JP6970187B2 - 修飾オリゴヌクレオチド及び使用方法 - Google Patents

修飾オリゴヌクレオチド及び使用方法 Download PDF

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JP6970187B2
JP6970187B2 JP2019514088A JP2019514088A JP6970187B2 JP 6970187 B2 JP6970187 B2 JP 6970187B2 JP 2019514088 A JP2019514088 A JP 2019514088A JP 2019514088 A JP2019514088 A JP 2019514088A JP 6970187 B2 JP6970187 B2 JP 6970187B2
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oligonucleotide
formula
nucleotides
nucleotide
hbv
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JP2019529406A5 (enExample
JP2019529406A (ja
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グリャズノフ，セルゲイ
ベイゲルマン，レオニド
ホン，ジン
ラジワンシ，ヴィヴェック
モンテロ，サウル マルティネス
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ヤンセン バイオファーマ インク．
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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
    • C12N15/1131—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 viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
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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
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    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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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
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
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    • C12N2310/32—Chemical structure of the sugar
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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 ===---===
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  • Saccharide Compounds (AREA)
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JP2019514088A 2016-09-14 2017-09-14 修飾オリゴヌクレオチド及び使用方法 Expired - Fee Related JP6970187B2 (ja)

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JP2021176346A JP7170820B2 (ja) 2016-09-14 2021-10-28 修飾オリゴヌクレオチド及び使用方法
JP2022175211A JP7478210B2 (ja) 2016-09-14 2022-11-01 修飾オリゴヌクレオチド及び使用方法

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US201662394738P 2016-09-14 2016-09-14
US201662394739P 2016-09-14 2016-09-14
US62/394,739 2016-09-14
US62/394,738 2016-09-14
PCT/US2017/051644 WO2018053185A1 (en) 2016-09-14 2017-09-14 Modified oligonucleotides and methods of use

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US (3) US10793859B2 (enExample)
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KR (3) KR102533038B1 (enExample)
CN (1) CN109952378B (enExample)
AU (2) AU2017326372B2 (enExample)
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IL (2) IL315071A (enExample)
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JP2020533387A (ja) * 2017-09-14 2020-11-19 ヤンセン バイオファーマ インク． 修飾ヌクレオシドホスホロアミダイト
JP2024054157A (ja) * 2017-09-14 2024-04-16 ヤンセン バイオファーマ インク． 修飾ヌクレオシドホスホロアミダイト
JP7478660B2 (ja) 2017-09-14 2024-05-07 ヤンセン バイオファーマ インク． 修飾ヌクレオシドホスホロアミダイト
US12428441B2 (en) 2017-09-14 2025-09-30 Janssen Biopharma, Inc. Modified nucleoside phosphoramidites

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