JP7427608B2 - 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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Publication number
JP7427608B2
JP7427608B2 JP2020562157A JP2020562157A JP7427608B2 JP 7427608 B2 JP7427608 B2 JP 7427608B2 JP 2020562157 A JP2020562157 A JP 2020562157A JP 2020562157 A JP2020562157 A JP 2020562157A JP 7427608 B2 JP7427608 B2 JP 7427608B2
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dmd
oligonucleotide
dmd oligonucleotide
present disclosure
disclosure provides
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JPWO2019217784A5 (enExample
JP2021522803A (ja
Inventor
ジンシン チャン，ジェイソン
レディ ボミネニ，ゴパル
マラッパン，スブラマニアン
ディヴァカラメノン，セトゥマドハヴァン
チャールズ ドネール バトラー，デイヴィット
ルー，ゲンリャン
ヤン，ハイリン
護 清水
モニアン，パラシャント
ヴァルギーズ，チャンドラ
直樹 岩本
シャクティ シヴァリラ，チック
コターリ，ナヤンタラ
フィーゲン ダービン，アン
ラマサミー，セルヴィ
カンダサミー，パチャムトゥ
クマラサミー，ジャヤカンタン
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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Priority claimed from PCT/US2019/027109 external-priority patent/WO2019200185A1/en
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Publication of JPWO2019217784A5 publication Critical patent/JPWO2019217784A5/ja
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H1/00—Processes for the preparation of sugar derivatives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020562157A 2018-05-11 2019-05-10 オリゴヌクレオチド組成物及びその使用方法 Active JP7427608B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024008384A JP2024056718A (ja) 2018-05-11 2024-01-24 オリゴヌクレオチド組成物及びその使用方法

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201862670709P 2018-05-11 2018-05-11
US62/670,709 2018-05-11
US201862715684P 2018-08-07 2018-08-07
US62/715,684 2018-08-07
US201862723375P 2018-08-27 2018-08-27
US62/723,375 2018-08-27
US201862776432P 2018-12-06 2018-12-06
US62/776,432 2018-12-06
PCT/US2019/027109 WO2019200185A1 (en) 2018-04-12 2019-04-11 Oligonucleotide compositions and methods of use thereof
USPCT/US2019/027109 2019-04-11
PCT/US2019/031672 WO2019217784A1 (en) 2018-05-11 2019-05-10 Oligonucleotide compositions and methods of use thereof

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Publications (3)

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JP2021522803A JP2021522803A (ja) 2021-09-02
JPWO2019217784A5 JPWO2019217784A5 (enExample) 2023-10-31
JP7427608B2 true JP7427608B2 (ja) 2024-02-05

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JP2024008384A Pending JP2024056718A (ja) 2018-05-11 2024-01-24 オリゴヌクレオチド組成物及びその使用方法

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US (1) US12391942B2 (enExample)
EP (1) EP3790596A4 (enExample)
JP (2) JP7427608B2 (enExample)
CN (1) CN112218664A (enExample)
AU (1) AU2019265904A1 (enExample)
CA (1) CA3098624A1 (enExample)
SG (1) SG11202010131QA (enExample)
TW (1) TWI844541B (enExample)
WO (1) WO2019217784A1 (enExample)

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