JP2016533751A5 - - Google Patents
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- JP2016533751A5 JP2016533751A5 JP2016537862A JP2016537862A JP2016533751A5 JP 2016533751 A5 JP2016533751 A5 JP 2016533751A5 JP 2016537862 A JP2016537862 A JP 2016537862A JP 2016537862 A JP2016537862 A JP 2016537862A JP 2016533751 A5 JP2016533751 A5 JP 2016533751A5
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- oligonucleotide
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- 150000001875 compounds Chemical class 0.000 claims description 88
- 108091034117 Oligonucleotide Proteins 0.000 claims description 62
- 239000002777 nucleoside Substances 0.000 claims description 54
- 125000003835 nucleoside group Chemical group 0.000 claims description 36
- 150000003833 nucleoside derivatives Chemical class 0.000 claims description 14
- 230000000295 complement effect Effects 0.000 claims description 13
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 12
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical group CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 12
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 9
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 8
- 239000000203 mixture Substances 0.000 claims description 8
- 229930024421 Adenine Natural products 0.000 claims description 6
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 6
- 229960000643 adenine Drugs 0.000 claims description 6
- 229940113082 thymine Drugs 0.000 claims description 6
- 208000028185 Angioedema Diseases 0.000 claims description 4
- 208000005189 Embolism Diseases 0.000 claims description 4
- 206010019860 Hereditary angioedema Diseases 0.000 claims description 4
- 208000001435 Thromboembolism Diseases 0.000 claims description 4
- 201000010099 disease Diseases 0.000 claims description 4
- 208000035475 disorder Diseases 0.000 claims description 4
- 206010030113 Oedema Diseases 0.000 claims description 3
- 239000003085 diluting agent Substances 0.000 claims description 3
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 2
- 206010048962 Brain oedema Diseases 0.000 claims description 2
- 206010061216 Infarction Diseases 0.000 claims description 2
- 208000001344 Macular Edema Diseases 0.000 claims description 2
- 206010025415 Macular oedema Diseases 0.000 claims description 2
- 208000010378 Pulmonary Embolism Diseases 0.000 claims description 2
- 208000006011 Stroke Diseases 0.000 claims description 2
- 206010042674 Swelling Diseases 0.000 claims description 2
- 208000007536 Thrombosis Diseases 0.000 claims description 2
- 206010047249 Venous thrombosis Diseases 0.000 claims description 2
- 208000006752 brain edema Diseases 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims description 2
- 210000000744 eyelid Anatomy 0.000 claims description 2
- 230000007574 infarction Effects 0.000 claims description 2
- 208000027866 inflammatory disease Diseases 0.000 claims description 2
- 201000010230 macular retinal edema Diseases 0.000 claims description 2
- 208000010125 myocardial infarction Diseases 0.000 claims description 2
- 150000003839 salts Chemical class 0.000 claims description 2
- 230000008961 swelling Effects 0.000 claims description 2
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 125000002619 bicyclic group Chemical group 0.000 claims 2
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical group P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 claims 2
- 239000002953 phosphate buffered saline Substances 0.000 claims 2
- 206010052139 Eye oedema Diseases 0.000 claims 1
- 230000000692 anti-sense effect Effects 0.000 claims 1
- 230000004048 modification Effects 0.000 description 9
- 238000012986 modification Methods 0.000 description 9
- 238000000034 method Methods 0.000 description 5
- 230000005764 inhibitory process Effects 0.000 description 4
- 108020004999 messenger RNA Proteins 0.000 description 4
- -1 ethyl nucleoside Chemical class 0.000 description 2
