JP2018518201A5 - - Google Patents
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- Publication number
- JP2018518201A5 JP2018518201A5 JP2018518771A JP2018518771A JP2018518201A5 JP 2018518201 A5 JP2018518201 A5 JP 2018518201A5 JP 2018518771 A JP2018518771 A JP 2018518771A JP 2018518771 A JP2018518771 A JP 2018518771A JP 2018518201 A5 JP2018518201 A5 JP 2018518201A5
- Authority
- JP
- Japan
- Prior art keywords
- sirna
- amine
- containing compound
- pharmaceutically acceptable
- cholesterol
- 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.)
- Granted
Links
- 108020004459 Small Interfering RNA Proteins 0.000 claims 24
- 239000002924 silencing RNA Substances 0.000 claims 24
- HVYWMOMLDIMFJA-DPAQBDIFSA-N (3β)-Cholest-5-en-3-ol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 18
- 150000001875 compounds Chemical class 0.000 claims 14
- 150000001412 amines Chemical class 0.000 claims 13
- 229940107161 Cholesterol Drugs 0.000 claims 9
- 235000012000 cholesterol Nutrition 0.000 claims 9
- 239000008194 pharmaceutical composition Substances 0.000 claims 9
- 239000003937 drug carrier Substances 0.000 claims 7
- 150000002632 lipids Chemical group 0.000 claims 7
- 239000002202 Polyethylene glycol Substances 0.000 claims 6
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 6
- 229920001223 polyethylene glycol Polymers 0.000 claims 6
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-ACETYL-D-GALACTOSAMINE Chemical compound CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 claims 5
- 125000005647 linker group Chemical group 0.000 claims 5
- 239000004698 Polyethylene (PE) Substances 0.000 claims 4
- 229920000972 Sense strand Polymers 0.000 claims 4
- 230000027455 binding Effects 0.000 claims 4
- 125000004122 cyclic group Chemical group 0.000 claims 4
- 239000002773 nucleotide Substances 0.000 claims 4
- 125000003729 nucleotide group Chemical group 0.000 claims 4
- -1 polyethylene Polymers 0.000 claims 4
- 229920000573 polyethylene Polymers 0.000 claims 4
- GZCGUPFRVQAUEE-KCDKBNATSA-N D-(+)-Galactose Natural products OC[C@@H](O)[C@H](O)[C@H](O)[C@@H](O)C=O GZCGUPFRVQAUEE-KCDKBNATSA-N 0.000 claims 3
- 208000006454 Hepatitis Diseases 0.000 claims 3
- 231100000283 hepatitis Toxicity 0.000 claims 3
- 229910052739 hydrogen Inorganic materials 0.000 claims 3
- 239000001257 hydrogen Substances 0.000 claims 3
- 229910052757 nitrogen Inorganic materials 0.000 claims 3
- 229910052717 sulfur Inorganic materials 0.000 claims 3
- 230000000692 anti-sense Effects 0.000 claims 2
- 230000001684 chronic Effects 0.000 claims 2
- 125000001183 hydrocarbyl group Chemical group 0.000 claims 2
- 150000002431 hydrogen Chemical class 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 150000002829 nitrogen Chemical group 0.000 claims 2
- IJGRMHOSHXDMSA-UHFFFAOYSA-N nitrogen Chemical compound N#N IJGRMHOSHXDMSA-UHFFFAOYSA-N 0.000 claims 2
- 229910052760 oxygen Inorganic materials 0.000 claims 2
- 125000000548 ribosyl group Chemical group C1([C@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 2
- 206010073071 Hepatocellular carcinoma Diseases 0.000 claims 1
- 229920001985 Small interfering RNA Polymers 0.000 claims 1
- 125000002252 acyl group Chemical group 0.000 claims 1
- 125000001931 aliphatic group Chemical group 0.000 claims 1
- 125000003118 aryl group Chemical group 0.000 claims 1
- 102000005427 asialoglycoprotein receptor family Human genes 0.000 claims 1
- 108010006523 asialoglycoprotein receptor family Proteins 0.000 claims 1
- 229910052799 carbon Inorganic materials 0.000 claims 1
