JPWO2021020412A1 - - Google Patents

Info

Publication number
JPWO2021020412A1
JPWO2021020412A1 JP2021535370A JP2021535370A JPWO2021020412A1 JP WO2021020412 A1 JPWO2021020412 A1 JP WO2021020412A1 JP 2021535370 A JP2021535370 A JP 2021535370A JP 2021535370 A JP2021535370 A JP 2021535370A JP WO2021020412 A1 JPWO2021020412 A1 JP WO2021020412A1
Authority
JP
Japan
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
JP2021535370A
Other versions
JPWO2021020412A5 (ja
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.)
Filing date
Publication date
Application filed filed Critical
Publication of JPWO2021020412A1 publication Critical patent/JPWO2021020412A1/ja
Publication of JPWO2021020412A5 publication Critical patent/JPWO2021020412A5/ja
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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 N.A.
    • 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 N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12Y—ENZYMES
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/02—Aminoacyltransferases (2.3.2)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021535370A 2019-07-30 2020-07-29 Pending JPWO2021020412A1 (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2019140142 2019-07-30
PCT/JP2020/028960 WO2021020412A1 (ja) 2019-07-30 2020-07-29 Ｍｕｒｆ１を標的とする核酸医薬

Publications (2)

Publication Number Publication Date
JPWO2021020412A1 true JPWO2021020412A1 (ja) 2021-02-04
JPWO2021020412A5 JPWO2021020412A5 (ja) 2023-07-25

Family

ID=74229918

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021535370A Pending JPWO2021020412A1 (ja) 2019-07-30 2020-07-29

Country Status (4)

Country Link
US (1) US20220267778A1 (ja)
EP (1) EP4005602A1 (ja)
JP (1) JPWO2021020412A1 (ja)
WO (1) WO2021020412A1 (ja)

Family Cites Families (37)

* Cited by examiner, † Cited by third party
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FR2692265B1 (fr) 1992-05-25 1996-11-08 Centre Nat Rech Scient Composes biologiquement actifs de type phosphotriesters.
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
NZ503765A (en) 1997-09-12 2002-04-26 Exiqon As Bi-cyclic and tri-cyclic nucleotide analogues
CA2368135C (en) 1999-03-18 2010-06-08 Exiqon A/S Xylo-lna analogues
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
DK2354148T3 (da) 2002-02-13 2013-10-14 Takeshi Imanishi Nukleosidanaloger og oligonukleotiderivate omfattende nukleotidanalog deraf
CA2495037A1 (en) 2002-08-14 2004-02-26 Pharmacia Corporation Antisense modulation of acyl-coa synthetase 1 expression
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
US20070141009A1 (en) 2003-01-03 2007-06-21 Shaharyar Khan Sirna mediated post-transriptional gene silencing of genes involved in alopecia
ATE555118T1 (de) 2003-08-28 2012-05-15 Takeshi Imanishi Neue synthetische nukleidsäuren vom typ mit quervernetzter n-o-bindung
FR2894582B1 (fr) * 2005-12-12 2008-02-22 Fabre Pierre Dermo Cosmetique Sirna anti myosine va et depigmentation de la peau
ES2471978T3 (es) 2006-05-05 2014-06-27 Isis Pharmaceuticals, Inc. Compuestos y procedimientos para modular la expresión de ApoB
CA3144493A1 (en) 2006-10-03 2008-04-10 Arbutus Biopharma Corporation Lipid containing formulations
WO2009069313A1 (en) 2007-11-28 2009-06-04 National University Corporation Tokyo Medical And Dental University System for delivering nucleic acids for suppressing target gene expression by utilizing endogenous chylomicron
JP2011505798A (ja) 2007-12-07 2011-03-03 サンタリス ファーマ アー／エス Ｍｃｌ−１を調節するためのＲＮＡアンタゴニスト化合物
ES2605618T3 (es) 2008-03-31 2017-03-15 National Institute Of Advanced Industrial Science And Technology ARN de doble cadena, modificado con lípidos con elevado efecto de interferencia por ARN
CA2721183C (en) 2008-04-11 2019-07-16 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
JP2010184879A (ja) 2009-02-10 2010-08-26 Univ Of Tokyo 抗筋萎縮剤
US8541562B2 (en) 2009-10-29 2013-09-24 Osaka University Bridged artificial nucleoside and nucleotide
EP2327783A1 (en) 2009-11-27 2011-06-01 Universitätsklinikum Freiburg Pharmaceutical composition comprising miRNA-100 and its use in the modulation of blood vessel growth
WO2011123468A1 (en) 2010-03-29 2011-10-06 Alnylam Pharmaceuticals, Inc. Sirna therapy for transthyretin (ttr) related ocular amyloidosis
CA2801342A1 (en) 2010-06-04 2011-12-08 Eric N. Olson Regulation of metabolism by mir-378
US9290760B2 (en) 2010-09-15 2016-03-22 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
EA201490993A1 (ru) 2011-11-18 2014-09-30 Элнилэм Фармасьютикалз, Инк. МОДИФИЦИРОВАННЫЕ СРЕДСТВА РНКи
KR20220045091A (ko) 2011-11-18 2022-04-12 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴（ＴＴＲ） 관련 질병을 치료하기 위한 ＲＮＡｉ 제제, 조성 및 그의 사용방법
DK2790736T3 (en) * 2011-12-12 2018-05-07 Oncoimmunin Inc In vivo delivery of oligonucleotides
AU2012353330B2 (en) 2011-12-16 2018-04-19 National University Corporation Tokyo Medical And Dental University Chimeric double-stranded nucleic acid
EA030211B1 (ru) 2012-04-25 2018-07-31 Регьюлэс Терапьютикс Инк. Соединения на основе микрорнк и способы модулирования активности mir-21
US9163235B2 (en) 2012-06-21 2015-10-20 MiRagen Therapeutics, Inc. Inhibitors of the miR-15 family of micro-RNAs
WO2014112463A1 (ja) 2013-01-15 2014-07-24 国立大学法人大阪大学 スルホンアミド構造を有するヌクレオシドおよびヌクレオチド
US9315535B2 (en) 2013-02-18 2016-04-19 Shionogi & Co., Ltd. Nucleoside and nucleotide having nitrogen-containing heterocycle structure
WO2015105083A1 (ja) 2014-01-07 2015-07-16 塩野義製薬株式会社 アンチセンスオリゴヌクレオチド及び糖誘導体を含む二本鎖オリゴヌクレオチド
WO2015168661A1 (en) 2014-05-01 2015-11-05 Smith Larry J METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
US10264976B2 (en) * 2014-12-26 2019-04-23 The University Of Akron Biocompatible flavonoid compounds for organelle and cell imaging
JP7190794B2 (ja) 2017-03-29 2022-12-16 塩野義製薬株式会社 核酸医薬及び多分岐脂質の複合体

Also Published As

Publication number Publication date
EP4005602A1 (en) 2022-06-01
US20220267778A1 (en) 2022-08-25
WO2021020412A1 (ja) 2021-02-04

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