JP2020533334A5 - - Google Patents
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- JP2020533334A5 JP2020533334A5 JP2020514257A JP2020514257A JP2020533334A5 JP 2020533334 A5 JP2020533334 A5 JP 2020533334A5 JP 2020514257 A JP2020514257 A JP 2020514257A JP 2020514257 A JP2020514257 A JP 2020514257A JP 2020533334 A5 JP2020533334 A5 JP 2020533334A5
- Authority
- JP
- Japan
- Prior art keywords
- rnai agent
- agent according
- nucleotides
- composition
- sense 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.)
- Granted
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- 108091030071 RNAI Proteins 0.000 claims description 106
- 239000003795 chemical substances by application Substances 0.000 claims description 106
- 230000009368 gene silencing by RNA Effects 0.000 claims description 106
- 125000003729 nucleotide group Chemical group 0.000 claims description 89
- 239000002773 nucleotide Substances 0.000 claims description 75
- 239000000203 mixture Substances 0.000 claims description 47
- 108091081021 Sense strand Proteins 0.000 claims description 46
- 230000000692 anti-sense effect Effects 0.000 claims description 36
- 239000003446 ligand Substances 0.000 claims description 32
- 230000008685 targeting Effects 0.000 claims description 32
- 238000000034 method Methods 0.000 claims description 24
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 23
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims description 16
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims description 16
- 201000010099 disease Diseases 0.000 claims description 15
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol 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 description 14
- 230000014509 gene expression Effects 0.000 claims description 14
- 101150001527 APOC3 gene Proteins 0.000 claims description 12
- 208000006575 hypertriglyceridemia Diseases 0.000 claims description 10
- UHDGCWIWMRVCDJ-UHFFFAOYSA-N 1-beta-D-Xylofuranosyl-NH-Cytosine Natural products O=C1N=C(N)C=CN1C1C(O)C(O)C(CO)O1 UHDGCWIWMRVCDJ-UHFFFAOYSA-N 0.000 claims description 8
- MIKUYHXYGGJMLM-GIMIYPNGSA-N Crotonoside Natural products C1=NC2=C(N)NC(=O)N=C2N1[C@H]1O[C@@H](CO)[C@H](O)[C@@H]1O MIKUYHXYGGJMLM-GIMIYPNGSA-N 0.000 claims description 8
- UHDGCWIWMRVCDJ-PSQAKQOGSA-N Cytidine Natural products O=C1N=C(N)C=CN1[C@@H]1[C@@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-PSQAKQOGSA-N 0.000 claims description 8
- NYHBQMYGNKIUIF-UHFFFAOYSA-N D-guanosine Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(O)C1O NYHBQMYGNKIUIF-UHFFFAOYSA-N 0.000 claims description 8
- 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 description 8
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims description 8
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 claims description 8
- FPUGCISOLXNPPC-IOSLPCCCSA-N cordysinin B Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(N)=C2N=C1 FPUGCISOLXNPPC-IOSLPCCCSA-N 0.000 claims description 8
- UHDGCWIWMRVCDJ-ZAKLUEHWSA-N cytidine Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@@H](O)[C@H](CO)O1 UHDGCWIWMRVCDJ-ZAKLUEHWSA-N 0.000 claims description 8
- 208000035475 disorder Diseases 0.000 claims description 8
- 229940029575 guanosine Drugs 0.000 claims description 8
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims description 8
- 229940045145 uridine Drugs 0.000 claims description 8
- 229930010555 Inosine Natural products 0.000 claims description 6
- 230000002401 inhibitory effect Effects 0.000 claims description 6
- 229960003786 inosine Drugs 0.000 claims description 6
