JP2025509552A - 修飾ギャップマーオリゴマー及びその使用方法 - Google Patents

修飾ギャップマーオリゴマー及びその使用方法 Download PDF

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JP2025509552A
JP2025509552A JP2024554760A JP2024554760A JP2025509552A JP 2025509552 A JP2025509552 A JP 2025509552A JP 2024554760 A JP2024554760 A JP 2024554760A JP 2024554760 A JP2024554760 A JP 2024554760A JP 2025509552 A JP2025509552 A JP 2025509552A
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aso
molecule
nucleotides
mesyl
phosphoramidate
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JP2025509552A5 (enExample
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ベイジェルマン，レオニド
クマル ラジュワンシ，ヴィヴェク
エルテプ，ラクスマン
マルティネス モンテロ，サウル
ホン，ジン
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アリゴス セラピューティクス，インコーポレーテッド
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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
    • 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
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    • 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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    • C12N15/113—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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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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
    • 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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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
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    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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JP2024554760A 2022-03-17 2023-03-16 修飾ギャップマーオリゴマー及びその使用方法 Pending JP2025509552A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263321019P 2022-03-17 2022-03-17
US63/321,019 2022-03-17
PCT/US2023/015398 WO2023177808A1 (en) 2022-03-17 2023-03-16 Modified gapmer oligomers and methods of use thereof

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JP2025509552A true JP2025509552A (ja) 2025-04-11
JP2025509552A5 JP2025509552A5 (enExample) 2026-03-25

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US (1) US20230383296A1 (enExample)
EP (1) EP4493694A1 (enExample)
JP (1) JP2025509552A (enExample)
KR (1) KR20240158340A (enExample)
CN (1) CN119032175A (enExample)
AU (1) AU2023236560A1 (enExample)
CA (1) CA3255691A1 (enExample)
TW (1) TW202345861A (enExample)
WO (1) WO2023177808A1 (enExample)

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CN118680948B (zh) * 2023-10-20 2025-08-22 思合基因(北京)生物科技有限公司 寡核苷酸及其在抗乙型肝炎病毒中的应用

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030203862A1 (en) * 1998-03-26 2003-10-30 Miraglia Loren J. Antisense modulation of MDM2 expression
US20150247141A1 (en) * 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
EP2899197B1 (en) * 2012-09-21 2017-03-22 Osaka University Oligonucleotide and artificial nucleoside having guanidine bridge
GB201408623D0 (en) * 2014-05-15 2014-07-02 Santaris Pharma As Oligomers and oligomer conjugates
TW201718618A (zh) * 2015-09-18 2017-06-01 田邊三菱製藥股份有限公司 架橋型核酸ＧｕＮＡ，其製造方法，及中間體化合物
EP3353305A4 (en) * 2015-09-25 2019-09-18 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US20200147124A1 (en) 2018-11-08 2020-05-14 Aligos Therapeutics, Inc. S-antigen transport inhibiting oligonucleotide polymers and methods
KR20220024192A (ko) * 2019-05-31 2022-03-03 알리고스 테라퓨틱스 인코포레이티드 변형된 갭머 올리고뉴클레오타이드 및 사용 방법
MX2023001222A (es) * 2020-07-28 2023-04-26 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir la expresion de app.

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TW202345861A (zh) 2023-12-01
CA3255691A1 (en) 2023-09-21
CN119032175A (zh) 2024-11-26
EP4493694A1 (en) 2025-01-22
US20230383296A1 (en) 2023-11-30
WO2023177808A1 (en) 2023-09-21
AU2023236560A1 (en) 2024-10-03
KR20240158340A (ko) 2024-11-04

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