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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Publication number
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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    • C12N2310/31—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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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
CN202010772542.6 2020-08-04
CN202010772542 2020-08-04
CN202110244977.8 2021-03-05
CN202110244977 2021-03-05
CN202110361502 2021-04-02
CN202110361502.7 2021-04-02
PCT/CN2021/110509 WO2022028462A1 (zh) 2020-08-04 2021-08-04 脱靶活性降低的修饰sirna

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JP2026064108A Division JP2026129742A (ja) 2020-08-04 2026-04-09 オフターゲット活性が低減された修飾ｓｉＲＮＡ

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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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KR (1) KR20230043195A (enExample)
CN (1) CN115955973A (enExample)
BR (1) BR112023002080A2 (enExample)
CA (1) CA3190097A1 (enExample)
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KR20260020955A (ko) 2023-05-31 2026-02-12 투오지에 바이오텍 (상하이) 컴퍼니 리미티드 LPA를 표적화하는 dsRNA 및 이의 응용
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KR20260046111A (ko) 2023-08-01 2026-04-06 투오지에 바이오텍 (상하이) 컴퍼니 리미티드 MAPT를 표적으로 하는 dsRNA 및 이의 의약적 용도
CN121586578A (zh) 2023-08-24 2026-02-27 上海拓界生物医药科技有限公司 靶向RAGE的RNAi剂及其医药用途
KR20260089666A (ko) * 2023-09-14 2026-06-15 아이오니스 파마수티컬즈, 인코포레이티드 Apociii 발현을 감소시키기 위한 화합물 및 방법
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
CN120209036A (zh) * 2023-12-26 2025-06-27 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
CN121175422A (zh) * 2024-10-08 2025-12-19 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
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
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TW202214858A (zh) 2022-04-16
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US20230287418A1 (en) 2023-09-14
CA3190097A1 (en) 2022-02-10
JP2023536320A (ja) 2023-08-24
WO2022028462A1 (zh) 2022-02-10
BR112023002080A2 (pt) 2023-04-25
KR20230043195A (ko) 2023-03-30
CN115955973A (zh) 2023-04-11

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