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 (https=) | 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 (https=) |
| EP (1) | EP3645013A4 (https=) |
| JP (1) | JP2020529197A (https=) |
| KR (1) | KR20200023427A (https=) |
| CN (1) | CN111050776A (https=) |
| AU (1) | AU2018294415A1 (https=) |
| CA (1) | CA3068630A1 (https=) |
| IL (2) | IL295086A (https=) |
| MX (1) | MX2020000154A (https=) |
| WO (1) | WO2019006375A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20200023427A (ko) | 2017-06-29 | 2020-03-04 | 다이서나 파마수이티컬, 인크. | Hmgb1 발현을 억제하기 위한 조성물 및 방법 |
| CA3094008A1 (en) * | 2018-04-13 | 2019-10-17 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides |
| AU2019397247A1 (en) * | 2018-12-12 | 2021-06-24 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules containing a triloop |
| MX2021007855A (es) * | 2018-12-28 | 2021-10-26 | Dicerna Pharmaceuticals Inc | Composiciones y metodos para inhibir la expresion de hmgb1. |
| US12084662B2 (en) * | 2021-04-14 | 2024-09-10 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for modulating PNPLA3 expression |
| WO2022223515A2 (en) * | 2021-04-19 | 2022-10-27 | Novo Nordisk A/S | Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression |
| JP2024532019A (ja) * | 2021-08-25 | 2024-09-05 | ノボ ノルディスク エー／エス | アルファ－１アンチトリプシン発現を阻害するための組成物及び方法 |
| WO2023102469A2 (en) * | 2021-12-01 | 2023-06-08 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for modulating apoc3 expression |
| 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 |
| WO2007076200A2 (en) * | 2005-11-28 | 2007-07-05 | Medimmune, Inc. | 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 |
| JP2012504389A (ja) * | 2008-09-22 | 2012-02-23 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法 |
| US9278108B2 (en) | 2009-07-16 | 2016-03-08 | Nec Solution Innovators, Ltd. | HMGB1 binding nucleic acid molecule and applications thereof |
| WO2012036215A1 (ja) * | 2010-09-17 | 2012-03-22 | Taniguchi Tadatsugu | Ｈｍｇｂタンパクによって仲介される免疫応答の活性化の抑制剤及びスクリーニング方法 |
| WO2014028494A1 (en) * | 2012-08-13 | 2014-02-20 | The Regents Of The University Of California | Detecting and treating liver damage |
| AU2015330670B2 (en) * | 2014-10-10 | 2022-01-06 | Novo Nordisk Health Care Ag | Therapeutic inhibition of lactate dehydrogenase and agents therefor |
| JP7105065B2 (ja) * | 2014-12-15 | 2022-07-22 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | リガンド修飾二本鎖核酸 |
| 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 |
| KR20200023427A (ko) | 2017-06-29 | 2020-03-04 | 다이서나 파마수이티컬, 인크. | Hmgb1 발현을 억제하기 위한 조성물 및 방법 |
| JP2021508491A (ja) * | 2017-12-18 | 2021-03-11 | 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | 高移動度グループボックス−１（ＨＭＧＢ１）ｉＲＮＡ組成物及びその使用方法 |
| US20190192151A1 (en) | 2017-12-21 | 2019-06-27 | Ethicon Llc | Surgical instrument having a display comprising image layers |
| MX2021007855A (es) | 2018-12-28 | 2021-10-26 | Dicerna Pharmaceuticals Inc | Composiciones y metodos para inhibir la expresion de hmgb1. |
-
2018
- 2018-06-29 KR KR1020207002542A patent/KR20200023427A/ko not_active Ceased
- 2018-06-29 IL IL295086A patent/IL295086A/en unknown
- 2018-06-29 MX MX2020000154A patent/MX2020000154A/es unknown
- 2018-06-29 WO PCT/US2018/040410 patent/WO2019006375A1/en not_active Ceased
- 2018-06-29 EP EP18824412.3A patent/EP3645013A4/en active Pending
- 2018-06-29 US US16/024,355 patent/US10675295B2/en active Active
- 2018-06-29 AU AU2018294415A patent/AU2018294415A1/en not_active Abandoned
- 2018-06-29 JP JP2019572382A patent/JP2020529197A/ja active Pending
- 2018-06-29 CA CA3068630A patent/CA3068630A1/en active Pending
- 2018-06-29 IL IL271680A patent/IL271680B/en unknown
- 2018-06-29 CN CN201880056295.0A patent/CN111050776A/zh active Pending
-
2020
- 2020-04-30 US US16/863,081 patent/US11478501B2/en active Active
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