JP2020529197A5 - - Google Patents
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- JP2020529197A5 JP2020529197A5 JP2019572382A JP2019572382A JP2020529197A5 JP 2020529197 A5 JP2020529197 A5 JP 2020529197A5 JP 2019572382 A JP2019572382 A JP 2019572382A JP 2019572382 A JP2019572382 A JP 2019572382A JP 2020529197 A5 JP2020529197 A5 JP 2020529197A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- oligonucleotide
- nucleotides
- antisense strand
- sense strand
- 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.)
- Pending
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- 108091034117 Oligonucleotide Proteins 0.000 claims description 113
- 125000003729 nucleotide group Chemical group 0.000 claims description 67
- 239000002773 nucleotide Substances 0.000 claims description 58
- 230000000692 anti-sense effect Effects 0.000 claims description 53
- 108091081021 Sense strand Proteins 0.000 claims description 48
- 102000055207 HMGB1 Human genes 0.000 claims description 23
- 108700010013 HMGB1 Proteins 0.000 claims description 23
- 230000000295 complement effect Effects 0.000 claims description 23
- 101100339431 Arabidopsis thaliana HMGB2 gene Proteins 0.000 claims description 22
- 101150021904 HMGB1 gene Proteins 0.000 claims description 22
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical group CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims description 13
- 239000003446 ligand Substances 0.000 claims description 11
- 238000012986 modification Methods 0.000 claims description 9
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 claims description 6
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 claims description 6
- 150000003013 phosphoric acid derivatives Chemical class 0.000 claims description 6
- 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 description 5
- 229910052799 carbon Inorganic materials 0.000 claims description 4
- 239000000203 mixture Substances 0.000 claims description 4
- 230000004048 modification Effects 0.000 claims description 4
- 206010008635 Cholestasis Diseases 0.000 claims description 3
- 208000027761 Hepatic autoimmune disease Diseases 0.000 claims description 3
- 239000002253 acid Substances 0.000 claims description 3
- 231100000359 cholestasis Toxicity 0.000 claims description 3
- 230000007870 cholestasis Effects 0.000 claims description 3
- 208000019425 cirrhosis of liver Diseases 0.000 claims description 3
- ZTWTYVWXUKTLCP-UHFFFAOYSA-L ethenyl-dioxido-oxo-$l^{5}-phosphane Chemical compound [O-]P([O-])(=O)C=C ZTWTYVWXUKTLCP-UHFFFAOYSA-L 0.000 claims description 3
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 3
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 230000008685 targeting Effects 0.000 claims 2
- MSWZFWKMSRAUBD-GASJEMHNSA-N 2-amino-2-deoxy-D-galactopyranose Chemical compound N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O MSWZFWKMSRAUBD-GASJEMHNSA-N 0.000 claims 1
- CTVFZKJXQWDXIO-UHFFFAOYSA-N P1(OC(CC(=O)O1)=O)=O Chemical compound P1(OC(CC(=O)O1)=O)=O CTVFZKJXQWDXIO-UHFFFAOYSA-N 0.000 claims 1
- MSWZFWKMSRAUBD-UHFFFAOYSA-N beta-D-galactosamine Natural products NC1C(O)OC(CO)C(O)C1O MSWZFWKMSRAUBD-UHFFFAOYSA-N 0.000 claims 1
- UEZVMMHDMIWARA-UHFFFAOYSA-M phosphonate Chemical compound [O-]P(=O)=O UEZVMMHDMIWARA-UHFFFAOYSA-M 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 238000000034 method Methods 0.000 description 14
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 description 4
- 239000003795 chemical substances by application Substances 0.000 description 3
- 231100000334 hepatotoxic Toxicity 0.000 description 3
- 230000003082 hepatotoxic effect Effects 0.000 description 3
- 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 description 2
- 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 description 2
- 150000007513 acids Chemical class 0.000 description 2
- 150000001720 carbohydrates Chemical class 0.000 description 2
- 235000014633 carbohydrates Nutrition 0.000 description 2
- 235000012000 cholesterol Nutrition 0.000 description 2
- 150000002337 glycosamines Chemical class 0.000 description 2
- 150000002632 lipids Chemical class 0.000 description 2
- 229920001184 polypeptide Polymers 0.000 description 2
- 102000004196 processed proteins & peptides Human genes 0.000 description 2
- 108090000765 processed proteins & peptides Proteins 0.000 description 2
- 108091023037 Aptamer Proteins 0.000 description 1
- 108091030071 RNAI Proteins 0.000 description 1
- 230000009368 gene silencing by RNA Effects 0.000 description 1
- 230000002440 hepatic effect Effects 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 210000002966 serum Anatomy 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762526971P | 2017-06-29 | 2017-06-29 | |
| US62/526,971 | 2017-06-29 | ||
