CN111050776A - 用于抑制hmgb1表达的组合物和方法 - Google Patents

用于抑制hmgb1表达的组合物和方法 Download PDF

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Publication number
CN111050776A
CN111050776A CN201880056295.0A CN201880056295A CN111050776A CN 111050776 A CN111050776 A CN 111050776A CN 201880056295 A CN201880056295 A CN 201880056295A CN 111050776 A CN111050776 A CN 111050776A
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oligonucleotide
nucleotides
seq
sequence
hmgb1
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CN201880056295.0A
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Chinese (zh)
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M.阿布拉姆斯
G.乔达
朴智慧
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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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
    • 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
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    • 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
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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
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    • 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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
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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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  • 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)
CN201880056295.0A 2017-06-29 2018-06-29 用于抑制hmgb1表达的组合物和方法 Pending CN111050776A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762526971P 2017-06-29 2017-06-29
US62/526971 2017-06-29
PCT/US2018/040410 WO2019006375A1 (en) 2017-06-29 2018-06-29 COMPOSITIONS AND METHODS FOR INHIBITING HMGB1 EXPRESSION

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CN111050776A true CN111050776A (zh) 2020-04-21

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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
CN113454221A (zh) * 2018-12-12 2021-09-28 迪克纳制药公司 含有三元环的双链核酸抑制剂分子
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

Families Citing this family (9)

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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
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

Citations (1)

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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

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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
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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113454221A (zh) * 2018-12-12 2021-09-28 迪克纳制药公司 含有三元环的双链核酸抑制剂分子
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

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Publication number Publication date
US10675295B2 (en) 2020-06-09
EP3645013A4 (en) 2021-07-21
IL295086A (en) 2022-09-01
JP2020529197A (ja) 2020-10-08
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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