CA3068630A1 - Compositions and methods for inhibiting hmgb1 expression - Google Patents

Compositions and methods for inhibiting hmgb1 expression Download PDF

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Publication number
CA3068630A1
CA3068630A1 CA3068630A CA3068630A CA3068630A1 CA 3068630 A1 CA3068630 A1 CA 3068630A1 CA 3068630 A CA3068630 A CA 3068630A CA 3068630 A CA3068630 A CA 3068630A CA 3068630 A1 CA3068630 A1 CA 3068630A1
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CA
Canada
Prior art keywords
oligonucleotide
nucleotides
hmgb1
seq
sequence
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
Application number
CA3068630A
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English (en)
French (fr)
Inventor
Marc Abrams
Girish Chopda
Jihye PARK
Original Assignee
Dicerna Pharmaceuticals Inc
Priority date (The priority date 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 date listed.)
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Publication date
Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of CA3068630A1 publication Critical patent/CA3068630A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • 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

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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)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (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)
CA3068630A 2017-06-29 2018-06-29 Compositions and methods for inhibiting hmgb1 expression Pending CA3068630A1 (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
CA3068630A1 true CA3068630A1 (en) 2019-01-03

Family

ID=64735107

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3068630A Pending CA3068630A1 (en) 2017-06-29 2018-06-29 Compositions and methods for inhibiting hmgb1 expression

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)

* Cited by examiner, † Cited by third party
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)

* 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
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

Also Published As

Publication number Publication date
US10675295B2 (en) 2020-06-09
EP3645013A4 (en) 2021-07-21
IL295086A (en) 2022-09-01
JP2020529197A (ja) 2020-10-08
CN111050776A (zh) 2020-04-21
US20190000870A1 (en) 2019-01-03
KR20200023427A (ko) 2020-03-04
AU2018294415A1 (en) 2020-01-16
US11478501B2 (en) 2022-10-25
IL271680B (en) 2022-09-01
IL271680A (en) 2020-02-27
US20200297754A1 (en) 2020-09-24
WO2019006375A1 (en) 2019-01-03
MX2020000154A (es) 2020-07-22
EP3645013A1 (en) 2020-05-06

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Effective date: 20220928

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