TW202214858A - 脫靶活性降低的修飾siRNA - Google Patents
脫靶活性降低的修飾siRNA Download PDFInfo
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- TW202214858A TW202214858A TW110128792A TW110128792A TW202214858A TW 202214858 A TW202214858 A TW 202214858A TW 110128792 A TW110128792 A TW 110128792A TW 110128792 A TW110128792 A TW 110128792A TW 202214858 A TW202214858 A TW 202214858A
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- 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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- 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
- C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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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
- C12N2310/313—Phosphorodithioates
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/319—Chemical structure of the backbone linked by 2'-5' linkages, i.e. having a free 3'-position
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3235—Chemical structure of the sugar modified ring structure having the O of the ribose replaced by another atom
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- 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
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
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- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
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- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (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)
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- Pharmacology & Pharmacy (AREA)
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Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202010772542.6 | 2020-08-04 | ||
| CN202010772542 | 2020-08-04 | ||
| CN202110244977.8 | 2021-03-05 | ||
| CN202110244977 | 2021-03-05 | ||
| CN202110361502.7 | 2021-04-02 | ||
| CN202110361502 | 2021-04-02 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| TW202214858A true TW202214858A (zh) | 2022-04-16 |
Family
ID=80120017
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW110128792A TW202214858A (zh) | 2020-08-04 | 2021-08-04 | 脫靶活性降低的修飾siRNA |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20230287418A1 (enExample) |
| EP (1) | EP4194553A4 (enExample) |
| JP (1) | JP7849353B2 (enExample) |
| KR (1) | KR20230043195A (enExample) |
| CN (1) | CN115955973A (enExample) |
| BR (1) | BR112023002080A2 (enExample) |
| CA (1) | CA3190097A1 (enExample) |
| TW (1) | TW202214858A (enExample) |
| WO (1) | WO2022028462A1 (enExample) |
| ZA (1) | ZA202303275B (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11597744B2 (en) | 2017-06-30 | 2023-03-07 | Sirius Therapeutics, Inc. | Chiral phosphoramidite auxiliaries and methods of their use |
| TW202302851A (zh) * | 2021-04-02 | 2023-01-16 | 大陸商上海拓界生物醫藥科技有限公司 | B型肝炎病毒siRNA和siRNA綴合物 |
| CA3216332A1 (en) * | 2021-04-22 | 2022-10-27 | Tuojie Biotech (Shanghai) Co., Ltd. | Sirna targeting 17b-hydroxysteroid dehydrogenase type 13 and sirna conjugate |
| 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 |
| TW202340469A (zh) * | 2022-01-20 | 2023-10-16 | 大陸商上海拓界生物醫藥科技有限公司 | 一種dsrna、其應用及製備方法 |
| JP2025541262A (ja) * | 2022-12-13 | 2025-12-18 | 上海拓界生物医薬科技有限公司 | 親油性モノマーを含むオリゴヌクレオチド及びその非肝臓送達における使用 |
| WO2024175013A1 (zh) * | 2023-02-21 | 2024-08-29 | 成都先导药物开发股份有限公司 | 核苷酸类似物及其制备方法和用途 |
| WO2024179573A1 (zh) | 2023-03-02 | 2024-09-06 | 上海拓界生物医药科技有限公司 | 靶向抑制素βE的siRNA、siRNA缀合物及其医药用途 |
| EP4700125A2 (en) * | 2023-04-21 | 2026-02-25 | Suzhou Siran Biotechnology Co., Ltd. | Oligomeric nucleic acid, and preparation method therefore and use thereof |
| CN120957994A (zh) | 2023-04-27 | 2025-11-14 | 上海森辉医药有限公司 | 用于制备寡核苷酸类似物或其盐的中间体及其制备方法 |
| CN121079106A (zh) | 2023-05-31 | 2025-12-05 | 上海拓界生物医药科技有限公司 | 一种靶向LPA的dsRNA及其应用 |
