JP2018512155A5 - - Google Patents
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- JP2018512155A5 JP2018512155A5 JP2017553347A JP2017553347A JP2018512155A5 JP 2018512155 A5 JP2018512155 A5 JP 2018512155A5 JP 2017553347 A JP2017553347 A JP 2017553347A JP 2017553347 A JP2017553347 A JP 2017553347A JP 2018512155 A5 JP2018512155 A5 JP 2018512155A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nucleotide
- dsrna
- nucleotides
- modified
- 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.)
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- 125000003729 nucleotide group Chemical group 0.000 claims 43
- 239000002773 nucleotide Substances 0.000 claims 32
- 101000693085 Homo sapiens Angiopoietin-related protein 3 Proteins 0.000 claims 12
- 230000000692 anti-sense effect Effects 0.000 claims 12
- 102100025668 Angiopoietin-related protein 3 Human genes 0.000 claims 9
- 108091081021 Sense strand Proteins 0.000 claims 8
- 238000012986 modification Methods 0.000 claims 7
- 230000004048 modification Effects 0.000 claims 7
- 230000002401 inhibitory effect Effects 0.000 claims 6
- 239000003446 ligand Substances 0.000 claims 6
- 229920002477 rna polymer Polymers 0.000 claims 4
- 229910019142 PO4 Inorganic materials 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 239000010452 phosphate Substances 0.000 claims 3
- 208000035762 Disorder of lipid metabolism Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 208000035475 disorder Diseases 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- MPCAJMNYNOGXPB-UHFFFAOYSA-N 1,5-Anhydro-mannit Natural products OCC1OCC(O)C(O)C1O MPCAJMNYNOGXPB-UHFFFAOYSA-N 0.000 claims 1
- UEUPTUCWIHOIMK-UHFFFAOYSA-N 2'-deoxy-AMP Natural products C1=NC=2C(N)=NC=NC=2N1C1CC(OP(O)(O)=O)C(CO)O1 UEUPTUCWIHOIMK-UHFFFAOYSA-N 0.000 claims 1
- UEUPTUCWIHOIMK-RRKCRQDMSA-N 2'-deoxyadenosine 3'-monophosphate Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@H]1C[C@H](OP(O)(O)=O)[C@@H](CO)O1 UEUPTUCWIHOIMK-RRKCRQDMSA-N 0.000 claims 1
- QQMSZHORHNORLP-KVQBGUIXSA-N 2'-deoxyguanosine 3'-monophosphate Chemical compound C1=NC=2C(=O)NC(N)=NC=2N1[C@H]1C[C@H](OP(O)(O)=O)[C@@H](CO)O1 QQMSZHORHNORLP-KVQBGUIXSA-N 0.000 claims 1
- QQMSZHORHNORLP-UHFFFAOYSA-N 2'-deoxyguanosine 3'-monophosphate Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1CC(OP(O)(O)=O)C(CO)O1 QQMSZHORHNORLP-UHFFFAOYSA-N 0.000 claims 1
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- KBDWGFZSICOZSJ-UHFFFAOYSA-N 5-methyl-2,3-dihydro-1H-pyrimidin-4-one Chemical group N1CNC=C(C1=O)C KBDWGFZSICOZSJ-UHFFFAOYSA-N 0.000 claims 1
- 208000035657 Abasia Diseases 0.000 claims 1
- 208000031226 Hyperlipidaemia Diseases 0.000 claims 1
- 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 1
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical class CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims 1
- 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 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 1
- 108091030071 RNAI Proteins 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical class OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 1
- 238000009825 accumulation Methods 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 239000003795 chemical substances by application Substances 0.000 claims 1
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol group Chemical group [C@@H]1(CC[C@H]2[C@@H]3CC=C4C[C@@H](O)CC[C@]4(C)[C@H]3CC[C@]12C)[C@H](C)CCCC(C)C HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 1
- 238000003776 cleavage reaction Methods 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 230000008878 coupling Effects 0.000 claims 1
- 238000010168 coupling process Methods 0.000 claims 1
- 238000005859 coupling reaction Methods 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 238000010586 diagram Methods 0.000 claims 1
- BNKAXGCRDYRABM-UHFFFAOYSA-N ethenyl dihydrogen phosphate Chemical compound OP(O)(=O)OC=C BNKAXGCRDYRABM-UHFFFAOYSA-N 0.000 claims 1
