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公开(公告)号:US20190233817A1
公开(公告)日:2019-08-01
申请号:US16269282
申请日:2019-02-06
发明人: Jan Weiler , William Chutkow , Jeremy Lee Baryza , Andrew Krueger , Junping Zhao
IPC分类号: C12N15/113 , C07H21/02 , A61K31/713
CPC分类号: C12N15/113 , A61K31/713 , C07H21/02 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/341 , C12N2310/344 , C12N2310/351 , C12N2310/3515 , C12N2320/31 , C12N2310/3521 , C12N2310/3525 , C12N2310/3533
摘要: The present disclosure relates to compositions and methods for treating APOC3-related diseases such as: hypertriglyceridemia (e.g., Type V Hypertriglyceridemia), abnormal lipid metabolism, abnormal cholesterol metabolism, atherosclerosis, hyperlipidemia, diabetes, including Type 2 diabetes, obesity, cardiovascular disease, and coronary artery disease, among other disorders relating to abnormal metabolism or otherwise, using a therapeutically effective amount of a RNAi agent to APOC3.
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公开(公告)号:US20190062744A1
公开(公告)日:2019-02-28
申请号:US16123554
申请日:2018-09-06
发明人: Christine Esau , Bridget Lollo , C. Frank Bennett , Susan M. Freier , Richard H. Griffey , Brenda F. Baker , Timothy A. Vickers , Eric G. Marcusson , Erich Koller , Eric E. Swayze , Ravi Jain , Balkrishen Bhat , Eigen Peralta
IPC分类号: C12N15/113 , C12N15/11
CPC分类号: C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/14 , C12N2310/141 , C12N2310/31 , C12N2310/315 , C12N2310/317 , C12N2310/3181 , C12N2310/32 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/11 , C12N2320/30 , C12N2320/50 , C12N2310/3525
摘要: Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.
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公开(公告)号:US20180320181A1
公开(公告)日:2018-11-08
申请号:US16021165
申请日:2018-06-28
IPC分类号: C12N15/113 , C12N15/11
CPC分类号: C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/15 , C12N2310/3181 , C12N2310/3231 , C12N2310/341 , C12N2310/3513 , C12N2310/3515 , C12N2310/53 , C12N2320/52
摘要: A method of reducing the level of a transcription product in a cell comprising contacting with the cell a composition comprising a double-stranded nucleic acid complex comprising a first nucleic acid strand annealed to a second nucleic acid strand, wherein: (i) the first nucleic acid strand hybridizes to the transcription product and comprises (a) a region consisting of at least 4 consecutive nucleotides that are recognized by RNase H when the strand is hybridized to the transcription product, (b) one or more nucleotide analogs located on 5′ terminal side of the region, (c) one or more nucleotide analogs located on 3′ terminal side of the region and (d) a total number of nucleotides and nucleotide analogs ranging from 8 to 35 nucleotides and (ii) the second nucleic acid strand comprises (a) nucleotides and optionally nucleotide analogs and (b) at least 4 consecutive RNA nucleotides.
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公开(公告)号:US20180312847A1
公开(公告)日:2018-11-01
申请号:US15971970
申请日:2018-05-04
发明人: Nanna ALBÆK , Maj HEDTJÄRN , Marie Wickstrom LINDHOLM , Niels Fisker NIELSEN , Andreas PETRI , Jacob RAVN
IPC分类号: C12N15/113 , A61K47/54 , A61K31/712
CPC分类号: C12N15/1137 , A61K31/712 , A61K47/545 , A61K47/549 , A61K47/554 , C12N15/113 , C12N2310/11 , C12N2310/3231 , C12N2310/341 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , C12N2330/30
摘要: The present invention relates to oligomeric compounds and conjugates thereof that target Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) PCSK9 mRNA in a cell, leading to reduced expression of PCSK9. Reduction of PCSK9 expression is beneficial for a range of medical disorders, such as hypercholesterolemia and related disorders.
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5.
公开(公告)号:US20180312536A1
公开(公告)日:2018-11-01
申请号:US15953290
申请日:2018-04-13
发明人: Sukumar Sakamuri , Curt W. Bradshaw , Son Lam , Joseph Stock , Edward Hyungsuk Ha , Laxman Eltepu , Dingguo Liu , Bin Liu , Giuseppe Dello Iacono , Bryan R. Meade , Ayman Kabakibi
IPC分类号: C07H21/04 , A61K31/7125 , A61K47/68 , C07K16/28 , C07K7/06 , A61P35/00 , A61K39/00 , A61K39/39 , A61K47/60
CPC分类号: C12N15/117 , C12N2310/17 , C12N2310/311 , C12N2310/315 , C12N2310/332 , C12N2310/3513 , C12N2310/3515 , C12N2310/51
摘要: Immunomodulating polynucleotides are disclosed. The immunomodulating polynucleotides may contain 5-modified uridine, 5-modified cytidine, a total of from 6 to 16 nucleotides, and/or one or more abasic spacers and/or internucleoside phosphotriesters. Also disclosed are conjugates containing a targeting moiety and one or more immunomodulating polynucleotides. The immunomodulating polynucleotides and conjugates may further contain one or more auxiliary moieties. Also disclosed are compositions containing the immunomodulating polynucleotides or the conjugates containing one or more stereochemically enriched internucleoside phosphorothioates. Further disclosed are pharmaceutical compositions containing the immunomodulating polynucleotides or the conjugates and methods of their use.
