JP2024543963A - 高い安定性及び遺伝子サイレンシング活性を有する低分子干渉rna分子のための修飾パターン - Google Patents

高い安定性及び遺伝子サイレンシング活性を有する低分子干渉rna分子のための修飾パターン Download PDF

Info

Publication number
JP2024543963A
JP2024543963A JP2024532428A JP2024532428A JP2024543963A JP 2024543963 A JP2024543963 A JP 2024543963A JP 2024532428 A JP2024532428 A JP 2024532428A JP 2024532428 A JP2024532428 A JP 2024532428A JP 2024543963 A JP2024543963 A JP 2024543963A
Authority
JP
Japan
Prior art keywords
modified
positions
sirna
sense strand
antisense strand
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
JP2024532428A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2023098908A5 (https=
JP2024543963A5 (https=
Inventor
チャン,イー-チュン
ヤン,チー-ファン
チェン,ホイ-ユー
チェン,イー-フェン
Original Assignee
マイクロバイオ (シャンハイ) カンパニー リミテッド
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 マイクロバイオ (シャンハイ) カンパニー リミテッド filed Critical マイクロバイオ (シャンハイ) カンパニー リミテッド
Publication of JP2024543963A publication Critical patent/JP2024543963A/ja
Publication of JPWO2023098908A5 publication Critical patent/JPWO2023098908A5/ja
Publication of JP2024543963A5 publication Critical patent/JP2024543963A5/ja
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/1131Non-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
    • 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
    • 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
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • 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
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • 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
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen

Landscapes

  • 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)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024532428A 2021-12-03 2022-12-02 高い安定性及び遺伝子サイレンシング活性を有する低分子干渉rna分子のための修飾パターン Pending JP2024543963A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CNPCT/CN2021/135465 2021-12-03
CN2021135465 2021-12-03
PCT/CN2022/136350 WO2023098908A1 (en) 2021-12-03 2022-12-02 Modification patterns for small interfering rna molecules with high stability and gene silencing activities

Publications (3)

Publication Number Publication Date
JP2024543963A true JP2024543963A (ja) 2024-11-26
JPWO2023098908A5 JPWO2023098908A5 (https=) 2025-12-09
JP2024543963A5 JP2024543963A5 (https=) 2025-12-09

Family

ID=86611568

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024532428A Pending JP2024543963A (ja) 2021-12-03 2022-12-02 高い安定性及び遺伝子サイレンシング活性を有する低分子干渉rna分子のための修飾パターン

Country Status (9)

Country Link
US (1) US20250101428A1 (https=)
EP (1) EP4441221A1 (https=)
JP (1) JP2024543963A (https=)
KR (1) KR20240130091A (https=)
CN (1) CN118401665A (https=)
AU (1) AU2022400971A1 (https=)
IL (1) IL313116A (https=)
TW (1) TW202342747A (https=)
WO (1) WO2023098908A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118001297B (zh) * 2024-02-01 2025-01-28 中国人民解放军军事科学院军事医学研究院 一种治疗SARS-CoV-2及其变体感染的多功能组合物及其制备方法
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
CN118976041A (zh) * 2024-07-23 2024-11-19 安徽医科大学 一种靶向抑制lncRNA Gm26917基因表达的siRNA在制备治疗脓毒症的急性肺损伤药物中的应用

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10131904B2 (en) * 2008-02-11 2018-11-20 Rxi Pharmaceuticals Corporation Modified RNAi polynucleotides and uses thereof
CN107709561B (zh) * 2015-07-31 2021-04-23 佳康尔研究所股份有限公司 修饰的siRNA及含有该修饰的siRNA的药物组合物
WO2018185253A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Ligand modified double-stranded nucleic acids
JP7365052B2 (ja) * 2017-12-01 2023-10-19 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
CN112423795A (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
JP7610268B2 (ja) * 2019-05-22 2025-01-08 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用

Also Published As

Publication number Publication date
IL313116A (en) 2024-07-01
EP4441221A1 (en) 2024-10-09
US20250101428A1 (en) 2025-03-27
KR20240130091A (ko) 2024-08-28
AU2022400971A1 (en) 2024-06-13
TW202342747A (zh) 2023-11-01
WO2023098908A1 (en) 2023-06-08
CN118401665A (zh) 2024-07-26

Similar Documents

Publication Publication Date Title
JP7625637B2 (ja) B型肝炎ウイルス感染のためのRNAi薬
CN108136206B (zh) 抗乙型肝炎病毒的组合物和药剂及其用途
WO2023098908A1 (en) Modification patterns for small interfering rna molecules with high stability and gene silencing activities
BR112019014282A2 (pt) Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso
CN113507920A (zh) 用于乙型肝炎病毒感染的RNAi剂
JPWO2018193902A1 (ja) マイクロrnaによるb型肝炎ウイルスに対する抗ウイルス効果
WO2023093896A1 (zh) 用于抑制乙型肝炎病毒(hbv)蛋白表达的组合物和方法
US9771589B2 (en) Treating hepatitis virus infection by modulating microRNAs miR-130a, miR-130b, miR-204, or miR-1236
WO2022117085A1 (en) Interfering rnas targeting severe acute respiratory syndrome-associated coronavirus and uses thereof for treating covid-19
WO2023001234A1 (en) Modified small interfering rna molecules with reduced off-target effects
WO2024129886A2 (en) Treatment of mst1 related diseases and disorders
WO2026067447A1 (en) Small Interfering RNAs Targeting ANT2 and Uses Thereof for Disease Treatment
EP4678747A2 (en) Double-stranded rnai agents for targeting and regulating hbv gene expression, and a use thereof
TW202605149A (zh) 靶向調控HBV基因表達的雙鏈RNAi劑及其應用
HK40124050A (zh) 靶向调控hbv基因表达的sirna及其应用
HK40124051A (zh) 靶向调控hbv基因表达的sirna及其应用
HK40124050B (zh) 靶向调控hbv基因表达的sirna及其应用
HK40062579A (en) Rnai agents for hepatitis b virus infection
HK40009627B (en) Rnai agents for hepatitis b virus infection
EA044937B1 (ru) АГЕНТЫ РНКи ПРОТИВ ИНФЕКЦИИ, ВЫЗВАННОЙ ВИРУСОМ ГЕПАТИТА В
EA052767B1 (ru) Средства для рнки альфа-1-антитрипсина (aat), композиции, содержащие средства для рнки aat, и способы их применения
EA053067B1 (ru) АГЕНТЫ РНКи ПРОТИВ ИНФЕКЦИИ, ВЫЗВАННОЙ ВИРУСОМ ГЕПАТИТА В

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20251201

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20251201