TW202345861A - 經修飾之間隔體寡聚物及其使用方法 - Google Patents

經修飾之間隔體寡聚物及其使用方法 Download PDF

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TW202345861A
TW202345861A TW112109666A TW112109666A TW202345861A TW 202345861 A TW202345861 A TW 202345861A TW 112109666 A TW112109666 A TW 112109666A TW 112109666 A TW112109666 A TW 112109666A TW 202345861 A TW202345861 A TW 202345861A
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aso
molecule
nucleotides
phosphate
cpsgps
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TW112109666A
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Chinese (zh)
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里歐尼德 畢戈曼
維韋克 庫瑪爾 拉萬施
樂思曼 雅特布
索爾 馬丁尼茲 蒙太羅
勁 洪
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美商艾利格斯醫療公司
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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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    • C12N15/11—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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    • C12N15/113—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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    • C12N2310/351—Conjugate
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    • C12N2320/00—Applications; Uses
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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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
TW112109666A 2022-03-17 2023-03-16 經修飾之間隔體寡聚物及其使用方法 TW202345861A (zh)

Applications Claiming Priority (2)

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US202263321019P 2022-03-17 2022-03-17
US63/321,019 2022-03-17

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TW202345861A true TW202345861A (zh) 2023-12-01

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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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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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CA3255691A1 (en) 2023-09-21
CN119032175A (zh) 2024-11-26
EP4493694A1 (en) 2025-01-22
JP2025509552A (ja) 2025-04-11
US20230383296A1 (en) 2023-11-30
WO2023177808A1 (en) 2023-09-21
AU2023236560A1 (en) 2024-10-03
KR20240158340A (ko) 2024-11-04

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