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 125000001153 fluoro group Chemical group F* 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 208000006575 hypertriglyceridemia Diseases 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 125000001921 locked nucleotide group Chemical group 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 1
- 230000003278 mimic effect Effects 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- ONKSSDKXDIVIHK-UHFFFAOYSA-N n,n-didecyldodecanamide Chemical group CCCCCCCCCCCC(=O)N(CCCCCCCCCC)CCCCCCCCCC ONKSSDKXDIVIHK-UHFFFAOYSA-N 0.000 claims 1
- PTMHPRAIXMAOOB-UHFFFAOYSA-N phosphoramidic acid Chemical compound NP(O)(O)=O PTMHPRAIXMAOOB-UHFFFAOYSA-N 0.000 claims 1
- 230000007017 scission Effects 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
- -1 thymidine-glycol nucleic acid Chemical class 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021147698A JP7817806B2 (ja) | 2015-04-13 | 2021-09-10 | アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法 |
| JP2023169168A JP2024009838A (ja) | 2015-04-13 | 2023-09-29 | アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562146604P | 2015-04-13 | 2015-04-13 | |
| US62/146,604 | 2015-04-13 | ||
| US201562261361P | 2015-12-01 | 2015-12-01 | |
| US62/261,361 | 2015-12-01 | ||
| PCT/US2016/027271 WO2016168286A1 (en) | 2015-04-13 | 2016-04-13 | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021147698A Division JP7817806B2 (ja) | 2015-04-13 | 2021-09-10 | アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018512155A JP2018512155A (ja) | 2018-05-17 |
| JP2018512155A5 true JP2018512155A5 (enExample) | 2019-05-23 |
| JP6975641B2 JP6975641B2 (ja) | 2021-12-01 |
Family
ID=55806850
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017553347A Active JP6975641B2 (ja) | 2015-04-13 | 2016-04-13 | アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法 |
| JP2021147698A Active JP7817806B2 (ja) | 2015-04-13 | 2021-09-10 | アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法 |
| JP2023169168A Pending JP2024009838A (ja) | 2015-04-13 | 2023-09-29 | アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021147698A Active JP7817806B2 (ja) | 2015-04-13 | 2021-09-10 | アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法 |
| JP2023169168A Pending JP2024009838A (ja) | 2015-04-13 | 2023-09-29 | アンギオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびそれらの使用方法 |
Country Status (13)
| Country | Link |
|---|---|
| US (4) | US10570393B2 (enExample) |
| EP (1) | EP3283631A1 (enExample) |
| JP (3) | JP6975641B2 (enExample) |
| KR (2) | KR20250005546A (enExample) |
| CN (2) | CN107743522B (enExample) |
| AU (2) | AU2016247922B2 (enExample) |
| BR (1) | BR112017021053A2 (enExample) |
| CA (1) | CA2982450A1 (enExample) |
| EA (1) | EA201792263A1 (enExample) |
| IL (3) | IL325194A (enExample) |
| MX (2) | MX392757B (enExample) |
| TW (3) | TWI723986B (enExample) |
| WO (1) | WO2016168286A1 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3444348A1 (en) | 2011-06-21 | 2019-02-20 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
| KR20250005546A (ko) | 2015-04-13 | 2025-01-09 | 알닐람 파마슈티칼스 인코포레이티드 | 안지오포이에틴-유사 3(angptl3) irna 조성물 및 이의 이용 방법 |
| PE20201287A1 (es) * | 2017-09-14 | 2020-11-24 | Arrowhead Pharmaceuticals Inc | Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso |
| CN118236391A (zh) | 2017-12-01 | 2024-06-25 | 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 |
| US11660347B2 (en) | 2017-12-01 | 2023-05-30 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing same, preparation method, and use thereof |
| JP7261494B2 (ja) | 2017-12-01 | 2023-04-20 | 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 |
| WO2019105435A1 (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 |
| CN110997919B (zh) | 2017-12-01 | 2024-04-02 | 苏州瑞博生物技术股份有限公司 | 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途 |
| CN110959011B (zh) * | 2017-12-29 | 2023-03-28 | 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 |
