antigen receptors (RCARs), where the intracellular signaling or proliferation of the RCAR can be controlled to optimize the use of an RCAR-expressing cell to provide an immune response, are provided. For example, a RCAR can comprise a dimerization switch that, upon the presence of a dimerization molecule, can couple an intracellular signaling domain to an extracellular recognition element, e.g., an antigen binding domain, an inhibitory counter ligand binding domain, or costimulatory ECD domain. An RCAR can be engineered to include an appropriate antigen binding domain that is specific to a desired antigen target and used in the treatment of a disease.
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公开(公告)号：US20230026049A1
公开(公告)日：2023-01-26
申请号：US17661207
申请日：2022-04-28
发明人： Jennifer Brogdon , Boris Engels , David Jonathan Glass , Brian Granda , John Hastewell , Andreas Loew , Joan Mannick , Michael C. Milone , Leon Murphy , William Raj Sellers , Huijuan Song , Brian Edward Vash , Jan Weiler , Qilong Wu , Li Zhou
IPC分类号： C07K16/28 , C07K14/725 , C07K14/705 , C07K14/74 , C12N9/90 , C12N9/12 , C07K14/71 , A61K31/436 , A61K35/17
摘要： Compositions and methods relating to regulatable chimeric antigen receptors (RCARs), where the intracellular signaling or proliferation of the RCAR can be controlled to optimize the use of an RCAR-expressing cell to provide an immune response, are provided. For example, a RCAR can comprise a dimerization switch that, upon the presence of a dimerization molecule, can couple an intracellular signaling domain to an extracellular recognition element, e.g., an antigen binding domain, an inhibitory counter ligand binding domain, or costimulatory ECD domain. An RCAR can be engineered to include an appropriate antigen binding domain that is specific to a desired antigen target and used in the treatment of a disease.
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公开(公告)号：US10227588B2
公开(公告)日：2019-03-12
申请号：US15025779
申请日：2014-10-01
申请人： Novartis AG
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： C07H21/04 , C12N15/11 , C07D215/12 , C07D215/14 , C07D311/80 , C07C69/40 , C07D217/14 , C07D401/04 , C07D213/30 , C07C43/23 , C07C233/01 , C07D217/16 , C07D217/18
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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公开(公告)号：US20220411796A1
公开(公告)日：2022-12-29
申请号：US17813843
申请日：2022-07-20
申请人： Novartis AG
IPC分类号： C12N15/113 , A61K31/7125
摘要： Provided herein are compositions and methods for decreasing tau mRNA and protein expression. These compositions and methods are useful in treating tau-related diseases and disorders.
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公开(公告)号：US11578130B2
公开(公告)日：2023-02-14
申请号：US16210480
申请日：2018-12-05
发明人： Jennifer Brogdon , Boris Engels , David Jonathan Glass , Brian Granda , John Hastewell , Andreas Loew , Joan Mannick , Michael C. Milone , Leon Murphy , William Raj Sellers , Huijuan Song , Brian Edward Vash , Jan Weiler , Qilong Wu , Li Zhou
IPC分类号： C07K16/28 , A61K31/436 , A61K35/17 , C07K14/705 , C07K14/71 , C07K14/725 , C07K14/74 , C12N9/12 , C12N9/90
摘要： Compositions and methods relating to regulatable chimeric antigen receptors (RCARs), where the intracellular signaling or proliferation of the RCAR can be controlled to optimize the use of an RCAR-expressing cell to provide an immune response, are provided. For example, a RCAR can comprise a dimerization switch that, upon the presence of a dimerization molecule, can couple an intracellular signaling domain to an extracellular recognition element, e.g., an antigen binding domain, an inhibitory counter ligand binding domain, or costimulatory ECD domain. An RCAR can be engineered to include an appropriate antigen binding domain that is specific to a desired antigen target and used in the treatment of a disease.
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公开(公告)号：US11008570B2
公开(公告)日：2021-05-18
申请号：US16249128
申请日：2019-01-16
申请人： Novartis AG
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： C12N15/113 , C12N15/11 , C07D215/12 , C07D215/14 , C07D311/80 , C07C69/40 , C07D217/14 , C07D401/04 , C07D213/30 , C07C43/23 , C07C233/01 , C07D217/16 , C07D217/18
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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公开(公告)号：US10287354B2
公开(公告)日：2019-05-14
申请号：US15105074
申请日：2014-12-19
发明人： Jennifer Brogdon , Boris Engels , David Jonathan Glass , Brian Granda , John Hastewell , Andreas Loew , Joan Mannick , Michael Milone , Leon Murphy , William Raj Sellers , Huijuan Song , Brian Edward Vash , Jan Weiler , Qilong Wu , Li Zhou
IPC分类号： C07K16/28 , A61K31/436 , A61K35/17 , C07K14/705 , C07K14/71 , C07K14/725 , C12N9/90 , C07K14/74
摘要： Compositions and methods relating to regulatable chimeric antigen receptors (RCARs), where the intracellular signaling or proliferation of the RCAR can be controlled to optimize the use of an RCAR-expressing cell to provide an immune response, are provided. For example, a RCAR can comprise a dimerization switch that, upon the presence of a dimerization molecule, can couple an intracellular signaling domain to an extracellular recognition element, e.g., an antigen binding domain, an inhibitory counter ligand binding domain, or costimulatory ECD domain. An RCAR can be engineered to include an appropriate antigen binding domain that is specific to a desired antigen target and used in the treatment of a disease.
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