JP2018519797A5 - - Google Patents
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- JP2018519797A5 JP2018519797A5 JP2017557175A JP2017557175A JP2018519797A5 JP 2018519797 A5 JP2018519797 A5 JP 2018519797A5 JP 2017557175 A JP2017557175 A JP 2017557175A JP 2017557175 A JP2017557175 A JP 2017557175A JP 2018519797 A5 JP2018519797 A5 JP 2018519797A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotides
- dsrna agent
- seq
- pharmaceutical composition
- nucleotide
- 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 52
- 239000002773 nucleotide Substances 0.000 claims 44
- 239000003795 chemical substances by application Substances 0.000 claims 41
- 239000008194 pharmaceutical composition Substances 0.000 claims 22
- 230000000692 anti-sense effect Effects 0.000 claims 11
- 206010019860 Hereditary angioedema Diseases 0.000 claims 10
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 10
- 108091081021 Sense strand Proteins 0.000 claims 9
- 108010080865 Factor XII Proteins 0.000 claims 8
- 102000000429 Factor XII Human genes 0.000 claims 8
- 230000002401 inhibitory effect Effects 0.000 claims 7
- 208000007536 Thrombosis Diseases 0.000 claims 6
- 230000004913 activation Effects 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 6
- 239000003446 ligand Substances 0.000 claims 6
- 230000037361 pathway Effects 0.000 claims 6
- 230000015572 biosynthetic process Effects 0.000 claims 5
- 101150072419 F12 gene Proteins 0.000 claims 3
- 208000035475 disorder Diseases 0.000 claims 3
- 230000009467 reduction Effects 0.000 claims 3
- 229920002477 rna polymer Polymers 0.000 claims 3
- 208000024891 symptom Diseases 0.000 claims 3
- MPCAJMNYNOGXPB-UHFFFAOYSA-N 1,5-anhydrohexitol Chemical compound OCC1OCC(O)C(O)C1O MPCAJMNYNOGXPB-UHFFFAOYSA-N 0.000 claims 2
- 208000028185 Angioedema Diseases 0.000 claims 2
- 101800004538 Bradykinin Proteins 0.000 claims 2
- 208000007530 Essential hypertension Diseases 0.000 claims 2
- QXZGBUJJYSLZLT-UHFFFAOYSA-N H-Arg-Pro-Pro-Gly-Phe-Ser-Pro-Phe-Arg-OH Natural products NC(N)=NCCCC(N)C(=O)N1CCCC1C(=O)N1C(C(=O)NCC(=O)NC(CC=2C=CC=CC=2)C(=O)NC(CO)C(=O)N2C(CCC2)C(=O)NC(CC=2C=CC=CC=2)C(=O)NC(CCCN=C(N)N)C(O)=O)CCC1 QXZGBUJJYSLZLT-UHFFFAOYSA-N 0.000 claims 2
- 102100035792 Kininogen-1 Human genes 0.000 claims 2
- 108700018490 Prekallikrein Deficiency Proteins 0.000 claims 2
- 230000008901 benefit Effects 0.000 claims 2
- QXZGBUJJYSLZLT-FDISYFBBSA-N bradykinin Chemical compound NC(=N)NCCC[C@H](N)C(=O)N1CCC[C@H]1C(=O)N1[C@H](C(=O)NCC(=O)N[C@@H](CC=2C=CC=CC=2)C(=O)N[C@@H](CO)C(=O)N2[C@@H](CCC2)C(=O)N[C@@H](CC=2C=CC=CC=2)C(=O)N[C@@H](CCCNC(N)=N)C(O)=O)CCC1 QXZGBUJJYSLZLT-FDISYFBBSA-N 0.000 claims 2
- ZDPUTNZENXVHJC-UHFFFAOYSA-N cumingianoside D Natural products C1=2NC(N)=NC(=O)C=2N=CN1C1OC(CO)C(OP(O)(O)=O)C1O ZDPUTNZENXVHJC-UHFFFAOYSA-N 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 230000004048 modification Effects 0.000 claims 2
- 238000012986 modification Methods 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- 230000002885 thrombogenetic effect Effects 0.000 claims 2
- 230000001732 thrombotic effect Effects 0.000 claims 2
- 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
- UOOOPKANIPLQPU-UHFFFAOYSA-N Cytidylic acid B Natural products O=C1N=C(N)C=CN1C1C(O)C(OP(O)(O)=O)C(CO)O1 UOOOPKANIPLQPU-UHFFFAOYSA-N 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
- 229910019142 PO4 Inorganic materials 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical group OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 claims 1
- 239000007983 Tris buffer Substances 0.000 claims 1