- 239000005547 deoxyribonucleotide Substances 0.000 claims 1
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- RTZKZFJDLAIYFH-UHFFFAOYSA-N ether Substances CCOCC RTZKZFJDLAIYFH-UHFFFAOYSA-N 0.000 claims 1
- 150000002170 ethers Chemical class 0.000 claims 1
- 229910052731 fluorine Inorganic materials 0.000 claims 1
- 239000011737 fluorine Substances 0.000 claims 1
- YCKRFDGAMUMZLT-UHFFFAOYSA-N fluorine atom Chemical compound [F] YCKRFDGAMUMZLT-UHFFFAOYSA-N 0.000 claims 1
- 230000014509 gene expression Effects 0.000 claims 1
- 125000001072 heteroaryl group Chemical group 0.000 claims 1
- UFHFLCQGNIYNRP-UHFFFAOYSA-N hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims 1
- 239000003446 ligand Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- JCFQSYXHEYSBQT-UHFFFAOYSA-N methylaminomethanediol Chemical compound CNC(O)O JCFQSYXHEYSBQT-UHFFFAOYSA-N 0.000 claims 1
- FYFFGSSZFBZTAH-UHFFFAOYSA-N methylaminomethanetriol Chemical compound CNC(O)(O)O FYFFGSSZFBZTAH-UHFFFAOYSA-N 0.000 claims 1
- IZXGZAJMDLJLMF-UHFFFAOYSA-N methylaminomethanol Chemical compound CNCO IZXGZAJMDLJLMF-UHFFFAOYSA-N 0.000 claims 1
- 125000004430 oxygen atoms Chemical group O* 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 239000011780 sodium chloride Substances 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 230000001629 suppression Effects 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical group [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
- WQZGKKKJIJFFOK-PHYPRBDBSA-N α-D-galactose Chemical group OC[C@H]1O[C@H](O)[C@H](O)[C@@H](O)[C@H]1O WQZGKKKJIJFFOK-PHYPRBDBSA-N 0.000 claims 1
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CN201510364229 | 2015-06-26 | ||
CN201510364229.8 | 2015-06-26 | ||
PCT/CN2016/087034 WO2016206626A1 (zh) | 2015-06-26 | 2016-06-24 | 一种siRNA、含有该siRNA的药物组合物和缀合物及它们的应用 |
Publications (3)
Publication Number | Publication Date |
---|---|
JP2018518201A JP2018518201A (ja) | 2018-07-12 |
JP2018518201A5 true JP2018518201A5 (US20040106767A1-20040603-C00005.png) | 2019-06-13 |
JP6715325B2 JP6715325B2 (ja) | 2020-07-01 |
Family
ID=57584730
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
JP2018518771A Active JP6715325B2 (ja) | 2015-06-26 | 2016-06-24 | siRNA、siRNAを含む医薬組成物及び結合体、並びにそれらの応用 |
Country Status (5)
Families Citing this family (27)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3675826B1 (en) * | 2017-08-31 | 2023-05-31 | Life Technologies Corporation | Cationic lipid compositions for tissue-specific delivery |
WO2019105403A1 (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 |
EP3719126A4 (en) * | 2017-12-01 | 2021-10-20 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING NUCLEIC ACID, ASSOCIATED PREPARATION PROCESS AND USE |
EP3719128A4 (en) * | 2017-12-01 | 2021-10-27 | Suzhou Ribo Life Science Co., Ltd. | DOUBLE STRANDED OLIGONUCLEOTIDE, COMPOSITION AND CONJUGATE WITH DOUBLE STRANDED OLIGONUCLEOTIDE, METHOD OF MANUFACTURING THEREOF AND USE THEREOF |
US20220395526A1 (en) * | 2017-12-01 | 2022-12-15 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing same, and preparation method and use |
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 |
WO2019105404A1 (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 |
CN110944675B (zh) * | 2017-12-01 | 2024-06-04 | 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 |
CN116375774A (zh) | 2017-12-29 | 2023-07-04 | 苏州瑞博生物技术股份有限公司 | 缀合物及其制备方法和用途 |
EP3842534A4 (en) * | 2018-08-21 | 2022-07-06 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING NUCLEIC ACID AND METHOD OF USE THEREOF |