- IHLOTZVBEUFDMD-UUOKFMHZSA-N 7-[(2r,3r,4s,5r)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-2,2-dioxo-1h-imidazo[4,5-c][1,2,6]thiadiazin-4-one Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(NS(=O)(=O)NC2=O)=C2N=C1 IHLOTZVBEUFDMD-UUOKFMHZSA-N 0.000 description 1
- 206010051055 Deep vein thrombosis Diseases 0.000 description 1
- 239000000074 antisense oligonucleotide Substances 0.000 description 1
- 238000012230 antisense oligonucleotides Methods 0.000 description 1
- 239000003814 drug Substances 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 108020004707 nucleic acids Proteins 0.000 description 1
- 102000039446 nucleic acids Human genes 0.000 description 1
- 150000007523 nucleic acids Chemical class 0.000 description 1
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 102000004169 proteins and genes Human genes 0.000 description 1
- 108090000623 proteins and genes Proteins 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361871175P | 2013-08-28 | 2013-08-28 | |
| US61/871,175 | 2013-08-28 | ||
| PCT/US2014/053266 WO2015031679A2 (en) | 2013-08-28 | 2014-08-28 | Modulation of prekallikrein (pkk) expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020009600A Division JP2020072732A (ja) | 2013-08-28 | 2020-01-24 | プレカリクレイン(pkk)発現の調節 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016533751A JP2016533751A (ja) | 2016-11-04 |
| JP2016533751A5 true JP2016533751A5 (cg-RX-API-DMAC7.html) | 2017-10-05 |
| JP6652922B2 JP6652922B2 (ja) | 2020-02-26 |
Family
ID=52587489
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016537862A Active JP6652922B2 (ja) | 2013-08-28 | 2014-08-28 | プレカリクレイン(pkk)発現の調節 |
| JP2020009600A Pending JP2020072732A (ja) | 2013-08-28 | 2020-01-24 | プレカリクレイン(pkk)発現の調節 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020009600A Pending JP2020072732A (ja) | 2013-08-28 | 2020-01-24 | プレカリクレイン(pkk)発現の調節 |
Country Status (14)
| Country | Link |
|---|---|
| US (5) | US9670492B2 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3038627B1 (cg-RX-API-DMAC7.html) |
| JP (2) | JP6652922B2 (cg-RX-API-DMAC7.html) |
| KR (1) | KR102365486B1 (cg-RX-API-DMAC7.html) |
| CN (1) | CN105517556B (cg-RX-API-DMAC7.html) |
| AU (2) | AU2014312196C1 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112016004093A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2921839A1 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2790574T3 (cg-RX-API-DMAC7.html) |
| IL (1) | IL244047A0 (cg-RX-API-DMAC7.html) |
| MX (1) | MX2016002587A (cg-RX-API-DMAC7.html) |
| NZ (1) | NZ716816A (cg-RX-API-DMAC7.html) |
| RU (1) | RU2712559C9 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2015031679A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102365486B1 (ko) | 2013-08-28 | 2022-02-18 | 아이오니스 파마수티컬즈, 인코포레이티드 | 프리칼리크레인 (pkk) 발현의 조절 |
| DK3137091T3 (da) | 2014-05-01 | 2021-01-25 | Ionis Pharmaceuticals Inc | Konjugater af modificerede antisense-oligonukleotider og anvendelse deraf til modulation af pkk-ekspression |
| IL315175A (en) * | 2016-09-16 | 2024-10-01 | Takeda Pharmaceuticals Co | RNA biomarkers for hereditary angioedema |
| JP2019535839A (ja) | 2016-11-29 | 2019-12-12 | ピュアテック ヘルス エルエルシー | 治療剤の送達のためのエクソソーム |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| KR20220152569A (ko) * | 2020-03-13 | 2022-11-16 | 아이오니스 파마수티컬즈, 인코포레이티드 | 프리칼리크레인-연합된 병태를 치료하고 예방하는 조성물 및 방법 |
| WO2021248027A1 (en) * | 2020-06-05 | 2021-12-09 | Ionis Pharmaceuticals, Inc. | Compositions and methods of inhibiting angiotensin-converting enzyme 2 (ace2) and transmembrane serine protease 2 (tmprss2) |
| LT4136092T (lt) * | 2020-11-18 | 2024-09-25 | Ionis Pharmaceuticals, Inc. | Junginiai ir būdai, skirti angiotenzinogeno raiškos moduliavimui |
| WO2022143531A1 (zh) * | 2020-12-29 | 2022-07-07 | 苏州瑞博生物技术股份有限公司 | 一种核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途 |
| WO2023034870A2 (en) * | 2021-09-01 | 2023-03-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing dmpk expression |
| US12042509B2 (en) | 2021-10-01 | 2024-07-23 | Adarx Pharmaceuticals, Inc. | Prekallikrein-modulating 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 |
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