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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molecule
nucleotides
mesyl
phosphoramidate
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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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    • C12N15/113—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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    • C12N15/113—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
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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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    • 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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    • C12N2320/50—Methods for regulating/modulating their activity
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    • 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 (https=) 2026-03-25

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

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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

Family Cites Families (9)

* 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
KR101707437B1 (ko) * 2012-09-21 2017-02-16 고꾸리쯔 다이가꾸 호우징 오사까 다이가꾸 구아니딘 가교를 갖는 인공 뉴클레오시드 및 올리고뉴클레오티드
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
US20210052631A1 (en) * 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
PE20211783A1 (es) 2018-11-08 2021-09-08 Aligos Therapeutics Inc Polimeros oligonucleotidos que inhiben el transporte de antigenos y metodos
SG11202112741XA (en) * 2019-05-31 2021-12-30 Aligos Therapeutics Inc Modified gapmer oligonucleotides and methods of use
CA3189922A1 (en) * 2020-07-28 2022-02-03 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression

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

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