| TW202503060A (zh) | 2023-06-13 | 2025-01-16 | 大陸商上海拓界生物醫藥科技有限公司 | 靶向MMP7的siRNA、siRNA綴合物及其醫藥用途 |
| WO2025007907A1 (zh) * | 2023-07-04 | 2025-01-09 | 大睿生物 | 包含肽的寡核苷酸递送配体 |
| AU2024315297A1 (en) | 2023-08-01 | 2026-02-12 | Tuojie Biotech (Shanghai) Co., Ltd. | Dsrna targeting mapt and pharmaceutical use thereof |
| TW202509217A (zh) | 2023-08-24 | 2025-03-01 | 大陸商上海拓界生物醫藥科技有限公司 | 靶向RAGE的RNAi劑及其醫藥用途 |
| TW202526017A (zh) * | 2023-09-14 | 2025-07-01 | 美商Ionis製藥公司 | 用於減少apociii表現的化合物及方法 |
| AU2024351190A1 (en) * | 2023-09-26 | 2026-03-26 | Tuojie Biotech (Shanghai) Co., Ltd. | Lipid-modified nucleic acid compound, and preparation method and use therefor |
| CN120209036A (zh) * | 2023-12-26 | 2025-06-27 | 广东东阳光药业股份有限公司 | 一种新型的化合物及其用途 |
| 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 |
| CN119410644A (zh) * | 2025-01-08 | 2025-02-11 | 北京安龙生物医药有限公司 | 修饰的双链寡核苷酸及其缀合物和组合物 |
| CN119528986B (zh) * | 2025-01-23 | 2025-09-16 | 北京悦康科创医药科技股份有限公司 | 一种核苷类似物及其在降低寡核苷酸药物脱靶活性中的应用 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP4887500B2 (ja) * | 2004-09-16 | 2012-02-29 | 国立大学法人岐阜大学 | ヌクレオシド類似体またはその塩 |
| CA2715289C (en) * | 2008-02-11 | 2019-12-24 | Rxi Pharmaceuticals Corporation | Modified rnai polynucleotides and uses thereof |
| SG171879A1 (en) * | 2008-12-03 | 2011-07-28 | Marina Biotech Inc | Usirna complexes |
| AU2013299717B2 (en) | 2012-08-06 | 2018-06-28 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated RNA agents and process for their preparation |
| JP2017535552A (ja) * | 2014-11-17 | 2017-11-30 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | アポリポタンパク質C3(APOC3)iRNA組成物およびその使用方法 |
| BR112018008344A2 (pt) * | 2015-12-13 | 2018-10-30 | Nitto Denko Corp | molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição |
| CN107400669A (zh) * | 2016-08-18 | 2017-11-28 | 广州市锐博生物科技有限公司 | 用于抑制HIF1A靶基因mRNA表达的寡核酸分子及其成套组合物 |
| US11504391B1 (en) * | 2016-11-23 | 2022-11-22 | Alnylam Pharmaceuticals, Inc. | Modified RNA agents with reduced off-target effect |
| JP7384833B2 (ja) * | 2018-05-16 | 2023-11-21 | アルニラム・ファーマシューティカルズ・インコーポレーテッド | オフターゲット効果が低下した修飾rna剤 |
| CN111378657B (zh) * | 2018-12-28 | 2024-03-15 | 苏州瑞博生物技术股份有限公司 | 抑制COL1A1基因表达的siRNA、含有该siRNA的药物组合物及其用途 |
| CN111378655B (zh) * | 2018-12-28 | 2024-03-19 | 苏州瑞博生物技术股份有限公司 | 抑制CTGF基因表达的siRNA、含有该siRNA的药物组合物及其用途 |
| CN111378656B (zh) * | 2018-12-28 | 2022-07-26 | 苏州瑞博生物技术股份有限公司 | 一种抑制埃博拉病毒的核酸、含有该核酸的药物组合物及其用途 |
| CA3216332A1 (en) * | 2021-04-22 | 2022-10-27 | Tuojie Biotech (Shanghai) Co., Ltd. | Sirna targeting 17b-hydroxysteroid dehydrogenase type 13 and sirna conjugate |
-
2021
- 2021-08-04 KR KR1020237006847A patent/KR20230043195A/ko active Pending
- 2021-08-04 TW TW110128792A patent/TW202214858A/zh unknown
- 2021-08-04 EP EP21852482.5A patent/EP4194553A4/en active Pending
- 2021-08-04 BR BR112023002080A patent/BR112023002080A2/pt unknown
- 2021-08-04 JP JP2023507494A patent/JP7849353B2/ja active Active
- 2021-08-04 WO PCT/CN2021/110509 patent/WO2022028462A1/zh not_active Ceased
- 2021-08-04 CA CA3190097A patent/CA3190097A1/en active Pending
- 2021-08-04 US US18/019,763 patent/US20230287418A1/en active Pending
- 2021-08-04 CN CN202180048996.1A patent/CN115955973A/zh active Pending
-
2023
- 2023-03-02 ZA ZA2023/03275A patent/ZA202303275B/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4194553A4 (en) | 2024-11-13 |
| CA3190097A1 (en) | 2022-02-10 |
| JP7849353B2 (ja) | 2026-04-21 |
| KR20230043195A (ko) | 2023-03-30 |
| BR112023002080A2 (pt) | 2023-04-25 |
| WO2022028462A1 (zh) | 2022-02-10 |
| CN115955973A (zh) | 2023-04-11 |
| EP4194553A1 (en) | 2023-06-14 |
| JP2023536320A (ja) | 2023-08-24 |
| US20230287418A1 (en) | 2023-09-14 |
| ZA202303275B (en) | 2025-12-17 |
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