- NPRNBAPBGVERRF-ZOQUXTDFSA-N [(2r,3r,4r,5r)-5-(2,4-dioxopyrimidin-1-yl)-2-(hydroxymethyl)-4-methoxyoxolan-3-yl] dihydrogen phosphate Chemical compound CO[C@@H]1[C@H](OP(O)(O)=O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 NPRNBAPBGVERRF-ZOQUXTDFSA-N 0.000 claims 1
- VXTNWQOOBHQEDS-IOSLPCCCSA-N [(2r,3r,4r,5r)-5-(6-aminopurin-9-yl)-2-(hydroxymethyl)-4-methoxyoxolan-3-yl] dihydrogen phosphate Chemical compound CO[C@@H]1[C@H](OP(O)(O)=O)[C@@H](CO)O[C@H]1N1C2=NC=NC(N)=C2N=C1 VXTNWQOOBHQEDS-IOSLPCCCSA-N 0.000 claims 1
- PTESHFKYBQMNFR-FJGDRVTGSA-N [(2r,3r,4s,5r)-5-(4-amino-2-oxopyrimidin-1-yl)-4-fluoro-4-hydroxy-2-(hydroxymethyl)oxolan-3-yl] dihydrogen phosphate Chemical compound O=C1N=C(N)C=CN1[C@H]1[C@](F)(O)[C@H](OP(O)(O)=O)[C@@H](CO)O1 PTESHFKYBQMNFR-FJGDRVTGSA-N 0.000 claims 1
- KLVQSNKMLGBSHV-GIWSHQQXSA-N [(2r,3r,4s,5r)-5-(6-aminopurin-9-yl)-4-fluoro-4-hydroxy-2-(hydroxymethyl)oxolan-3-yl] dihydrogen phosphate Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](OP(O)(O)=O)[C@]1(O)F KLVQSNKMLGBSHV-GIWSHQQXSA-N 0.000 claims 1
- 239000000427 antigen Substances 0.000 claims 1
- 108091007433 antigens Proteins 0.000 claims 1
- 102000036639 antigens Human genes 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 230000000694 effects Effects 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
- 239000012634 fragment Substances 0.000 claims 1
- 238000002657 hormone replacement therapy Methods 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- YACKEPLHDIMKIO-UHFFFAOYSA-N methylphosphonic acid Chemical group CP(O)(O)=O YACKEPLHDIMKIO-UHFFFAOYSA-N 0.000 claims 1
- 125000004573 morpholin-4-yl group Chemical group N1(CCOCC1)* 0.000 claims 1
- 239000010452 phosphate Substances 0.000 claims 1
- PTMHPRAIXMAOOB-UHFFFAOYSA-L phosphoramidate Chemical compound NP([O-])([O-])=O PTMHPRAIXMAOOB-UHFFFAOYSA-L 0.000 claims 1
- 230000008439 repair process Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020075777A JP7530204B2 (ja) | 2015-05-06 | 2020-04-22 | 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562157890P | 2015-05-06 | 2015-05-06 | |
| US62/157,890 | 2015-05-06 | ||
| US201562260887P | 2015-11-30 | 2015-11-30 | |
| US62/260,887 | 2015-11-30 | ||
| US201562266958P | 2015-12-14 | 2015-12-14 | |
| US62/266,958 | 2015-12-14 | ||
| PCT/US2016/030876 WO2016179342A2 (en) | 2015-05-06 | 2016-05-05 | Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020075777A Division JP7530204B2 (ja) | 2015-05-06 | 2020-04-22 | 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018519797A JP2018519797A (ja) | 2018-07-26 |
| JP2018519797A5 true JP2018519797A5 (enExample) | 2019-06-06 |
| JP6695902B2 JP6695902B2 (ja) | 2020-05-20 |
Family
ID=55971215
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017557175A Active JP6695902B2 (ja) | 2015-05-06 | 2016-05-05 | 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法 |
| JP2020075777A Active JP7530204B2 (ja) | 2015-05-06 | 2020-04-22 | 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法 |
| JP2022106599A Pending JP2022141707A (ja) | 2015-05-06 | 2022-06-30 | 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法 |
| JP2023209185A Active JP7799673B2 (ja) | 2015-05-06 | 2023-12-12 | 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020075777A Active JP7530204B2 (ja) | 2015-05-06 | 2020-04-22 | 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法 |
| JP2022106599A Pending JP2022141707A (ja) | 2015-05-06 | 2022-06-30 | 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法 |
| JP2023209185A Active JP7799673B2 (ja) | 2015-05-06 | 2023-12-12 | 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法 |
Country Status (13)
| Country | Link |
|---|---|
| US (5) | US20180100150A1 (enExample) |
| EP (1) | EP3292201A2 (enExample) |