- 230000001404 mediated effect Effects 0.000 claims description 6
- 208000024891 symptom Diseases 0.000 claims description 6
- 201000001320 Atherosclerosis Diseases 0.000 claims description 5
- 208000024172 Cardiovascular disease Diseases 0.000 claims description 5
- 208000031226 Hyperlipidaemia Diseases 0.000 claims description 5
- 208000001145 Metabolic Syndrome Diseases 0.000 claims description 5
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 5
- 208000008589 Obesity Diseases 0.000 claims description 5
- 206010033645 Pancreatitis Diseases 0.000 claims description 5
- 201000000690 abdominal obesity-metabolic syndrome Diseases 0.000 claims description 5
- 230000002159 abnormal effect Effects 0.000 claims description 5
- 208000029078 coronary artery disease Diseases 0.000 claims description 5
- 206010012601 diabetes mellitus Diseases 0.000 claims description 5
- 150000002632 lipids Chemical class 0.000 claims description 5
- 230000004060 metabolic process Effects 0.000 claims description 5
- 235000020824 obesity Nutrition 0.000 claims description 5
- 208000011580 syndromic disease Diseases 0.000 claims description 5
- UUDVSZSQPFXQQM-GIWSHQQXSA-N (2r,3s,4r,5r)-2-(6-aminopurin-9-yl)-3-fluoro-5-(hydroxymethyl)oxolane-3,4-diol Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@]1(O)F UUDVSZSQPFXQQM-GIWSHQQXSA-N 0.000 claims description 4
- FPUGCISOLXNPPC-UHFFFAOYSA-N 2'-O-Methyladenosine Natural products COC1C(O)C(CO)OC1N1C2=NC=NC(N)=C2N=C1 FPUGCISOLXNPPC-UHFFFAOYSA-N 0.000 claims description 4
- BXJSAMKIFDDLGI-UHFFFAOYSA-N 2-(dimethylamino)-N-[2-[3-[[5-[3-(dimethylcarbamoyl)phenyl]-2-methoxyphenyl]sulfonylamino]anilino]ethyl]benzamide Chemical compound COc1ccc(cc1S(=O)(=O)Nc1cccc(NCCNC(=O)c2ccccc2N(C)C)c1)-c1cccc(c1)C(=O)N(C)C BXJSAMKIFDDLGI-UHFFFAOYSA-N 0.000 claims description 4
- 102100030970 Apolipoprotein C-III Human genes 0.000 claims description 4
- 102000005427 Asialoglycoprotein Receptor Human genes 0.000 claims description 4
- 101000793223 Homo sapiens Apolipoprotein C-III Proteins 0.000 claims description 4
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims description 4
- 108010007622 LDL Lipoproteins Proteins 0.000 claims description 4
- 102000007330 LDL Lipoproteins Human genes 0.000 claims description 4
- 108010006523 asialoglycoprotein receptor Proteins 0.000 claims description 4
- 230000037396 body weight Effects 0.000 claims description 4
- 239000005547 deoxyribonucleotide Substances 0.000 claims description 4
- 239000003814 drug Substances 0.000 claims description 4
- RHLMXWCISNJNDH-UHFFFAOYSA-N n-[2-[3-[[5-[3-(dimethylcarbamoyl)phenyl]-2-methoxyphenyl]sulfonylamino]anilino]ethyl]-3-methylbenzamide Chemical compound COC1=CC=C(C=2C=C(C=CC=2)C(=O)N(C)C)C=C1S(=O)(=O)NC(C=1)=CC=CC=1NCCNC(=O)C1=CC=CC(C)=C1 RHLMXWCISNJNDH-UHFFFAOYSA-N 0.000 claims description 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 4
- 206010014476 Elevated cholesterol Diseases 0.000 claims description 2
- 206010014486 Elevated triglycerides Diseases 0.000 claims description 2
- 208000035150 Hypercholesterolemia Diseases 0.000 claims description 2
- JVWLUVNSQYXYBE-UHFFFAOYSA-N Ribitol Natural products OCC(C)C(O)C(O)CO JVWLUVNSQYXYBE-UHFFFAOYSA-N 0.000 claims description 2
- 239000002253 acid Substances 0.000 claims description 2
- 239000002214 arabinonucleotide Substances 0.000 claims description 2
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims description 2
- 208000016097 disease of metabolism Diseases 0.000 claims description 2
- 230000002526 effect on cardiovascular system Effects 0.000 claims description 2
