CN115955973A - 脱靶活性降低的修饰siRNA - Google Patents
脱靶活性降低的修饰siRNA Download PDFInfo
- Publication number
- CN115955973A CN115955973A CN202180048996.1A CN202180048996A CN115955973A CN 115955973 A CN115955973 A CN 115955973A CN 202180048996 A CN202180048996 A CN 202180048996A CN 115955973 A CN115955973 A CN 115955973A
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- sirna
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- alkynyl
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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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- C12N15/09—Recombinant DNA-technology
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- 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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- C12N15/09—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
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/313—Phosphorodithioates
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- C12N2310/00—Structure or type of the nucleic acid
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- 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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- C12N2310/33—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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- 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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- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
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- 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)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202010772542 | 2020-08-04 | ||
| CN2020107725426 | 2020-08-04 | ||
| CN2021102449778 | 2021-03-05 | ||
| CN202110244977 | 2021-03-05 | ||
| CN202110361502 | 2021-04-02 | ||
| CN2021103615027 | 2021-04-02 | ||
| PCT/CN2021/110509 WO2022028462A1 (zh) | 2020-08-04 | 2021-08-04 | 脱靶活性降低的修饰sirna |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN115955973A true CN115955973A (zh) | 2023-04-11 |
Family
ID=80120017
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202180048996.1A Pending CN115955973A (zh) | 2020-08-04 | 2021-08-04 | 脱靶活性降低的修饰siRNA |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20230287418A1 (https=) |
| EP (1) | EP4194553A4 (https=) |
| JP (1) | JP7849353B2 (https=) |
| KR (1) | KR20230043195A (https=) |
| CN (1) | CN115955973A (https=) |
| BR (1) | BR112023002080A2 (https=) |
| CA (1) | CA3190097A1 (https=) |
| TW (1) | TW202214858A (https=) |
| WO (1) | WO2022028462A1 (https=) |
| ZA (1) | ZA202303275B (https=) |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024245410A1 (zh) * | 2023-05-31 | 2024-12-05 | 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 |
| 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 |
| WO2025140299A1 (zh) * | 2023-12-26 | 2025-07-03 | 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 |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019006455A1 (en) | 2017-06-30 | 2019-01-03 | Solstice Biologics, Ltd. | AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF |
| 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 |
| US20240229037A1 (en) * | 2021-04-22 | 2024-07-11 | Tuojie Biotech(Shanghai) Co., Ltd. | SIRNA TARGETING 17Beta-HYDROXYSTEROID DEHYDROGENASE TYPE 13 AND SIRNA CONJUGATE |
| WO2023109932A1 (zh) * | 2021-12-16 | 2023-06-22 | 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 |
| US20260076990A1 (en) | 2022-01-20 | 2026-03-19 | Tuojie Biotech (Shanghai) Co., Ltd. | Dsrna, use thereof and preparation method therefor |
| KR20250123805A (ko) * | 2022-12-13 | 2025-08-18 | 투오지에 바이오텍 (상하이) 컴퍼니 리미티드 | 친유성 단량체를 포함하는 올리고뉴클레오티드 및 비-간 전달에서의 용도 |
| JP2026503329A (ja) * | 2023-02-21 | 2026-01-28 | 成都先導薬物開発股▲フン▼有限公司 | ヌクレオチド類似体、その調製方法及び使用 |
| EP4674965A1 (en) | 2023-03-02 | 2026-01-07 | Tuojie Biotech (Shanghai) Co., Ltd. | Sirna targeting inhibin ?e, sirna conjugate, and medical use thereof |
| WO2024217152A2 (zh) * | 2023-04-21 | 2024-10-24 | 苏州时安生物技术有限公司 | 一种寡聚核酸及其制备方法和应用 |
| AU2024262610A1 (en) | 2023-04-27 | 2025-10-16 | Jiangsu Hengrui Pharmaceuticals Co., Ltd. | Intermediate for preparing oligonucleotide analog or salt thereof, and preparation method therefor |
| AU2024302964A1 (en) | 2023-06-13 | 2025-12-18 | Tuojie Biotech (Shanghai) Co., Ltd. | Sirna targeting mmp7, sirna conjugate and medical use thereof |
| 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 |
| CN121586578A (zh) | 2023-08-24 | 2026-02-27 | 上海拓界生物医药科技有限公司 | 靶向RAGE的RNAi剂及其医药用途 |
| 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 |
| WO2025067317A1 (zh) * | 2023-09-26 | 2025-04-03 | 上海拓界生物医药科技有限公司 | 一种脂质修饰的核酸化合物、其制备方法及用途 |
| 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 |
| 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 |
|---|---|---|---|---|
| EP1798225A1 (en) * | 2004-09-16 | 2007-06-20 | Gifu University | Nucleoside analogues or salts thereof |
| US10131904B2 (en) * | 2008-02-11 | 2018-11-20 | 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 |
| HK1244843A1 (zh) * | 2014-11-17 | 2018-08-17 | Alnylam Pharmaceuticals, Inc. | 载脂蛋白c3(apoc3)irna组合物及其使用方法 |
| JP6457704B2 (ja) * | 2015-12-13 | 2019-01-23 | 日東電工株式会社 | 高活性及びオフターゲット削減のためのsiRNA構造 |
| CN107164379B (zh) * | 2016-08-18 | 2020-06-02 | 广州市锐博生物科技有限公司 | 用于抑制VEGFA靶基因mRNA表达的寡核酸分子及其成套组合物 |
| KR102645243B1 (ko) * | 2016-11-23 | 2024-03-11 | 알닐람 파마슈티칼스 인코포레이티드 | 표적외 효과가 감소된 변형 rna 작용제 |
| WO2019222479A1 (en) * | 2018-05-16 | 2019-11-21 | Alnylam Pharmaceuticals, Inc. | Modified rna agents with reduced off-target effect |
| 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 |
| 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 |
| 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 |
| US20240229037A1 (en) * | 2021-04-22 | 2024-07-11 | Tuojie Biotech(Shanghai) Co., Ltd. | SIRNA TARGETING 17Beta-HYDROXYSTEROID DEHYDROGENASE TYPE 13 AND SIRNA CONJUGATE |
-
2021
- 2021-08-04 TW TW110128792A patent/TW202214858A/zh unknown
- 2021-08-04 BR BR112023002080A patent/BR112023002080A2/pt unknown
- 2021-08-04 KR KR1020237006847A patent/KR20230043195A/ko active Pending
- 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 EP EP21852482.5A patent/EP4194553A4/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
- 2021-08-04 JP JP2023507494A patent/JP7849353B2/ja active Active
-
2023
- 2023-03-02 ZA ZA2023/03275A patent/ZA202303275B/en unknown
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024245410A1 (zh) * | 2023-05-31 | 2024-12-05 | 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 |
| WO2025140299A1 (zh) * | 2023-12-26 | 2025-07-03 | 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 |
| 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 |
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230043195A (ko) | 2023-03-30 |
| TW202214858A (zh) | 2022-04-16 |
| ZA202303275B (en) | 2025-12-17 |
| WO2022028462A1 (zh) | 2022-02-10 |
| JP2023536320A (ja) | 2023-08-24 |
| EP4194553A1 (en) | 2023-06-14 |
| JP7849353B2 (ja) | 2026-04-21 |
| EP4194553A4 (en) | 2024-11-13 |
| US20230287418A1 (en) | 2023-09-14 |
| CA3190097A1 (en) | 2022-02-10 |
| BR112023002080A2 (pt) | 2023-04-25 |
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