JP2020529197A - Ｈｍｇｂ１発現を阻害するための組成物及び方法 - Google Patents

Ｈｍｇｂ１発現を阻害するための組成物及び方法 Download PDF

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Publication number
JP2020529197A
JP2020529197A JP2019572382A JP2019572382A JP2020529197A JP 2020529197 A JP2020529197 A JP 2020529197A JP 2019572382 A JP2019572382 A JP 2019572382A JP 2019572382 A JP2019572382 A JP 2019572382A JP 2020529197 A JP2020529197 A JP 2020529197A
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oligonucleotide
hmgb1
seq
nucleotides
oligonucleotide according
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JP2020529197A5 (enExample
Inventor
マーク エイブラムス
マーク エイブラムス
ギリシュ チョプダ
ギリシュ チョプダ
ジヘ パク
ジヘ パク
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Dicerna Pharmaceuticals Inc
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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
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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)
JP2019572382A 2017-06-29 2018-06-29 Ｈｍｇｂ１発現を阻害するための組成物及び方法 Pending JP2020529197A (ja)

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)

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JP2020529197A true JP2020529197A (ja) 2020-10-08
JP2020529197A5 JP2020529197A5 (enExample) 2021-08-05

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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)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024002985A (ja) * 2022-06-24 2024-01-11 ノヴォ ノルディスク アー／エス 膜貫通型セリンプロテアーゼ６（ｔｍｐｒｓｓ６）発現を阻害するための組成物および方法
JP2024514880A (ja) * 2021-04-14 2024-04-03 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 Ｐｎｐｌａ３発現を調節するための組成物及び方法

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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
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
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

Citations (4)

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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
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
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
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

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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
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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

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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024514880A (ja) * 2021-04-14 2024-04-03 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 Ｐｎｐｌａ３発現を調節するための組成物及び方法
JP7877355B2 (ja) 2021-04-14 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 Ｐｎｐｌａ３発現を調節するための組成物及び方法
JP2024002985A (ja) * 2022-06-24 2024-01-11 ノヴォ ノルディスク アー／エス 膜貫通型セリンプロテアーゼ６（ｔｍｐｒｓｓ６）発現を阻害するための組成物および方法
JP7560616B2 (ja) 2022-06-24 2024-10-02 ノヴォ ノルディスク アー／エス 膜貫通型セリンプロテアーゼ６（ｔｍｐｒｓｓ６）発現を阻害するための組成物および方法

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Publication number Publication date
US10675295B2 (en) 2020-06-09
EP3645013A4 (en) 2021-07-21
IL295086A (en) 2022-09-01
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
CA3068630A1 (en) 2019-01-03

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