CA3225632A1 - Nucleic acid ligand and conjugate thereof, and preparation method therefor and use thereof - Google Patents

Nucleic acid ligand and conjugate thereof, and preparation method therefor and use thereof Download PDF

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Publication number
CA3225632A1
CA3225632A1 CA3225632A CA3225632A CA3225632A1 CA 3225632 A1 CA3225632 A1 CA 3225632A1 CA 3225632 A CA3225632 A CA 3225632A CA 3225632 A CA3225632 A CA 3225632A CA 3225632 A1 CA3225632 A1 CA 3225632A1
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Prior art keywords
nhac
oac
oacoac
odmtr
date received
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Pending
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CA3225632A
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English (en)
French (fr)
Inventor
Jinyu Huang
Min Luo
Ke YIN
Zhe HOU
Yunfei Li
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Tuojie Biotech Shanghai Co Ltd
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Tuojie Biotech Shanghai Co Ltd
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Application filed by Tuojie Biotech Shanghai Co Ltd filed Critical Tuojie Biotech Shanghai Co Ltd
Publication of CA3225632A1 publication Critical patent/CA3225632A1/en
Pending legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02Acyclic radicals, not substituted by cyclic structures
    • C07H15/04Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H13/00Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids
    • C07H13/12Compounds 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/18Acyclic radicals, substituted by carbocyclic rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/26Acyclic or carbocyclic radicals, substituted by hetero rings
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special 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)
CA3225632A 2021-07-02 2022-07-01 Nucleic acid ligand and conjugate thereof, and preparation method therefor and use thereof Pending CA3225632A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN202110753225 2021-07-02
CN202110753225.4 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
CA3225632A1 true CA3225632A1 (en) 2023-01-05

Family

ID=84690494

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3225632A Pending CA3225632A1 (en) 2021-07-02 2022-07-01 Nucleic acid ligand and conjugate thereof, and preparation method therefor and use thereof

Country Status (9)

Country Link
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)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN119143818B (zh) * 2024-06-13 2025-10-31 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
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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
JP2024524330A (ja) 2024-07-05
CN117377765A (zh) 2024-01-09
EP4365290A1 (en) 2024-05-08
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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