JP7849353B2 - オフターゲット活性が低減された修飾ｓｉＲＮＡ - Google Patents

オフターゲット活性が低減された修飾ｓｉＲＮＡ

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JP7849353B2
JP7849353B2 JP2023507494A JP2023507494A JP7849353B2 JP 7849353 B2 JP7849353 B2 JP 7849353B2 JP 2023507494 A JP2023507494 A JP 2023507494A JP 2023507494 A JP2023507494 A JP 2023507494A JP 7849353 B2 JP7849353 B2 JP 7849353B2
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sirna
modified
nucleotide
compound
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JP2023536320A5 (enExample
JP2023536320A (ja
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金宇 黄
敏 羅
科 尹
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Tuojie Biotech Shanghai Co Ltd
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Tuojie Biotech Shanghai Co Ltd
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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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    • C12N15/09—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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    • C12N15/09—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
    • C12N15/1136—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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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/319—Chemical structure of the backbone linked by 2'-5' linkages, i.e. having a free 3'-position
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
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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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
JP2023507494A 2020-08-04 2021-08-04 オフターゲット活性が低減された修飾ｓｉＲＮＡ Active JP7849353B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
CN202010772542 2020-08-04
CN202010772542.6 2020-08-04
CN202110244977 2021-03-05
CN202110244977.8 2021-03-05
CN202110361502.7 2021-04-02
CN202110361502 2021-04-02
PCT/CN2021/110509 WO2022028462A1 (zh) 2020-08-04 2021-08-04 脱靶活性降低的修饰sirna

Publications (3)

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JP2023536320A JP2023536320A (ja) 2023-08-24
JP2023536320A5 JP2023536320A5 (enExample) 2024-07-30
JP7849353B2 true JP7849353B2 (ja) 2026-04-21

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US (1) US20230287418A1 (enExample)
EP (1) EP4194553A4 (enExample)
JP (1) JP7849353B2 (enExample)
KR (1) KR20230043195A (enExample)
CN (1) CN115955973A (enExample)
BR (1) BR112023002080A2 (enExample)
CA (1) CA3190097A1 (enExample)
TW (1) TW202214858A (enExample)
WO (1) WO2022028462A1 (enExample)
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EP4467645A4 (en) * 2022-01-20 2026-01-21 Tuojie Biotech Shanghai Co Ltd DOUBLE-STRANDED RNA, ITS USES AND PREPARATION PROCESS
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JP2026507146A (ja) 2023-03-02 2026-02-27 上海拓界生物医薬科技有限公司 インヒビンβＥを標的とするｓｉＲＮＡ、ｓｉＲＮＡ複合体及びその医薬的使用
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CN121569035A (zh) * 2023-07-04 2026-02-24 大睿生物医药科技(上海)有限公司 包含肽的寡核苷酸递送配体
KR20260046111A (ko) 2023-08-01 2026-04-06 투오지에 바이오텍 (상하이) 컴퍼니 리미티드 MAPT를 표적으로 하는 dsRNA 및 이의 의약적 용도
AU2024329194A1 (en) 2023-08-24 2026-02-19 Tuojie Biotech (Shanghai) Co., Ltd. Rage-targeted rnai agent and pharmaceutical use thereof
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WO2025067317A1 (zh) * 2023-09-26 2025-04-03 上海拓界生物医药科技有限公司 一种脂质修饰的核酸化合物、其制备方法及用途
WO2025140299A1 (zh) * 2023-12-26 2025-07-03 广东东阳光药业股份有限公司 一种新型的化合物及其用途
WO2026002138A1 (zh) * 2024-06-27 2026-01-02 成都新泽利医药科技有限公司 特定修饰的双链寡核苷酸及其组合物
CN119410644A (zh) * 2025-01-08 2025-02-11 北京安龙生物医药有限公司 修饰的双链寡核苷酸及其缀合物和组合物
CN119528986B (zh) * 2025-01-23 2025-09-16 北京悦康科创医药科技股份有限公司 一种核苷类似物及其在降低寡核苷酸药物脱靶活性中的应用

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ZA202303275B (en) 2025-12-17
US20230287418A1 (en) 2023-09-14
KR20230043195A (ko) 2023-03-30
CA3190097A1 (en) 2022-02-10
BR112023002080A2 (pt) 2023-04-25
TW202214858A (zh) 2022-04-16
WO2022028462A1 (zh) 2022-02-10
EP4194553A1 (en) 2023-06-14
CN115955973A (zh) 2023-04-11
JP2023536320A (ja) 2023-08-24

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