- 125000001921 locked nucleotide group Chemical group 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- HEBKCHPVOIAQTA-UHFFFAOYSA-N meso ribitol Natural products OCC(O)C(O)C(O)CO HEBKCHPVOIAQTA-UHFFFAOYSA-N 0.000 claims description 2
- 208000030159 metabolic disease Diseases 0.000 claims description 2
- 239000002777 nucleoside Substances 0.000 claims description 2
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- HEBKCHPVOIAQTA-ZXFHETKHSA-N ribitol Chemical compound OC[C@H](O)[C@H](O)[C@H](O)CO HEBKCHPVOIAQTA-ZXFHETKHSA-N 0.000 claims description 2
- 238000010254 subcutaneous injection Methods 0.000 claims description 2
- 239000007929 subcutaneous injection Substances 0.000 claims description 2
- 229940124597 therapeutic agent Drugs 0.000 claims description 2
- UFTFJSFQGQCHQW-UHFFFAOYSA-N triformin Chemical compound O=COCC(OC=O)COC=O UFTFJSFQGQCHQW-UHFFFAOYSA-N 0.000 claims description 2
- 108020004635 Complementary DNA Proteins 0.000 claims 1
- 230000000295 complement effect Effects 0.000 description 1
- 239000000463 material Substances 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023051640A JP2023073358A (ja) | 2017-09-11 | 2023-03-28 | アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762556818P | 2017-09-11 | 2017-09-11 | |
| US62/556,818 | 2017-09-11 | ||
| US201862643927P | 2018-03-16 | 2018-03-16 | |
| US62/643,927 | 2018-03-16 | ||
| US201862720434P | 2018-08-21 | 2018-08-21 | |
| US62/720,434 | 2018-08-21 | ||
| PCT/US2018/050248 WO2019051402A1 (en) | 2017-09-11 | 2018-09-10 | RNA INTERFERENCE AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3) |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023051640A Division JP2023073358A (ja) | 2017-09-11 | 2023-03-28 | アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020533334A JP2020533334A (ja) | 2020-11-19 |
| JP2020533334A5 true JP2020533334A5 (enExample) | 2021-10-07 |
| JP7438103B2 JP7438103B2 (ja) | 2024-02-26 |
Family
ID=65630632
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020514257A Active JP7438103B2 (ja) | 2017-09-11 | 2018-09-10 | アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物 |
| JP2023051640A Pending JP2023073358A (ja) | 2017-09-11 | 2023-03-28 | アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023051640A Pending JP2023073358A (ja) | 2017-09-11 | 2023-03-28 | アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物 |
Country Status (24)
| Country | Link |
|---|---|
| US (4) | US10597657B2 (enExample) |
| EP (1) | EP3681516A4 (enExample) |
| JP (2) | JP7438103B2 (enExample) |
| KR (2) | KR20240161202A (enExample) |
| CN (2) | CN111107853B (enExample) |
| AU (1) | AU2018329190B2 (enExample) |
| BR (1) | BR112020002413A2 (enExample) |
| CA (1) | CA3074303A1 (enExample) |
| CL (2) | CL2020000593A1 (enExample) |
| CO (1) | CO2020002586A2 (enExample) |
| CR (1) | CR20200108A (enExample) |
| EC (1) | ECSP20017905A (enExample) |
| IL (2) | IL273172B1 (enExample) |
| JO (1) | JOP20200054A1 (enExample) |
| MX (1) | MX2020002648A (enExample) |
| MY (1) | MY202025A (enExample) |
| PE (1) | PE20201283A1 (enExample) |
| PH (1) | PH12020500332A1 (enExample) |
| SG (1) | SG11201912178VA (enExample) |
| TN (1) | TN2020000039A1 (enExample) |
| TW (2) | TWI811238B (enExample) |
| UY (1) | UY37871A (enExample) |
| WO (1) | WO2019051402A1 (enExample) |
| ZA (1) | ZA202100806B (enExample) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CN116942841A (zh) * | 2016-09-02 | 2023-10-27 | 箭头药业股份有限公司 | 靶向配体 |
| CN110520531A (zh) | 2017-04-05 | 2019-11-29 | 赛伦斯治疗有限责任公司 | 产品和组合物 |
| SG11201912178VA (en) * | 2017-09-11 | 2020-01-30 | Arrowhead Pharmaceuticals Inc | Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3) |
