CN110997692B - 寡核苷酸组合物及其使用方法 - Google Patents

寡核苷酸组合物及其使用方法 Download PDF

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Publication number
CN110997692B
CN110997692B CN201880049516.1A CN201880049516A CN110997692B CN 110997692 B CN110997692 B CN 110997692B CN 201880049516 A CN201880049516 A CN 201880049516A CN 110997692 B CN110997692 B CN 110997692B
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oligonucleotides
oligonucleotide
composition
sequence
chiral
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Chinese (zh)
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CN110997692A (zh
Inventor
C·瓦格斯
N·岩本
D·C·D·巴特勒
S·马拉帕
陆根良
张敬新
V·瓦蒂帕迪卡尔
L·H·阿波尼
H·M·维斯涅夫斯卡
成夏耘
曺永镇
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
Pfizer Inc
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • 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
    • C12N15/1137—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 against enzymes
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • 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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    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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    • A61K47/548—Phosphates or phosphonates, e.g. bone-seeking
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
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    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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    • A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P3/06—Antihyperlipidemics
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    • C12Y301/01003—Triacylglycerol lipase (3.1.1.3)

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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CN201880049516.1A 2017-06-02 2018-06-01 寡核苷酸组合物及其使用方法 Active CN110997692B (zh)

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US201762514769P 2017-06-02 2017-06-02
US62/514,769 2017-06-02
US201862670698P 2018-05-11 2018-05-11
US62/670,698 2018-05-11
PCT/US2018/035721 WO2018223081A1 (en) 2017-06-02 2018-06-01 Oligonucleotide compositions and methods of use thereof

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CN110997692B true CN110997692B (zh) 2025-01-07

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US (2) US11597927B2 (https=)
EP (1) EP3630789A4 (https=)
JP (2) JP7557941B2 (https=)
CN (2) CN110997692B (https=)
CA (1) CA3065523A1 (https=)
TW (1) TW201904587A (https=)
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