JP7557941B2 - オリゴヌクレオチド組成物及びその使用方法 - Google Patents

オリゴヌクレオチド組成物及びその使用方法 Download PDF

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JP7557941B2
JP7557941B2 JP2019566839A JP2019566839A JP7557941B2 JP 7557941 B2 JP7557941 B2 JP 7557941B2 JP 2019566839 A JP2019566839 A JP 2019566839A JP 2019566839 A JP2019566839 A JP 2019566839A JP 7557941 B2 JP7557941 B2 JP 7557941B2
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oligonucleotides
oligonucleotide
sequence
composition
pnpla3
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JP2020524485A (ja
JP2020524485A5 (https=
Inventor
ヴァージェシー，チャンドラ
直樹 岩本
チャールズ ドネル バトラー，デイビット
マラッパン，スブラマニアン
ル，ゲンリャン
ジンシン チャン，ジェイソン
ヴァシパディーカル，ヴィノッド
エンリケ アッポニ，ルチアーノ
マリア ウィスニウスカ，ハンナ
チェン，シアユン
ジン チョ，ヤング
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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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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    • C12N15/09—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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    • A61K47/54—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
    • A61K47/548—Phosphates or phosphonates, e.g. bone-seeking
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K47/50—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
    • 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
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    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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    • 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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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JP2019566839A 2017-06-02 2018-06-01 オリゴヌクレオチド組成物及びその使用方法 Active JP7557941B2 (ja)

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