CN118401665A - 具有高稳定性和基因沉默活性的小干扰rna分子的修饰模式 - Google Patents
具有高稳定性和基因沉默活性的小干扰rna分子的修饰模式 Download PDFInfo
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- CN118401665A CN118401665A CN202280080053.1A CN202280080053A CN118401665A CN 118401665 A CN118401665 A CN 118401665A CN 202280080053 A CN202280080053 A CN 202280080053A CN 118401665 A CN118401665 A CN 118401665A
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
- 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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- C12N15/00—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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- 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
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- 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]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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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)
- 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)
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 (1)
| Publication Number | Publication Date |
|---|---|
| CN118401665A true CN118401665A (zh) | 2024-07-26 |
Family
ID=86611568
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202280080053.1A Pending CN118401665A (zh) | 2021-12-03 | 2022-12-02 | 具有高稳定性和基因沉默活性的小干扰rna分子的修饰模式 |
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)
| 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 |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN104975020A (zh) * | 2008-02-11 | 2015-10-14 | 阿克赛医药公司 | 经修饰的RNAi多核苷酸及其用途 |
| WO2017022650A1 (ja) * | 2015-07-31 | 2017-02-09 | 株式会社ジーンケア研究所 | 修飾siRNA及びそれを含む医薬組成物 |
| WO2018185253A1 (en) * | 2017-04-05 | 2018-10-11 | Silence Therapeutics Gmbh | Ligand modified double-stranded nucleic acids |
| WO2019105419A1 (zh) * | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | 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 |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
-
2022
- 2022-12-02 JP JP2024532428A patent/JP2024543963A/ja active Pending
- 2022-12-02 KR KR1020247022063A patent/KR20240130091A/ko active Pending
- 2022-12-02 CN CN202280080053.1A patent/CN118401665A/zh active Pending
- 2022-12-02 US US18/715,175 patent/US20250101428A1/en active Pending
- 2022-12-02 AU AU2022400971A patent/AU2022400971A1/en active Pending
- 2022-12-02 EP EP22900712.5A patent/EP4441221A1/en active Pending
- 2022-12-02 IL IL313116A patent/IL313116A/en unknown
- 2022-12-02 WO PCT/CN2022/136350 patent/WO2023098908A1/en not_active Ceased
- 2022-12-02 TW TW111146424A patent/TW202342747A/zh unknown
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN104975020A (zh) * | 2008-02-11 | 2015-10-14 | 阿克赛医药公司 | 经修饰的RNAi多核苷酸及其用途 |
| WO2017022650A1 (ja) * | 2015-07-31 | 2017-02-09 | 株式会社ジーンケア研究所 | 修飾siRNA及びそれを含む医薬組成物 |
| WO2018185253A1 (en) * | 2017-04-05 | 2018-10-11 | Silence Therapeutics Gmbh | Ligand modified double-stranded nucleic acids |
| WO2019105419A1 (zh) * | 2017-12-01 | 2019-06-06 | 苏州瑞博生物技术有限公司 | 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 |
Non-Patent Citations (2)
| Title |
|---|
| FOSTER, D.J.等: "Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates", MOLECULAR THERAPY, vol. 26, no. 3, 31 March 2018 (2018-03-31), pages 708 - 717 * |
| PAVLOVA, A.S.等: "An Influence of Modification with Phosphoryl Guanidine Combined with a 2\'-O-Methyl or 2\'-Fluoro Group on the Small-Interfering-RNA Effect", INTERNATIONAL JOURNAL OF MOLECULAR SCIENCE, vol. 22, 10 September 2021 (2021-09-10), pages 9784 * |
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 |
| JP2024543963A (ja) | 2024-11-26 |
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