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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: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:US10023862B2
公开(公告)日:2018-07-17
申请号:US14371131
申请日:2013-01-08
申请人: Dieter Huesken , Brian Bettencourt , David Bumcrot , Satyanarayana Kuchimanchi , Stuart Milstein , Michael Schlabach, Jr. , Frank P. Stegmeier , Markus Warmuth , Jan Weiler , NOVARTIS AG
发明人: Brian Richard Bettencourt , David Anton Bumcrot , Dieter Huesken , Satyanarayana Kuchimanchi , Stuart Milstein , Michael Ray Schlabach, Jr. , Frank Peter Stegmeier , Markus Warmuth , Jan Weiler
IPC分类号: A61P35/00 , A61P17/02 , C12N15/113 , A61K31/713
摘要: The present disclosure relates to RNAi agents useful in methods of treating Beta-Catenin-related diseases such as adenomatous polyposis of the colon, colorectal cancer, basal cell carcinoma, breast cancer, kidney cancer, Wilms tumors, medulloblastoma, ovarian cancer, adrenocortical tumors, gastric cancer, liver cancer, melanoma, pancreatic cancers, prostate cancer, renal cancer, ectopic teeth and taste papillae, skin cancer, pilomatrixoma, anaplastic thyroid carcinoma, and uterine carcinosarcoma, oligodontia, osteoporosis, ageing, degenerative diseases, bedsores, chronic wounds and impaired wound healing, and similar and related diseases, using a therapeutically effective amount of a RNAi agent to Beta-Catenin.
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公开(公告)号:US20150291954A1
公开(公告)日:2015-10-15
申请号:US14371131
申请日:2013-01-08
申请人: NOVARTIS AG
发明人: Brian Richard Bettencourt , David Anton Bumcrot , Dieter Huesken , Satyanarayana Kuchimanchi , Stuart Milstein , Michael Ray Schlabach, JR. , Frank Peter Stegmeier , Markus Warmuth , Jan Weiler
IPC分类号: C12N15/113 , A61K31/713
CPC分类号: C12N15/113 , A61K31/713 , C12N2310/14 , C12N2310/321 , C12N2310/344
摘要: The present disclosure relates to RNAi agents useful in methods of treating Beta-Catenin-related diseases such as adenomatous polyposis of the colon, colorectal cancer, basal cell carcinoma, breast cancer, kidney cancer, Wilms tumors, medulloblastoma, ovarian cancer, adrenocortical tumors, gastric cancer, liver cancer, melanoma, pancreatic cancers, prostate cancer, renal cancer, ectopic teeth and taste papillae, skin cancer, pilomatrixoma, anaplastic thyroid carcinoma, and uterine carcinosarcoma, oligodontia, osteoporosis, ageing, degenerative diseases, bedsores, chronic wounds and impaired wound healing, and similar and related diseases, using a therapeutically effective amount of a RNAi agent to Beta-Catenin.
摘要翻译: 本公开涉及可用于治疗β-联蛋白相关疾病如结肠腺瘤性息肉病,结肠直肠癌,基底细胞癌,乳腺癌,肾癌,Wilms肿瘤,成神经管细胞瘤,卵巢癌,肾上腺皮质肿瘤, 胃癌,肝癌,黑素瘤,胰腺癌,前列腺癌,肾癌,异位牙齿和口腔乳头,皮肤癌,皮质细胞瘤,间变性甲状腺癌和子宫癌肉瘤,少痣,骨质疏松症,衰老,退行性疾病,褥疮,慢性伤口和 损伤的伤口愈合以及类似和相关疾病,使用治疗有效量的RNAi剂到β-联蛋白。
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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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