| TWI851574B (zh) * | 2018-05-14 | 2024-08-11 | 美商阿尼拉製藥公司 | 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法 |
| EP3842534A4 (en) | 2018-08-21 | 2022-07-06 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND CONJUGATE WITH NUCLEIC ACID AND THEIR USE |
| EP3862024A4 (en) | 2018-09-30 | 2022-08-17 | Suzhou Ribo Life Science Co., Ltd. | SIRNA CONJUGATE, METHOD FOR ITS PRODUCTION AND ITS USE |
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| CN112423794A (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 |
| JP7663951B2 (ja) | 2019-05-22 | 2025-04-17 | 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 |
| AU2020280439B2 (en) | 2019-05-22 | 2025-08-14 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| CN113811613B (zh) | 2019-05-22 | 2024-04-05 | 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 |
| WO2020238766A1 (zh) | 2019-05-24 | 2020-12-03 | 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 |
| AU2020399633A1 (en) * | 2019-12-09 | 2022-07-07 | Empirico Inc. | Oligonucleotides for treatment of Angiopoietin like 4 (ANGPTL4) related diseases |
| GB2627644B (en) * | 2020-03-04 | 2024-11-20 | Verve Therapeutics Inc | Method for the preparation of GalNAc-lipid nanoparticle |
| CA3174872A1 (en) * | 2020-04-07 | 2021-10-14 | Sander Jan Hendrik Van Deventer | Gene constructs for silencing angiopoietin-like 3 (angptl3) and uses thereof |
| CN116333013B (zh) * | 2020-09-30 | 2025-09-02 | 纳肽得(青岛)生物医药有限公司 | 一种靶向配体 |
| EP4229200A1 (en) * | 2020-10-16 | 2023-08-23 | Sanofi | Novel rna compositions and methods for inhibiting angptl3 |
| CN114685585B (zh) * | 2020-12-31 | 2024-06-04 | 苏州瑞博生物技术股份有限公司 | 核苷酸序列、双链寡核苷酸、药物组合物与缀合物及制备方法和用途 |
| AU2022231003A1 (en) * | 2021-03-04 | 2023-09-14 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
| JP2024511473A (ja) * | 2021-03-24 | 2024-03-13 | アタランタ セラピューティクス，インコーポレーテッド | パターン化化学修飾を有する二本鎖ｓｉｒｎａ |
| EP4380576A4 (en) | 2021-08-03 | 2026-04-29 | Verve Therapeutics Inc | RNA TARGETED COMPOSITIONS AND ADMINISTRATION METHODS |
| US20250136978A1 (en) * | 2021-09-17 | 2025-05-01 | Alnylam Pharmaceuticals, Inc. | iRNA Compositions and Methods for Silencing ANGPTL4 |
| WO2023045994A1 (en) * | 2021-09-23 | 2023-03-30 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of angiopoietin-like 3 (angptl3) protein |
| WO2023092102A1 (en) * | 2021-11-19 | 2023-05-25 | Sanegene Bio Usa Inc. | Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof |
| CA3241896A1 (en) | 2021-12-22 | 2023-06-29 | Regeneron Pharmaceuticals, Inc. | Treatment of kidney diseases with angiopoietin like 3 (angptl3) inhibitors |
| WO2023134705A1 (zh) * | 2022-01-11 | 2023-07-20 | 上海金中锘美生物医药科技有限公司 | 抑制angptl3表达的rna干扰剂及其用途 |
| CN116987696A (zh) * | 2022-08-04 | 2023-11-03 | 北京福元医药股份有限公司 | 用于抑制angptl3基因表达的双链核糖核酸及其修饰物、缀合物和用途 |
| CN119585431A (zh) * | 2022-08-08 | 2025-03-07 | 大睿生物医药科技(上海)有限公司 | 调控ANGPTL3基因活性的siRNA分子 |
| WO2024102913A1 (en) | 2022-11-10 | 2024-05-16 | Regeneron Pharmaceuticals, Inc. | Treatment of kidney diseases with a combination of angiopoietin like 3 (angptl3) inhibitors and solute carrier family 5 member 2 (slc5a2) inhibitors |
| WO2024227059A2 (en) * | 2023-04-27 | 2024-10-31 | Aligos Therapeutics, Inc. | Short interfering nucleic acid (sina) molecules targeting angptl 3 and angptl 8 and methods of using the same |
| TW202530409A (zh) * | 2023-10-07 | 2025-08-01 | 大陸商齊魯製藥有限公司 | ｉＲＮＡ組合物及其使用方法 |
| WO2025169133A1 (en) | 2024-02-09 | 2025-08-14 | Astrazeneca Ab | Double-stranded rnai agents and compositions for reducing expression of angiopoietin-like 3 (angptl3) and methods of use thereof |
| WO2025201277A1 (en) * | 2024-03-25 | 2025-10-02 | Shanghai Argo Biopharmaceutical Co., Ltd. | Method for treating dyslipidemia with angiopoietin-like 3 (angptl3) -targeted rnai agents |
| WO2025232842A1 (zh) * | 2024-05-09 | 2025-11-13 | 石药集团中奇制药技术（石家庄）有限公司 | 一种抑制angptl3基因表达的核酸分子 |
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