IL295086A - Compositions and methods for inhibiting expression of hmgb1 - Google Patents
Compositions and methods for inhibiting expression of hmgb1Info
- Publication number
- IL295086A IL295086A IL295086A IL29508622A IL295086A IL 295086 A IL295086 A IL 295086A IL 295086 A IL295086 A IL 295086A IL 29508622 A IL29508622 A IL 29508622A IL 295086 A IL295086 A IL 295086A
- Authority
- IL
- Israel
- Prior art keywords
- oligonucleotide
- seq
- nucleotides
- antisense strand
- sequence
- Prior art date
Links
- 238000000034 method Methods 0.000 title claims 17
- 239000000203 mixture Substances 0.000 title claims 3
- 230000002401 inhibitory effect Effects 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 93
- 125000003729 nucleotide group Chemical group 0.000 claims 39
- 230000000692 anti-sense effect Effects 0.000 claims 38
- 239000002773 nucleotide Substances 0.000 claims 37
- 108091081021 Sense strand Proteins 0.000 claims 33
- 102000055207 HMGB1 Human genes 0.000 claims 12
- 108700010013 HMGB1 Proteins 0.000 claims 12
- 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 12
- 230000000295 complement effect Effects 0.000 claims 12
- 101100339431 Arabidopsis thaliana HMGB2 gene Proteins 0.000 claims 11
- 101150021904 HMGB1 gene Proteins 0.000 claims 11
- 239000003446 ligand Substances 0.000 claims 7
- 230000008685 targeting Effects 0.000 claims 7
- 238000012986 modification Methods 0.000 claims 6
- 102000000849 HMGB Proteins Human genes 0.000 claims 4
- 108010001860 HMGB Proteins Proteins 0.000 claims 4
- 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 claims 4
- 230000004048 modification Effects 0.000 claims 4
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 claims 4
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 claims 4
- 125000002467 phosphate group Chemical class [H]OP(=O)(O[H])O[*] 0.000 claims 4
- 239000003795 chemical substances by application Substances 0.000 claims 3
- 231100000334 hepatotoxic Toxicity 0.000 claims 3
- 230000003082 hepatotoxic effect Effects 0.000 claims 3
- 206010008635 Cholestasis Diseases 0.000 claims 2
- 208000027761 Hepatic autoimmune disease Diseases 0.000 claims 2
- 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 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 claims 2
- 239000002253 acid Substances 0.000 claims 2
- 150000001720 carbohydrates Chemical class 0.000 claims 2
- 235000014633 carbohydrates Nutrition 0.000 claims 2
- 229910052799 carbon Inorganic materials 0.000 claims 2
- 230000001587 cholestatic effect Effects 0.000 claims 2
- 235000012000 cholesterol Nutrition 0.000 claims 2
- 208000019425 cirrhosis of liver Diseases 0.000 claims 2
- ZTWTYVWXUKTLCP-UHFFFAOYSA-L ethenyl-dioxido-oxo-$l^{5}-phosphane Chemical compound [O-]P([O-])(=O)C=C ZTWTYVWXUKTLCP-UHFFFAOYSA-L 0.000 claims 2
- 150000002337 glycosamines Chemical class 0.000 claims 2
- 150000002632 lipids Chemical class 0.000 claims 2
- 229920001184 polypeptide Polymers 0.000 claims 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- 108091023037 Aptamer Proteins 0.000 claims 1
- 108091030071 RNAI Proteins 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 108020004999 messenger RNA Proteins 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762526971P | 2017-06-29 | 2017-06-29 | |
| PCT/US2018/040410 WO2019006375A1 (en) | 2017-06-29 | 2018-06-29 | COMPOSITIONS AND METHODS FOR INHIBITING HMGB1 EXPRESSION |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL295086A true IL295086A (en) | 2022-09-01 |
Family
ID=64735107
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL295086A IL295086A (en) | 2017-06-29 | 2018-06-29 | Compositions and methods for inhibiting expression of hmgb1 |
| IL271680A IL271680B (en) | 2017-06-29 | 2018-06-29 | Compositions and methods for inhibiting hmgb1 expression |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL271680A IL271680B (en) | 2017-06-29 | 2018-06-29 | Compositions and methods for inhibiting hmgb1 expression |
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
Also Published As
| Publication number | Publication date |
|---|---|
| MX2020000154A (es) | 2020-07-22 |
| EP3645013A4 (en) | 2021-07-21 |
| US20200297754A1 (en) | 2020-09-24 |
| JP2020529197A (ja) | 2020-10-08 |
| AU2018294415A1 (en) | 2020-01-16 |
| WO2019006375A1 (en) | 2019-01-03 |
| US20190000870A1 (en) | 2019-01-03 |
| US11478501B2 (en) | 2022-10-25 |
| IL271680B (en) | 2022-09-01 |
| IL271680A (en) | 2020-02-27 |
| US10675295B2 (en) | 2020-06-09 |
| CA3068630A1 (en) | 2019-01-03 |
| CN111050776A (zh) | 2020-04-21 |
| EP3645013A1 (en) | 2020-05-06 |
| KR20200023427A (ko) | 2020-03-04 |
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