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公开(公告)号:US10072265B2
公开(公告)日:2018-09-11
申请号:US15664005
申请日:2017-07-31
IPC分类号: C12N15/113 , C12N15/11
CPC分类号: C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/14 , C12N2310/141 , C12N2310/31 , C12N2310/315 , C12N2310/317 , C12N2310/3181 , C12N2310/32 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/11 , C12N2320/30 , C12N2320/50 , C12N2310/3525
摘要: Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.
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公开(公告)号:US20180251766A1
公开(公告)日:2018-09-06
申请号:US15900169
申请日:2018-02-20
申请人: ETH Zurich
发明人: Markus Stoffel , Mirko Trajkovski
IPC分类号: A61K48/00 , C12N15/115 , A61K31/7088 , A61K45/06
CPC分类号: C12N15/115 , A61K31/7088 , A61K45/06 , C12N15/113 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/341 , C12N2310/346 , C12N2310/3515 , C12N2320/30 , C12N2320/31 , C12N2320/35
摘要: Provided herein are methods and compositions for the treatment of metabolic disorders. Also provided herein are methods and compositions for the reduction of blood glucose level, the reduction of gluceoneogenesis, the improvement of insulin resistance and the reduction of plasma cholesterol level. In certain embodiments, the methods comprise inhibiting the activity of miR-103. In certain embodiments, the methods comprise inhibiting the activity of miR-107. In certain embodiments, the activity of both miR-103 and miR-107 is inhibited. In certain embodiments, such methods comprise administering a compound comprising an oligonucleotide targeted to a microRNA.
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8.
公开(公告)号:US20180245084A1
公开(公告)日:2018-08-30
申请号:US15876454
申请日:2018-01-22
IPC分类号: C12N15/113 , A61K9/00 , A61K47/12
CPC分类号: C12N15/1138 , A61K9/0031 , A61K9/0053 , A61K31/7008 , A61K31/7105 , A61K31/7115 , A61K31/712 , A61K31/713 , A61K47/12 , A61K47/549 , C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , C12N2320/51 , C12N2310/3525
摘要: Provided herein are compositions and methods for non-parenteral delivery of conjugated oligomeric compounds. In certain embodiments, compositions and methods are provided for oral delivery of conjugated oligomeric compounds. In certain embodiments, the oligomeric compounds are conjugated to one or more N-acetylgalactosamines or N-acetylgalactosamine analogues.
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公开(公告)号:US20180216114A1
公开(公告)日:2018-08-02
申请号:US15747571
申请日:2016-07-26
IPC分类号: C12N15/113 , A61K47/14 , A61K31/713 , A61K9/00
CPC分类号: C12N15/1137 , A61K9/0019 , A61K31/713 , A61K47/14 , C12N2310/14 , C12N2310/3125 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/335 , C12N2310/351 , C12N2310/3515 , C12N2310/3521 , C12N2310/3533
摘要: The present invention relates to RNAi agents, e.g., double stranded RNAi agents, targeting a xanthine dehydrogenase (XDH) gene, and methods of using such double stranded RNAi agents to inhibit expression of an XDH gene and methods of treating subjects having an XDH-associated disease.
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公开(公告)号:US20180216113A1
公开(公告)日:2018-08-02
申请号:US15747433
申请日:2016-08-18
发明人: Andrew Dillin , Hyun-Eui Kim
IPC分类号: C12N15/113 , A61K31/336 , A61K31/4458 , A61K31/198 , A61K31/205 , A61P43/00
CPC分类号: C12N15/1137 , A61K31/198 , A61K31/205 , A61K31/336 , A61K31/4458 , A61K31/7105 , A61K31/712 , A61K31/7125 , A61K31/713 , A61P43/00 , C12N2310/11 , C12N2310/14 , C12N2310/3233 , C12N2310/3515 , C12N2310/531 , C12Y203/01021
摘要: The present disclosure provides methods of reducing protein misfolding and/or aggregation in a cell. The present disclosure provides methods of treating diseases and disorders associated with protein misfolding and/or aggregation.
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