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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- modified
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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 (https=) | 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 (https=) |
| EP (2) | EP3038627B1 (https=) |
| JP (2) | JP6652922B2 (https=) |
| KR (1) | KR102365486B1 (https=) |
| CN (1) | CN105517556B (https=) |
| AU (2) | AU2014312196C1 (https=) |
| BR (1) | BR112016004093A2 (https=) |
| CA (1) | CA2921839A1 (https=) |
| ES (1) | ES2790574T3 (https=) |
| IL (1) | IL244047A0 (https=) |
| MX (1) | MX2016002587A (https=) |
| NZ (1) | NZ716816A (https=) |
| RU (1) | RU2712559C9 (https=) |
| WO (1) | WO2015031679A2 (https=) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102365486B1 (ko) | 2013-08-28 | 2022-02-18 | 아이오니스 파마수티컬즈, 인코포레이티드 | 프리칼리크레인 (pkk) 발현의 조절 |
| EP3862362A3 (en) | 2014-05-01 | 2021-10-27 | Ionis Pharmaceuticals, Inc. | Conjugates of modified antisense oligonucleotides and their use for modulating pkk expression |
| EA201990730A1 (ru) * | 2016-09-16 | 2019-08-30 | Дайэкс Корп. | Рнк-биомаркеры для наследственного ангионевротического отека |
| EP4035659A1 (en) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | Exosomes for delivery of therapeutic agents |
| 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 |
| WO2019105437A1 (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 |
| 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 |
| 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 |
| CA3083968C (en) | 2017-12-01 | 2024-04-23 | Suzhou Ribo Life Science Co., Ltd. | Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof |
| EP3732185B1 (en) | 2017-12-29 | 2025-02-26 | Suzhou Ribo Life Science Co., Ltd. | Conjugates and preparation and use thereof |
| 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 |
| EP3862024A4 (en) | 2018-09-30 | 2022-08-17 | Suzhou Ribo Life Science Co., Ltd. | SIRNA CONJUGATE, METHOD FOR ITS PRODUCTION AND ITS USE |
| 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 |
| WO2020233680A1 (zh) | 2019-05-22 | 2020-11-26 | 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 |
| JP7610268B2 (ja) | 2019-05-22 | 2025-01-08 | 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 |
| AU2020280438B2 (en) | 2019-05-22 | 2025-03-06 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| US20220315929A1 (en) * | 2019-05-24 | 2022-10-06 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition and conjugate, preparation method therefor and use thereof |
| EP3978609A4 (en) | 2019-05-24 | 2024-02-07 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use |
| JP2023512758A (ja) * | 2020-02-07 | 2023-03-29 | インテリア セラピューティクス,インコーポレイテッド | カリクレイン(klkb1)遺伝子編集のための組成物および方法 |
| 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) |
| EP4136092B1 (en) | 2020-11-18 | 2024-07-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating angiotensinogen expression |
| EP4273245A4 (en) * | 2020-12-29 | 2024-12-11 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND SIRNA CONJUGATE WITH THE NUCLEIC ACID, MANUFACTURING PROCESS THEREOF AND USE THEREOF |
| EP4396352A4 (en) * | 2021-09-01 | 2025-10-01 | Ionis Pharmaceuticals Inc | COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION |
| MX2024004011A (es) | 2021-10-01 | 2024-07-01 | Adarx Pharmaceuticals Inc | Composiciones moduladoras de precalicreína y métodos de uso de estas. |
| 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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2014
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- 2014-08-28 CN CN201480046661.6A patent/CN105517556B/zh active Active
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- 2014-08-28 MX MX2016002587A patent/MX2016002587A/es unknown
- 2014-08-28 BR BR112016004093A patent/BR112016004093A2/pt active Search and Examination
- 2014-08-28 CA CA2921839A patent/CA2921839A1/en not_active Abandoned
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- 2014-08-28 US US14/915,039 patent/US9670492B2/en active Active
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- 2014-08-28 WO PCT/US2014/053266 patent/WO2015031679A2/en not_active Ceased
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