| CN110997917B (zh) | 2017-12-01 | 2024-04-09 | 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 |
| CN110945130B (zh) | 2017-12-01 | 2024-04-09 | 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 |
| US12496347B2 (en) * | 2018-12-28 | 2025-12-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof |
| CN113227376B (zh) | 2019-05-22 | 2024-04-09 | 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 |
| KR20220044551A (ko) * | 2019-08-05 | 2022-04-08 | 애로우헤드 파마슈티컬스 인코포레이티드 | Apoc3-관련 질환 및 장애의 치료 방법 |
| AU2021224778A1 (en) * | 2020-02-18 | 2022-09-29 | Alnylam Pharmaceuticals, Inc. | Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof |
| CN114480382A (zh) * | 2020-11-12 | 2022-05-13 | 北京大学 | 用于抑制SURF4基因表达的RNAi试剂和组合物 |
| WO2022162155A1 (en) * | 2021-01-30 | 2022-08-04 | E-Therapeutics Plc | Nucleic acids containing abasic nucleotides |
| WO2022272108A2 (en) * | 2021-06-24 | 2022-12-29 | Sirnaomics, Inc. | Products and compositions |
| AU2022309416A1 (en) * | 2021-07-16 | 2024-02-01 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing same, and preparation method and use |
| CA3232053A1 (en) * | 2021-10-08 | 2023-04-13 | E-Therapeutics Plc | Nucleic acids containing abasic nucleosides |
| KR20240111757A (ko) | 2021-12-01 | 2024-07-17 | 다이서나 파마수이티컬, 인크. | Apoc3 발현을 조절하기 위한 조성물 및 방법 |
| MX2024008804A (es) * | 2022-01-20 | 2024-07-25 | Tuojie Biotech Shanghai Co Ltd | Arnbc, uso y metodo de preparacion para el mismo. |
| TW202345865A (zh) * | 2022-01-24 | 2023-12-01 | 大陸商上海舶望製藥有限公司 | 抑制LPA(Apo(a))蛋白表達的組合物和方法 |
| AU2023211407A1 (en) | 2022-01-30 | 2024-08-15 | Rona Bioscience, Limited | Targeting ligand containing n-acetylgalactosamine |
| EP4541893A1 (en) | 2022-06-14 | 2025-04-23 | Rona Bioscience, Limited | Sirna molecule for regulating pcsk9 gene activity |
| EP4545641A1 (en) | 2022-06-27 | 2025-04-30 | Rona Bioscience, Limited | Sirna for inhibiting apolipoprotein c3 expression |
| AU2023307152A1 (en) * | 2022-07-11 | 2025-01-09 | Sanegene Bio Usa Inc. | Optimized 2'- modified ribose derivatives and methods of use |
| US20250376680A1 (en) * | 2022-07-27 | 2025-12-11 | E-Therapeutics Plc | Nucleic acid compounds |
| KR20250043472A (ko) * | 2022-07-27 | 2025-03-28 | 이-테라퓨틱스 피엘씨 | 핵산 화합물 |
| JP2025524135A (ja) * | 2022-07-27 | 2025-07-25 | イー－セラピューティクス・ピーエルシー | 核酸化合物 |
| CN116814621B (zh) * | 2022-08-05 | 2025-09-09 | 厦门甘宝利生物医药有限公司 | 一种抑制apoc3基因表达的rna抑制剂及其应用 |
| WO2024032680A1 (zh) * | 2022-08-11 | 2024-02-15 | 益杰立科(上海)生物科技有限公司 | 一种表观编辑靶点的方法及用途 |
| WO2024165571A2 (en) * | 2023-02-06 | 2024-08-15 | E-Therapeutics Plc | Inhibitors of expression and/or function |
| AU2024218976A1 (en) * | 2023-02-06 | 2025-09-11 | E-Therapeutics Plc | Inhibitors of expression and/or function |
| WO2024188173A1 (zh) * | 2023-03-10 | 2024-09-19 | 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 |
| 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 |
| WO2025087442A1 (zh) * | 2023-10-27 | 2025-05-01 | 北京安龙生物医药有限公司 | 靶向脂蛋白a的寡核苷酸及其用途 |
| WO2025125576A2 (en) * | 2023-12-15 | 2025-06-19 | E-Therapeutics Plc | Inhibitors of expression and/or function |
| WO2025133348A1 (en) * | 2023-12-22 | 2025-06-26 | E-Therapeutics Plc | Inhibitors of expression and/or function |
| WO2025184301A1 (en) * | 2024-02-29 | 2025-09-04 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for dual inhibition of expression of apolipoprotein c-iii (apoc3) and proprotein convertase subtilisin kexin 9 (pcsk9), compositions thereof, and methods of use |
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