CN117377765A - 一种核酸配体及其缀合物、其制备方法和用途 - Google Patents

一种核酸配体及其缀合物、其制备方法和用途 Download PDF

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Publication number
CN117377765A
CN117377765A CN202280037013.9A CN202280037013A CN117377765A CN 117377765 A CN117377765 A CN 117377765A CN 202280037013 A CN202280037013 A CN 202280037013A CN 117377765 A CN117377765 A CN 117377765A
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China
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ligand
nucleic acid
independently
alkyl
compound
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Chinese (zh)
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黄金宇
罗敏
尹科
侯哲
李云飞
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Shanghai Tuojie Biomedical Technology Co ltd
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Shanghai Tuojie Biomedical Technology Co ltd
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
    • C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H13/00—Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids
    • C07H13/12—Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids by acids having the group -X-C(=X)-X-, or halides thereof, in which each X means nitrogen, oxygen, sulfur, selenium or tellurium, e.g. carbonic acid, carbamic acid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/18—Acyclic radicals, substituted by carbocyclic rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/26—Acyclic or carbocyclic radicals, substituted by hetero rings
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (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)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
CN202280037013.9A 2021-07-02 2022-07-01 一种核酸配体及其缀合物、其制备方法和用途 Pending CN117377765A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2021107532254 2021-07-02
CN202110753225 2021-07-02
PCT/CN2022/103275 WO2023274395A1 (zh) 2021-07-02 2022-07-01 一种核酸配体及其缀合物、其制备方法和用途

Publications (1)

Publication Number Publication Date
CN117377765A true CN117377765A (zh) 2024-01-09

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CN202280037013.9A Pending CN117377765A (zh) 2021-07-02 2022-07-01 一种核酸配体及其缀合物、其制备方法和用途

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EP (1) EP4365290A1 (enExample)
JP (1) JP2024524330A (enExample)
KR (1) KR20240028502A (enExample)
CN (1) CN117377765A (enExample)
AU (1) AU2022304946A1 (enExample)
CA (1) CA3225632A1 (enExample)
MX (1) MX2023015423A (enExample)
TW (1) TW202317197A (enExample)
WO (1) WO2023274395A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121270626A (zh) * 2024-06-13 2026-01-06 广州必贝特医药股份有限公司 用于靶向递送寡核苷酸药物的化合物、缀合物及其应用

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JP2024542929A (ja) * 2021-10-05 2024-11-19 セーンジーン バイオ ユーエスエー インコーポレイティド ポリヒドロキシル化されたシクロペンタン誘導体及び使用方法
EP4467645A4 (en) 2022-01-20 2026-01-21 Tuojie Biotech Shanghai Co Ltd DOUBLE-STRANDED RNA, ITS USES AND PREPARATION PROCESS
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
CN119019475B (zh) * 2023-05-24 2026-02-06 北京炫景瑞医药科技有限公司 GalNAc化合物、缀合物、组合物以及它们的用途
CN119019354B (zh) * 2023-05-24 2025-07-22 北京炫景瑞医药科技有限公司 化合物及其制备方法和用途
WO2024240101A1 (zh) * 2023-05-24 2024-11-28 北京炫景瑞医药科技有限公司 GalNAc化合物、缀合物、组合物以及它们的用途
AU2024280186A1 (en) 2023-05-31 2025-12-04 Tuojie Biotech (Shanghai) Co., Ltd. Dsrna targeting lpa and use thereof
KR20260026026A (ko) 2023-06-13 2026-02-25 투오지에 바이오텍 (상하이) 컴퍼니 리미티드 MMP7을 표적으로 하는 siRNA, siRNA 접합체 및 이의 의학적 용도
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
CN117563009B (zh) * 2023-09-01 2025-07-01 杭州天龙药业有限公司 一种含有核糖环或其衍生结构的GalNAc化合物及其寡核苷酸缀合物
WO2025140434A1 (zh) * 2023-12-26 2025-07-03 成都倍特药业股份有限公司 一种肝靶向化合物及其在药物缀合物中的用途
WO2025232840A1 (zh) * 2024-05-09 2025-11-13 石药集团中奇制药技术（石家庄）有限公司 GalNAc衍生物及其寡聚核苷酸缀合物
CN119241613A (zh) * 2024-09-26 2025-01-03 北京悦康科创医药科技股份有限公司 含有核糖环或其衍生结构的GalNAc单体及其在小核酸药物肝靶向递送中的应用
CN120192442B (zh) * 2025-05-22 2025-09-12 北京悦康科创医药科技股份有限公司 一种含核糖环的GalNAc化合物的制备方法
CN120737137B (zh) * 2025-09-08 2025-12-09 北京悦康科创医药科技股份有限公司 一种含芳环骨架的GalNAc化合物及其应用

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AU2013299717B2 (en) 2012-08-06 2018-06-28 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated RNA agents and process for their preparation
CA2921162A1 (en) * 2013-05-01 2014-11-06 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US10570169B2 (en) * 2014-05-22 2020-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
CA2968114A1 (en) * 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
US10246709B2 (en) * 2016-03-07 2019-04-02 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
CN110997919B (zh) * 2017-12-01 2024-04-02 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
CN110959011B (zh) * 2017-12-29 2023-03-28 苏州瑞博生物技术股份有限公司 缀合物及其制备方法和用途
CN112390835A (zh) * 2019-08-14 2021-02-23 苏州瑞博生物技术股份有限公司 肝靶向化合物及缀合物
WO2021254360A1 (zh) 2020-06-16 2021-12-23 上海拓界生物医药科技有限公司 一种碳水化合物分子簇及其制备方法和医药用途

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121270626A (zh) * 2024-06-13 2026-01-06 广州必贝特医药股份有限公司 用于靶向递送寡核苷酸药物的化合物、缀合物及其应用

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JP2024524330A (ja) 2024-07-05
EP4365290A1 (en) 2024-05-08
CA3225632A1 (en) 2023-01-05
WO2023274395A1 (zh) 2023-01-05
MX2023015423A (es) 2024-02-20
TW202317197A (zh) 2023-05-01
AU2022304946A1 (en) 2024-02-15
KR20240028502A (ko) 2024-03-05

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