WO2020063198A1 (zh) * | 2018-09-30 | 2020-04-02 | 苏州瑞博生物技术有限公司 | 一种siRNA缀合物及其制备方法和用途 |
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 |
CN111377985B (zh) * | 2018-12-29 | 2023-11-10 | 苏州瑞博生物技术股份有限公司 | 化合物和缀合物及其制备方法和用途 |
WO2020147847A1 (zh) * | 2019-01-18 | 2020-07-23 | 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 |
CN111979237A (zh) * | 2019-05-22 | 2020-11-24 | 苏州瑞博生物技术股份有限公司 | 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途 |
CA3139195A1 (en) * | 2019-05-22 | 2020-11-26 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
CN111973618B (zh) * | 2019-05-23 | 2024-02-02 | 苏州瑞博生物技术股份有限公司 | 核酸、药物组合物与siRNA缀合物及制备方法和用途 |
CN111973617A (zh) * | 2019-05-23 | 2020-11-24 | 苏州瑞博生物技术股份有限公司 | 核酸、药物组合物与缀合物及制备方法和用途 |
JP2022546055A (ja) * | 2019-08-29 | 2022-11-02 | 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 | 化合物、薬物複合体及びその調製方法と使用 |
US20230220386A1 (en) * | 2019-12-06 | 2023-07-13 | Medshine Discovery Inc. | Sirna conjugate, double-stranded sirna conjugate, salt thereof and application thereof |
CN111110866B (zh) * | 2019-12-30 | 2023-04-11 | 福州大学 | 还原性聚谷氨酸/聚乙烯亚胺/siRNA复合纳米粒及制备与应用 |
CN115702006A (zh) * | 2020-06-10 | 2023-02-14 | 南京明德新药研发有限公司 | 缀合基团及其缀合物 |
CN113663089B (zh) * | 2021-06-25 | 2022-10-28 | 北京理工大学 | 可电离脂质纳米颗粒组合物、制备方法及应用 |
WO2023116764A1 (zh) * | 2021-12-23 | 2023-06-29 | 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其用途 |
CN114634984A (zh) * | 2022-02-02 | 2022-06-17 | 复旦大学 | 一种脑胶质瘤生物标记物mlkl基因及其应用 |
CN115487308A (zh) * | 2022-09-29 | 2022-12-20 | 北京大学 | 反义核酸糖基缀合物及其制备方法和在肝癌治疗中的应用 |
WO2024124218A1 (en) * | 2022-12-08 | 2024-06-13 | 1 Globe Health Institute Llc | Antisense rna (asrna) technology and use thereof |
Family Cites Families (12)
Publication number | Priority date | Publication date | Assignee | Title |
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AU2005222902B2 (en) * | 2004-03-12 | 2010-06-10 | Alnylam Pharmaceuticals, Inc. | iRNA agents targeting VEGF |
KR101147147B1 (ko) * | 2004-04-01 | 2012-05-25 | 머크 샤프 앤드 돔 코포레이션 | Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드 |
KR101012595B1 (ko) * | 2005-03-09 | 2011-02-07 | 재단법인 목암생명공학연구소 | 작은 간섭 rna 및 이를 포함하는 b형 간염 바이러스 치료용 약학 조성물 |
CN101426912A (zh) * | 2005-08-17 | 2009-05-06 | 瑟纳治疗公司 | 介导rna干扰的化学修饰短干扰核酸分子 |
CN102140461B (zh) * | 2010-01-29 | 2012-12-05 | 苏州瑞博生物技术有限公司 | 小干扰核酸和药物组合物及其制药应用 |
CN102140458B (zh) * | 2010-01-29 | 2013-05-22 | 苏州瑞博生物技术有限公司 | 小干扰核酸和药物组合物及其制药应用 |
DK2640700T3 (en) * | 2010-11-15 | 2019-01-14 | Life Technologies Corp | AMINOUS TRANSFECTION REAGENTS AND METHODS FOR PREPARING USE THEREOF |
WO2013061295A1 (en) * | 2011-10-28 | 2013-05-02 | University Of The Witwatersrand, Johannesburg | Inhibition of viral gene expression |
SI3301177T1 (sl) * | 2011-11-18 | 2020-07-31 | Alnylam Pharmaceuticals, Inc. | Sredstva RNAi, sestavki in postopki njihove uporabe za zdravljenje s transtiretinom (TTR) povezanih bolezni |
JP2015525797A (ja) * | 2012-08-06 | 2015-09-07 | 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 | 糖質コンジュゲートrna剤およびその調製方法 |
CN105189541A (zh) * | 2012-12-14 | 2015-12-23 | 戴瑟纳制药公司 | 用于通过双链rna特异性抑制ckap5的方法和组合物 |
CN114015692A (zh) * | 2013-03-14 | 2022-02-08 | 阿尔尼拉姆医药品有限公司 | 补体组分C5 iRNA组合物及其使用方法 |
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2016
- 2016-06-24 CN CN201680036513.5A patent/CN107849567A/zh active Pending
- 2016-06-24 EP EP16813742.0A patent/EP3315608B1/en active Active
- 2016-06-24 WO PCT/CN2016/087034 patent/WO2016206626A1/zh active Application Filing
- 2016-06-24 JP JP2018518771A patent/JP6715325B2/ja active Active
- 2016-06-24 US US15/738,006 patent/US20190062749A1/en not_active Abandoned