JP2024536088A - Ｍｅｃｐ２転写物を編集するための組成物及びその方法 - Google Patents

Ｍｅｃｐ２転写物を編集するための組成物及びその方法 Download PDF

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JP2024536088A
JP2024536088A JP2024518725A JP2024518725A JP2024536088A JP 2024536088 A JP2024536088 A JP 2024536088A JP 2024518725 A JP2024518725 A JP 2024518725A JP 2024518725 A JP2024518725 A JP 2024518725A JP 2024536088 A JP2024536088 A JP 2024536088A
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oligonucleotide
nucleic acid
sugar
oligonucleotides
sugars
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Japanese (ja)
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JPWO2023049477A5 (https=
JP2024536088A5 (https=
Inventor
マイケル アッカー，クリストファー
チバタカーン，オナノン
モニアン，パラシャント
シャクティ シヴァリラ，チック
マラッパン，スブラマニアン
ヴァルギーズ，チャンドラ
カンダサミー，パチャムトゥ
ルー，ゲンリャン
ユ，ホイ
チャールズ ドネール バトラー，デイヴィット
エンリケ アポニ，ルチアーノ
護 清水
ミッシェル スタンドレー，ステファニー
ジョン ブーレイ，デイビッド
グジオール ホス，アンドリュー
デサイ，ジガール
デイビッド ゴッドフリー，ジャック
ヤン，ハイリン
直樹 岩本
クマラサミー，ジャヤカンタン
ラマッティーナ，アンソニー
チャンドラー ハーディング，イアン
ターナー，ジェシー
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Wave Life Sciences Pte Ltd
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Wave Life Sciences Pte Ltd
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Publication of JP2024536088A publication Critical patent/JP2024536088A/ja
Publication of JPWO2023049477A5 publication Critical patent/JPWO2023049477A5/ja
Publication of JP2024536088A5 publication Critical patent/JP2024536088A5/ja
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • C07H21/04—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 deoxyribosyl as saccharide radical
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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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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • 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
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    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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  • Chemical & Material Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
JP2024518725A 2021-09-26 2022-09-26 Ｍｅｃｐ２転写物を編集するための組成物及びその方法 Pending JP2024536088A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163248524P 2021-09-26 2021-09-26
US63/248,524 2021-09-26
US202263341391P 2022-05-12 2022-05-12
US63/341,391 2022-05-12
PCT/US2022/044767 WO2023049477A2 (en) 2021-09-26 2022-09-26 Compositions for editing mecp2 transcripts and methods thereof

Publications (3)

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JP2024536088A true JP2024536088A (ja) 2024-10-04
JPWO2023049477A5 JPWO2023049477A5 (https=) 2025-10-06
JP2024536088A5 JP2024536088A5 (https=) 2025-10-06

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US (1) US20250135036A1 (https=)
EP (1) EP4404942A2 (https=)
JP (1) JP2024536088A (https=)
AU (1) AU2022349529A1 (https=)
CA (1) CA3232965A1 (https=)
WO (1) WO2023049477A2 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
CN108135921B (zh) 2015-07-22 2023-10-17 波涛生命科学有限公司 寡核苷酸组合物及其方法
CN109562122A (zh) 2016-06-03 2019-04-02 波涛生命科学有限公司 寡核苷酸、组合物及其方法
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides
US11718638B2 (en) 2017-06-21 2023-08-08 Wave Life Sciences Ltd. Compounds, compositions and methods for synthesis
CN111108096A (zh) 2017-09-18 2020-05-05 波涛生命科学有限公司 寡核苷酸制备技术
IL277889B2 (en) 2018-04-12 2025-01-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods of using them
EP3790596A4 (en) 2018-05-11 2022-04-06 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE
CN113748116A (zh) 2019-03-20 2021-12-03 波涛生命科学有限公司 可用于寡核苷酸制备的技术
AU2024354150A1 (en) * 2023-09-28 2026-03-12 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
WO2026077951A1 (en) 2024-10-07 2026-04-16 Vico Therapeutics B.V. Improved aon for rna editing

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030190626A1 (en) * 2002-04-09 2003-10-09 Vasulinga Ravikumar Phosphorothioate monoester modified oligomers
WO2018213708A1 (en) * 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
US20220387622A1 (en) * 2019-05-21 2022-12-08 Beam Therapeutics Inc. Methods of editing a single nucleotide polymorphism using programmable base editor systems
EP4240849A4 (en) * 2020-11-08 2026-04-01 Wave Life Sciences Ltd OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES

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EP4404942A2 (en) 2024-07-31
WO2023049477A3 (en) 2023-08-31
WO2023049477A2 (en) 2023-03-30
AU2022349529A1 (en) 2024-05-02
US20250135036A1 (en) 2025-05-01
CA3232965A1 (en) 2023-03-30

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