| PCT/US2018/040410 WO2019006375A1 (en) | 2017-06-29 | 2018-06-29 | COMPOSITIONS AND METHODS FOR INHIBITING HMGB1 EXPRESSION |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020529197A JP2020529197A (ja) | 2020-10-08 |
| JP2020529197A5 true JP2020529197A5 (enExample) | 2021-08-05 |
Family
ID=64735107
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019572382A Pending JP2020529197A (ja) | 2017-06-29 | 2018-06-29 | Ｈｍｇｂ１発現を阻害するための組成物及び方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US10675295B2 (enExample) |
| EP (1) | EP3645013A4 (enExample) |
| JP (1) | JP2020529197A (enExample) |
| KR (1) | KR20200023427A (enExample) |
| CN (1) | CN111050776A (enExample) |
| AU (1) | AU2018294415A1 (enExample) |
| CA (1) | CA3068630A1 (enExample) |
| IL (2) | IL271680B (enExample) |
| MX (1) | MX2020000154A (enExample) |
| WO (1) | WO2019006375A1 (enExample) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10675295B2 (en) | 2017-06-29 | 2020-06-09 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting HMGB1 expression |
| CN111971051A (zh) * | 2018-04-13 | 2020-11-20 | 迪克纳制药公司 | 用增加tm的核苷酸修饰的双链核酸抑制剂分子 |
| JP7535047B2 (ja) * | 2018-12-12 | 2024-08-15 | ディセルナ ファーマシューティカルズ インコーポレイテッド | トリループを含む二本鎖核酸インヒビター分子 |
| IL284327B2 (en) * | 2018-12-28 | 2024-11-01 | Dicerna Pharmaceuticals Inc | Compositions and methods for inhibiting hmgb1 expression |
| KR20230171431A (ko) * | 2021-04-14 | 2023-12-20 | 다이서나 파마수이티컬, 인크. | Pnpla3 발현을 조절하기 위한 조성물 및 방법 |
| IL307154A (en) * | 2021-04-19 | 2023-11-01 | Novo Nordisk As | Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression |
| CN117280031A (zh) * | 2021-04-19 | 2023-12-22 | 诺和诺德股份有限公司 | 用于抑制核受体亚家族1组h成员3（nr1h3）表达的组合物和方法 |
| KR20240046843A (ko) * | 2021-08-25 | 2024-04-11 | 노보 노르디스크 에이/에스 | 알파-1 항트립신 발현을 억제하기 위한 조성물 및 방법 |
| JP7877458B2 (ja) * | 2021-12-01 | 2026-06-22 | ディセルナ ファーマシューティカルズ インコーポレイテッド | Ａｐｏｃ３発現を調節するための組成物及び方法 |
| TWI868755B (zh) * | 2022-06-24 | 2025-01-01 | 丹麥商諾佛 儂迪克股份有限公司 | 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法 |
| CN117264954A (zh) * | 2022-09-20 | 2023-12-22 | 北京福元医药股份有限公司 | 用于抑制hmgb1基因表达的双链核糖核酸及其修饰物、缀合物和用途 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| US20080311122A1 (en) | 2005-11-28 | 2008-12-18 | Medimmune, Llc | Antagonists of Hmgb1 and/or Rage and Methods of Use Thereof |
| US8178503B2 (en) * | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
| US20110081362A1 (en) * | 2008-01-31 | 2011-04-07 | The Brigham And Women's Hospital, Inc. | Treatment of cancer |
| WO2010033225A2 (en) * | 2008-09-22 | 2010-03-25 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications |
| WO2011007876A1 (ja) * | 2009-07-16 | 2011-01-20 | Ｎｅｃソフト株式会社 | Ｈｍｇｂ１結合核酸分子およびその用途 |
| EP2617426B1 (en) * | 2010-09-17 | 2016-08-10 | Japan Science and Technology Agency | Inhibitor of hmgb protein-mediated immune response activation |
| WO2014028494A1 (en) * | 2012-08-13 | 2014-02-20 | The Regents Of The University Of California | Detecting and treating liver damage |
| US10351854B2 (en) * | 2014-10-10 | 2019-07-16 | Dicerna Pharmaceuticals, Inc. | Therapeutic inhibition of lactate dehydrogenase and agents therefor |
| EP3865576A1 (en) * | 2014-12-15 | 2021-08-18 | Dicerna Pharmaceuticals, Inc. | Ligand-modified double-stranded nucleic acids |
| WO2017079227A1 (en) * | 2015-11-05 | 2017-05-11 | University Of Connecticut | Compositions and methods for the treatment of liver fibrosis |
| CN106244589A (zh) * | 2016-08-01 | 2016-12-21 | 中国人民解放军第四军医大学 | 靶向hmgb1基因的rna干扰片段及其应用 |
| US10675295B2 (en) | 2017-06-29 | 2020-06-09 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting HMGB1 expression |
| WO2019126097A1 (en) * | 2017-12-18 | 2019-06-27 | Alnylam Pharmaceuticals, Inc. | High mobility group box-1 (hmgb1) irna compositions and methods of use thereof |
| US11883019B2 (en) | 2017-12-21 | 2024-01-30 | Cilag Gmbh International | Stapling instrument comprising a staple feeding system |
| IL284327B2 (en) | 2018-12-28 | 2024-11-01 | Dicerna Pharmaceuticals Inc | Compositions and methods for inhibiting hmgb1 expression |
-
2018
- 2018-06-29 US US16/024,355 patent/US10675295B2/en active Active
- 2018-06-29 KR KR1020207002542A patent/KR20200023427A/ko not_active Ceased
- 2018-06-29 CN CN201880056295.0A patent/CN111050776A/zh active Pending
- 2018-06-29 MX MX2020000154A patent/MX2020000154A/es unknown
- 2018-06-29 IL IL271680A patent/IL271680B/en unknown
- 2018-06-29 JP JP2019572382A patent/JP2020529197A/ja active Pending
- 2018-06-29 EP EP18824412.3A patent/EP3645013A4/en active Pending
- 2018-06-29 AU AU2018294415A patent/AU2018294415A1/en not_active Abandoned
- 2018-06-29 WO PCT/US2018/040410 patent/WO2019006375A1/en not_active Ceased
- 2018-06-29 CA CA3068630A patent/CA3068630A1/en active Pending
- 2018-06-29 IL IL295086A patent/IL295086A/en unknown
-
2020
- 2020-04-30 US US16/863,081 patent/US11478501B2/en active Active
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