AU2018294415A1 - Compositions and methods for inhibiting HMGB1 expression - Google Patents

Compositions and methods for inhibiting HMGB1 expression Download PDF

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Publication number
AU2018294415A1
AU2018294415A1 AU2018294415A AU2018294415A AU2018294415A1 AU 2018294415 A1 AU2018294415 A1 AU 2018294415A1 AU 2018294415 A AU2018294415 A AU 2018294415A AU 2018294415 A AU2018294415 A AU 2018294415A AU 2018294415 A1 AU2018294415 A1 AU 2018294415A1
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Australia
Prior art keywords
oligonucleotide
nucleotides
seq
sequence
antisense strand
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Abandoned
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AU2018294415A
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English (en)
Inventor
Marc Abrams
Girish CHOPDA
JiHye PARK
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of AU2018294415A1 publication Critical patent/AU2018294415A1/en
Abandoned legal-status Critical Current

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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/533Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands

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  • 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)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2018294415A 2017-06-29 2018-06-29 Compositions and methods for inhibiting HMGB1 expression Abandoned AU2018294415A1 (en)

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 (1)

Publication Number Publication Date
AU2018294415A1 true AU2018294415A1 (en) 2020-01-16

Family

ID=64735107

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2018294415A Abandoned AU2018294415A1 (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) IL271680B (https=)
MX (1) MX2020000154A (https=)
WO (1) WO2019006375A1 (https=)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL271680B (en) 2017-06-29 2022-09-01 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting hmgb1 expression
JP2021520355A (ja) * 2018-04-13 2021-08-19 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 Tm上昇ヌクレオチドで修飾された二本鎖核酸阻害剤分子
WO2020123083A1 (en) * 2018-12-12 2020-06-18 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules containing a triloop
SG11202106857VA (en) * 2018-12-28 2021-07-29 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting hmgb1 expression
CA3209418A1 (en) * 2021-04-14 2022-10-20 Utsav SAXENA 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
EP4392556A1 (en) * 2021-08-25 2024-07-03 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting ?lpha-1 antitrypsin expression
JP2024543195A (ja) * 2021-12-01 2024-11-19 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
WO2024061202A1 (zh) * 2022-09-20 2024-03-28 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)

* Cited by examiner, † Cited by third party
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
WO2009099991A2 (en) * 2008-01-31 2009-08-13 The Brigham And Women's Hospital, Inc. Treatment of cancer
DK2341943T3 (en) * 2008-09-22 2019-02-25 Dicerna Pharmaceuticals Inc COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS
EP2455469A4 (en) 2009-07-16 2013-03-13 Nec Software Ltd FOR BINDING TO HMGB1 ABLE NUCLEIC ACID MOLECULE AND USE THEREOF
JP5686814B2 (ja) * 2010-09-17 2015-03-18 独立行政法人科学技術振興機構 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
DK3569711T3 (da) * 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
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
IL271680B (en) 2017-06-29 2022-09-01 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting hmgb1 expression
EP3728593A1 (en) * 2017-12-18 2020-10-28 Alnylam Pharmaceuticals, Inc. High mobility group box-1 (hmgb1) irna compositions and methods of use thereof
US11751867B2 (en) 2017-12-21 2023-09-12 Cilag Gmbh International Surgical instrument comprising sequenced systems
SG11202106857VA (en) 2018-12-28 2021-07-29 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting hmgb1 expression

Also Published As

Publication number Publication date
EP3645013A4 (en) 2021-07-21
US10675295B2 (en) 2020-06-09
IL271680A (en) 2020-02-27
EP3645013A1 (en) 2020-05-06
IL295086A (en) 2022-09-01
CA3068630A1 (en) 2019-01-03
US11478501B2 (en) 2022-10-25
US20190000870A1 (en) 2019-01-03
JP2020529197A (ja) 2020-10-08
KR20200023427A (ko) 2020-03-04
MX2020000154A (es) 2020-07-22
CN111050776A (zh) 2020-04-21
US20200297754A1 (en) 2020-09-24
WO2019006375A1 (en) 2019-01-03
IL271680B (en) 2022-09-01

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