JP2022517742A - 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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JP2022517742A
JP2022517742A JP2021538378A JP2021538378A JP2022517742A JP 2022517742 A JP2022517742 A JP 2022517742A JP 2021538378 A JP2021538378 A JP 2021538378A JP 2021538378 A JP2021538378 A JP 2021538378A JP 2022517742 A JP2022517742 A JP 2022517742A
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seq
set forth
sequence set
antisense strand
oligonucleotide
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JPWO2020139764A5 (https=
JP2022517742A5 (https=
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マーク エイブラムス
ギリシュ チョプダ
ジヘ パク
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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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of 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]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2021538378A 2018-12-28 2019-12-20 Ｈｍｇｂ１発現を阻害するための組成物及び方法 Pending JP2022517742A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024067819A JP2024105308A (ja) 2018-12-28 2024-04-18 Ｈｍｇｂ１発現を阻害するための組成物及び方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862786287P 2018-12-28 2018-12-28
US62/786,287 2018-12-28
US201862787038P 2018-12-31 2018-12-31
US62/787,038 2018-12-31
US201962788111P 2019-01-03 2019-01-03
US62/788,111 2019-01-03
PCT/US2019/067883 WO2020139764A1 (en) 2018-12-28 2019-12-20 Compositions and methods for inhibiting hmgb1 expression

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JP2024067819A Division JP2024105308A (ja) 2018-12-28 2024-04-18 Ｈｍｇｂ１発現を阻害するための組成物及び方法

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JP2022517742A true JP2022517742A (ja) 2022-03-10
JPWO2020139764A5 JPWO2020139764A5 (https=) 2023-02-24
JP2022517742A5 JP2022517742A5 (https=) 2023-02-24

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JP2024067819A Withdrawn JP2024105308A (ja) 2018-12-28 2024-04-18 Ｈｍｇｂ１発現を阻害するための組成物及び方法

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US (1) US20220072024A1 (https=)
EP (1) EP3883581A4 (https=)
JP (2) JP2022517742A (https=)
KR (1) KR20210126004A (https=)
CN (1) CN113874025A (https=)
AU (1) AU2019417585A1 (https=)
BR (1) BR112021012516A2 (https=)
CA (1) CA3124664A1 (https=)
CL (2) CL2021001718A1 (https=)
IL (2) IL284327B2 (https=)
MX (1) MX2021007855A (https=)
SG (1) SG11202106857VA (https=)
WO (1) WO2020139764A1 (https=)

Cited By (1)

* 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

Families Citing this family (4)

* Cited by examiner, † Cited by third party
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
WO2023208023A1 (zh) * 2022-04-26 2023-11-02 上海拓界生物医药科技有限公司 氘代化学修饰和包含其的寡核苷酸
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
WO2024233864A2 (en) * 2023-05-10 2024-11-14 Dicerna Pharmaceuticals, Inc. Galnac-conjugated rnai oligonucleotides

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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
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
WO2017079227A1 (en) * 2015-11-05 2017-05-11 University Of Connecticut Compositions and methods for the treatment of liver fibrosis

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WO2007076200A2 (en) * 2005-11-28 2007-07-05 Medimmune, Inc. Antagonists of hmgb1 and/or rage and methods of use thereof
WO2007150071A1 (en) * 2006-06-23 2007-12-27 Myriad Genetics, Inc. Gene amplifications and deletions
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
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
KR102350647B1 (ko) * 2016-09-02 2022-01-14 다이서나 파마수이티컬, 인크. 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
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

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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
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
WO2017079227A1 (en) * 2015-11-05 2017-05-11 University Of Connecticut Compositions and methods for the treatment of liver fibrosis

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SCIENTIFIC REPORTS, vol. 5:16013, JPN6023042788, 2015, pages 1 - 13, ISSN: 0005343908 *
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Cited By (3)

* 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
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
US12503701B2 (en) 2022-06-24 2025-12-23 Novo Nordisk A/S Compositions and methods for inhibiting transmembrane serine protease 6 (TMPRSS6) expression

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MX2021007855A (es) 2021-10-26
CA3124664A1 (en) 2020-07-02
CN113874025A (zh) 2021-12-31
SG11202106857VA (en) 2021-07-29
EP3883581A4 (en) 2023-03-29
KR20210126004A (ko) 2021-10-19
WO2020139764A1 (en) 2020-07-02
CL2023002984A1 (es) 2024-03-08
CL2021001718A1 (es) 2022-02-18
IL284327A (en) 2021-08-31
IL313425A (en) 2024-08-01
US20220072024A1 (en) 2022-03-10
IL284327B2 (en) 2024-11-01
EP3883581A1 (en) 2021-09-29
BR112021012516A2 (pt) 2021-09-14
IL284327B1 (en) 2024-07-01
AU2019417585A1 (en) 2021-07-08
JP2024105308A (ja) 2024-08-06

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