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 (cg-RX-API-DMAC7.html) | 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 Pending JP2024037849A (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 Pending JP2024037849A (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 (4) | US20180100150A1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3292201A2 (cg-RX-API-DMAC7.html) |
| JP (4) | JP6695902B2 (cg-RX-API-DMAC7.html) |
| KR (2) | KR102827366B1 (cg-RX-API-DMAC7.html) |
| CN (2) | CN108271386B (cg-RX-API-DMAC7.html) |
| AU (3) | AU2016257996A1 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112017021967A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2984636A1 (cg-RX-API-DMAC7.html) |
| EA (1) | EA201792444A1 (cg-RX-API-DMAC7.html) |
| HK (1) | HK1249920A1 (cg-RX-API-DMAC7.html) |
| TW (4) | TW202536180A (cg-RX-API-DMAC7.html) |
| UY (2) | UY36671A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2016179342A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2016233364A1 (en) * | 2015-03-17 | 2017-09-21 | Arrowhead Pharmaceuticals, Inc. | Compositions and methods for inhibiting gene expression of factor Xll |
| WO2016179342A2 (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 |
| CN119548520A (zh) * | 2017-01-30 | 2025-03-04 | 箭头药业股份有限公司 | 用于抑制因子xii基因表达的组合物和方法 |
| NZ757927A (en) | 2017-04-11 | 2023-07-28 | Arbutus Biopharma Corp | Targeted compositions |
| US20200208150A1 (en) * | 2017-07-07 | 2020-07-02 | Alnylam Pharmaceuticals, Inc. | METHODS FOR TREATING OR PREVENTING CONTACT-ACTIVATION PATHWAY-ASSOCIATED DISEASES USING iRNA COMPOSITIONS TARGETING FACTOR XII (HAGEMAN FACTOR) (F12) |
| US12083142B2 (en) | 2017-12-01 | 2024-09-10 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
| JP7727308B2 (ja) | 2017-12-01 | 2025-08-21 | 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 | 二本鎖オリゴヌクレオチド、二本鎖オリゴヌクレオチドを含む組成物および複合体ならびに調製方法と使用 |
| 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 |
| US11414661B2 (en) | 2017-12-01 | 2022-08-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof |
| JP7273417B2 (ja) | 2017-12-01 | 2023-05-15 | 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および複合体ならびに調製方法と使用 |
| DK3732185T3 (da) | 2017-12-29 | 2025-03-31 | Suzhou Ribo Life Science Co Ltd | Konjugater og fremstilling og anvendelse heraf |
| US11918600B2 (en) | 2018-08-21 | 2024-03-05 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition and conjugate containing nucleic acid, and use thereof |
| CN111655297A (zh) | 2018-09-30 | 2020-09-11 | 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 |
| EP3903830A4 (en) | 2018-12-28 | 2023-04-26 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH NUCLEIC ACID, PROCESS FOR THEIR PREPARATION AND ITS USE |
| CN113330117B (zh) * | 2019-01-18 | 2024-05-28 | 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 |
| US20240200060A1 (en) * | 2019-05-22 | 2024-06-20 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| AU2020280438B2 (en) | 2019-05-22 | 2025-03-06 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| JP7614650B2 (ja) * | 2019-05-24 | 2025-01-16 | 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並びに調製方法と使用 |
| CA3169813A1 (en) * | 2020-02-07 | 2021-08-12 | Intellia Therapeutics, Inc. | Compositions and methods for kallikrein ( klkb1) gene editing |
| WO2021195574A1 (en) * | 2020-03-27 | 2021-09-30 | Alnylam Pharmaceuticals, Inc. | Fc FRAGMENT OF IgG RECEPTOR AND TRANSPORTER (FCGRT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF |
| AR124408A1 (es) | 2020-12-18 | 2023-03-22 | Ionis Pharmaceuticals Inc | Compuestos y métodos para modular el factor xii |
| JP2024534766A (ja) | 2021-08-13 | 2024-09-26 | アルナイラム ファーマシューティカルズ， インコーポレイテッド | 第ＸＩＩ因子（Ｆ１２）ｉＲＮＡ組成物およびその使用方法 |
| 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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