WO2026082165A1 - Acide ribonucléique double brin modifié - Google Patents

Acide ribonucléique double brin modifié

Info

Publication number
WO2026082165A1
WO2026082165A1 PCT/CN2025/128407 CN2025128407W WO2026082165A1 WO 2026082165 A1 WO2026082165 A1 WO 2026082165A1 CN 2025128407 W CN2025128407 W CN 2025128407W WO 2026082165 A1 WO2026082165 A1 WO 2026082165A1
Authority
WO
WIPO (PCT)
Prior art keywords
modification
nucleotides
nucleotide
sequence
deoxy
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
PCT/CN2025/128407
Other languages
English (en)
Chinese (zh)
Inventor
徐宏江
葛兴枫
郑佳佳
陈硕
卢丹丹
杨玲
许雅琼
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Chia Tai Tianqing Pharmaceutical Group Co Ltd
Original Assignee
Chia Tai Tianqing Pharmaceutical Group Co Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Chia Tai Tianqing Pharmaceutical Group Co Ltd filed Critical Chia Tai Tianqing Pharmaceutical Group Co Ltd
Publication of WO2026082165A1 publication Critical patent/WO2026082165A1/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La présente demande appartient au domaine de la biomédecine et concerne un acide ribonucléique double brin modifié (ARNdb), un sel pharmaceutiquement acceptable de celui-ci, un conjugué de ligand de celui-ci, ou une composition pharmaceutique.
PCT/CN2025/128407 2024-10-18 2025-10-17 Acide ribonucléique double brin modifié Pending WO2026082165A1 (fr)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
CN202411464670.9 2024-10-18
CN202411464670 2024-10-18
CN202510081923 2025-01-17
CN202510081923.2 2025-01-17
CN202510241093 2025-02-28
CN202510241093.5 2025-02-28
CN202510949137 2025-07-10
CN202510949137.X 2025-07-10
CN202511485940 2025-10-16
CN202511485940.9 2025-10-16

Publications (1)

Publication Number Publication Date
WO2026082165A1 true WO2026082165A1 (fr) 2026-04-23

Family

ID=99513280

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/CN2025/128407 Pending WO2026082165A1 (fr) 2024-10-18 2025-10-17 Acide ribonucléique double brin modifié

Country Status (1)

Country Link
WO (1) WO2026082165A1 (fr)

Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110582283A (zh) * 2016-11-23 2019-12-17 阿尔尼拉姆医药品有限公司 具有降低的脱靶效应的修饰的rna试剂
WO2020233650A1 (fr) * 2019-05-22 2020-11-26 苏州瑞博生物技术股份有限公司 Acide nucléique, composition pharmaceutique, conjugué, procédé de préparation et utilisation
CN112020556A (zh) * 2018-03-21 2020-12-01 瑞泽恩制药公司 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法
CN112423794A (zh) * 2018-12-28 2021-02-26 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN113164509A (zh) * 2018-09-19 2021-07-23 箭头药业股份有限公司 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法
CN113544272A (zh) * 2018-12-21 2021-10-22 Ionis制药公司 Hsd17b13表达的调节剂
CN116622710A (zh) * 2022-05-27 2023-08-22 北京福元医药股份有限公司 用于抑制c3基因表达的双链核糖核酸及其修饰物、缀合物和用途
CN118339294A (zh) * 2021-12-16 2024-07-12 上海拓界生物医药科技有限公司 一种dsRNA、其制备方法及应用
CN118355121A (zh) * 2021-12-16 2024-07-16 上海拓界生物医药科技有限公司 一种dsRNA、其制备方法及应用
WO2024173593A1 (fr) * 2023-02-14 2024-08-22 Basecure Therapeutics Agents d'arn double brin modifiés

Patent Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110582283A (zh) * 2016-11-23 2019-12-17 阿尔尼拉姆医药品有限公司 具有降低的脱靶效应的修饰的rna试剂
CN112020556A (zh) * 2018-03-21 2020-12-01 瑞泽恩制药公司 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法
CN113164509A (zh) * 2018-09-19 2021-07-23 箭头药业股份有限公司 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法
CN113544272A (zh) * 2018-12-21 2021-10-22 Ionis制药公司 Hsd17b13表达的调节剂
CN112423794A (zh) * 2018-12-28 2021-02-26 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
WO2020233650A1 (fr) * 2019-05-22 2020-11-26 苏州瑞博生物技术股份有限公司 Acide nucléique, composition pharmaceutique, conjugué, procédé de préparation et utilisation
CN118339294A (zh) * 2021-12-16 2024-07-12 上海拓界生物医药科技有限公司 一种dsRNA、其制备方法及应用
CN118355121A (zh) * 2021-12-16 2024-07-16 上海拓界生物医药科技有限公司 一种dsRNA、其制备方法及应用
CN116622710A (zh) * 2022-05-27 2023-08-22 北京福元医药股份有限公司 用于抑制c3基因表达的双链核糖核酸及其修饰物、缀合物和用途
WO2024173593A1 (fr) * 2023-02-14 2024-08-22 Basecure Therapeutics Agents d'arn double brin modifiés

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