| JP (4) | JP6695902B2 (enExample) |
| KR (2) | KR102827366B1 (enExample) |
| CN (2) | CN115322991A (enExample) |
| AU (3) | AU2016257996A1 (enExample) |
| BR (1) | BR112017021967A2 (enExample) |
| CA (1) | CA2984636A1 (enExample) |
| EA (1) | EA201792444A1 (enExample) |
| HK (1) | HK1249920A1 (enExample) |
| TW (4) | TW202536180A (enExample) |
| UY (2) | UY40330A (enExample) |
| WO (1) | WO2016179342A2 (enExample) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3271482A4 (en) * | 2015-03-17 | 2019-02-13 | Arrowhead Pharmaceuticals, Inc. | COMPOSITIONS AND METHODS FOR INHIBITING THE GENE EXPRESSION OF FACTOR XII |
| CA2984636A1 (en) | 2015-05-06 | 2016-11-10 | Alnylam Pharmaceuticals, Inc. | Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof |
| WO2017120397A1 (en) * | 2016-01-08 | 2017-07-13 | Alnylam Pharmaceuticals, Inc. | Polynucleotide agents targeting factor xii (hageman factor) (f12) and methods of use thereof |
| WO2018140920A1 (en) * | 2017-01-30 | 2018-08-02 | Arrowhead Pharmaceuticals Inc. | Compositions and methods for inhibition of factor xii gene expression |
| EP3610017A2 (en) * | 2017-04-11 | 2020-02-19 | Arbutus Biopharma Corporation | Targeted compositions |
| EP3649243A1 (en) * | 2017-07-07 | 2020-05-13 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing contact-activation pathway-associated diseases using irna compositions targeting factor xii (hageman factor) (f12) |
| US11414665B2 (en) | 2017-12-01 | 2022-08-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
| CA3083970A1 (en) | 2017-12-01 | 2019-06-06 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
| EP3719127A4 (en) | 2017-12-01 | 2021-10-20 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH IT, MANUFACTURING METHOD AND USE |
| CN118291457A (zh) | 2017-12-01 | 2024-07-05 | 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 |
| KR102756073B1 (ko) | 2017-12-01 | 2025-01-20 | 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 | 이중-가닥 올리고뉴클레오티드, 조성물 및 이중-가닥 올리고뉴클레오티드를 포함하는 접합체, 그의 제조 방법 및 그의 용도 |
| WO2019128611A1 (en) | 2017-12-29 | 2019-07-04 | Suzhou Ribo Life Science Co., Ltd. | Conjugates and preparation and use thereof |
| 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 |
| JP7376952B2 (ja) | 2018-09-30 | 2023-11-09 | 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 | ｓｉＲＮＡ複合体及びその調製方法と使用 |
| WO2020135673A1 (zh) | 2018-12-28 | 2020-07-02 | 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 |
| CN118562796A (zh) * | 2019-01-18 | 2024-08-30 | 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 |
| 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 |
| CN118256498A (zh) | 2019-05-22 | 2024-06-28 | 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 |
| CN113891939B (zh) * | 2019-05-24 | 2024-04-02 | 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 |
| WO2021158858A1 (en) * | 2020-02-07 | 2021-08-12 | Intellia Therapeutics, Inc. | Compositions and methods for kallikrein ( klkb1) gene editing |
| US20230126881A1 (en) * | 2020-03-27 | 2023-04-27 | Alnylam Pharmaceuticals, Inc. | Fc FRAGMENT OF IgG RECEPTOR AND TRANSPORTER (FCGRT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| TW202242111A (zh) | 2020-12-18 | 2022-11-01 | 美商Ionis製藥公司 | 用於調節因子ｘｉｉ之化合物及方法 |
| CA3228733A1 (en) * | 2021-08-13 | 2023-02-16 | Alnylam Pharmaceuticals, Inc. | Factor xii (f12) irna compositions and methods of use thereof |
| TW202346586A (zh) * | 2022-01-28 | 2023-12-01 | 大陸商上海舶望製藥有限公司 | 用於抑制前激肽釋放酶（pkk）蛋白表達的組合物和方法 |
| WO2024169577A1 (zh) * | 2023-02-14 | 2024-08-22 | 北京福元医药股份有限公司 | 用于抑制fxii基因表达的双链核糖核酸及其修饰物、缀合物和用途 |
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