JP2023529457A - 治療用ポリヌクレオチドに使用するためのグアノシン類似体 - Google Patents

治療用ポリヌクレオチドに使用するためのグアノシン類似体 Download PDF

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JP2023529457A
JP2023529457A JP2022575711A JP2022575711A JP2023529457A JP 2023529457 A JP2023529457 A JP 2023529457A JP 2022575711 A JP2022575711 A JP 2022575711A JP 2022575711 A JP2022575711 A JP 2022575711A JP 2023529457 A JP2023529457 A JP 2023529457A
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polynucleotide
guanosine
nucleosides
antisense oligonucleotide
seq
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ナンナ アルベク
ペーター ハーゲドルン
メッテ ラディフォギッド
リッケ ペデルセン
ヤコブ ラヴィン
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ロシュ イノベーション センター コペンハーゲン エーエス
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
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  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2022575711A 2020-06-09 2021-06-08 治療用ポリヌクレオチドに使用するためのグアノシン類似体 Pending JP2023529457A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20179011 2020-06-09
EP20179011.0 2020-06-09
PCT/EP2021/065266 WO2021249993A1 (en) 2020-06-09 2021-06-08 Guanosine analogues for use in therapeutic polynucleotides

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JP2023529457A true JP2023529457A (ja) 2023-07-10
JP2023529457A5 JP2023529457A5 (https=) 2024-06-14

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JP2022575711A Pending JP2023529457A (ja) 2020-06-09 2021-06-08 治療用ポリヌクレオチドに使用するためのグアノシン類似体

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US (1) US20230212572A1 (https=)
EP (1) EP4162047A1 (https=)
JP (1) JP2023529457A (https=)
CN (1) CN115702243A (https=)
WO (1) WO2021249993A1 (https=)

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JP2017121239A (ja) * 2012-04-02 2017-07-13 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． タンパク質のインビボ産生
WO2018118587A1 (en) * 2016-12-22 2018-06-28 Agenovir Corporation Modified polynucleotides for antiviral therapy

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JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
EP1272630A2 (en) 2000-03-16 2003-01-08 Genetica, Inc. Methods and compositions for rna interference
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
WO2004046160A2 (en) 2002-11-18 2004-06-03 Santaris Pharma A/S Amino-lna, thio-lna and alpha-l-oxy-ln
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
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JP2017121239A (ja) * 2012-04-02 2017-07-13 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． タンパク質のインビボ産生
US20170191064A1 (en) * 2015-11-12 2017-07-06 Roche Innovation Center Copenhagen A/S Oligonucleotides for inducing paternal ube3a expression
WO2018118587A1 (en) * 2016-12-22 2018-06-28 Agenovir Corporation Modified polynucleotides for antiviral therapy

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EP4162047A1 (en) 2023-04-12
CN115702243A (zh) 2023-02-14
WO2021249993A1 (en) 2021-12-16

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