JP2021500026A - βカテニン核酸阻害剤分子 - Google Patents

βカテニン核酸阻害剤分子 Download PDF

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Publication number
JP2021500026A
JP2021500026A JP2020521889A JP2020521889A JP2021500026A JP 2021500026 A JP2021500026 A JP 2021500026A JP 2020521889 A JP2020521889 A JP 2020521889A JP 2020521889 A JP2020521889 A JP 2020521889A JP 2021500026 A JP2021500026 A JP 2021500026A
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Japan
Prior art keywords
nucleic acid
seq
inhibitor molecule
catenin
acid inhibitor
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Ceased
Application number
JP2020521889A
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English (en)
Japanese (ja)
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JP2021500026A5 (enExample
Inventor
シャンティ ガネーシュ
シャンティ ガネーシュ
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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Application filed by Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of JP2021500026A publication Critical patent/JP2021500026A/ja
Publication of JP2021500026A5 publication Critical patent/JP2021500026A5/ja
Priority to JP2023132823A priority Critical patent/JP2023175693A/ja
Ceased legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/395Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/39558Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against tumor tissues, cells, antigens
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51Nanocapsules; Nanoparticles
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C07K16/2818Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily against CD28 or CD152
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dispersion Chemistry (AREA)
  • Dermatology (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
JP2020521889A 2017-10-18 2018-10-17 βカテニン核酸阻害剤分子 Ceased JP2021500026A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023132823A JP2023175693A (ja) 2017-10-18 2023-08-17 βカテニン核酸阻害剤分子

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762573999P 2017-10-18 2017-10-18
US62/573,999 2017-10-18
PCT/US2018/056317 WO2019079472A1 (en) 2017-10-18 2018-10-17 BETA-CATENIN NUCLEIC ACID INHIBITOR MOLECULE

Related Child Applications (1)

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JP2023132823A Division JP2023175693A (ja) 2017-10-18 2023-08-17 βカテニン核酸阻害剤分子

Publications (2)

Publication Number Publication Date
JP2021500026A true JP2021500026A (ja) 2021-01-07
JP2021500026A5 JP2021500026A5 (enExample) 2021-11-25

Family

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JP2020521889A Ceased JP2021500026A (ja) 2017-10-18 2018-10-17 βカテニン核酸阻害剤分子
JP2023132823A Pending JP2023175693A (ja) 2017-10-18 2023-08-17 βカテニン核酸阻害剤分子

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023132823A Pending JP2023175693A (ja) 2017-10-18 2023-08-17 βカテニン核酸阻害剤分子

Country Status (11)

Country Link
US (2) US11008571B2 (enExample)
EP (1) EP3672980A4 (enExample)
JP (2) JP2021500026A (enExample)
KR (1) KR20200078538A (enExample)
CN (1) CN111225922B (enExample)
AU (1) AU2018350983A1 (enExample)
BR (1) BR112020007443A2 (enExample)
CA (1) CA3076924A1 (enExample)
IL (1) IL273772A (enExample)
MX (1) MX2020004045A (enExample)
WO (1) WO2019079472A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023122316A2 (en) * 2021-12-23 2023-06-29 Aligos Therapeutics, Inc. SPLICE SWITCH OLIGONUCLEOTIDE (SSO) MOLECULES FOR TARGETING β-CATENIN AND USES THEREOF
WO2023122317A2 (en) * 2021-12-23 2023-06-29 Aligos Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (SINA) MOLECULES FOR TARGETING β-CATENIN AND USES THEREOF

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012512651A (ja) * 2008-12-18 2012-06-07 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 遺伝子発現の特異的阻害のための拡張dicer基質薬剤および方法
JP2013535212A (ja) * 2010-08-02 2013-09-12 メルク・シャープ・エンド・ドーム・コーポレイション 低分子干渉核酸(siNA)を用いたカテニン(カドヘリン結合型タンパク質)β1(CTNNB1)遺伝子発現のRNA干渉媒介性阻害
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
WO2017160983A1 (en) * 2016-03-16 2017-09-21 Dicerna Pharmaceuticals, Inc. Compositions and methods for the treatment of a beta-catenin-associated disease or disorder

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
ES2734743T3 (es) * 2010-07-06 2019-12-11 Dicerna Pharmaceuticals Inc Métodos y composiciones para la inhibición específica de beta-catenina por RNA bicatenario
FI3366775T4 (fi) * 2011-11-18 2025-11-14 Alnylam Pharmaceuticals Inc Modifioituja rnai-agensseja

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012512651A (ja) * 2008-12-18 2012-06-07 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 遺伝子発現の特異的阻害のための拡張dicer基質薬剤および方法
JP2013535212A (ja) * 2010-08-02 2013-09-12 メルク・シャープ・エンド・ドーム・コーポレイション 低分子干渉核酸(siNA)を用いたカテニン(カドヘリン結合型タンパク質)β1(CTNNB1)遺伝子発現のRNA干渉媒介性阻害
WO2016100401A1 (en) * 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
WO2017160983A1 (en) * 2016-03-16 2017-09-21 Dicerna Pharmaceuticals, Inc. Compositions and methods for the treatment of a beta-catenin-associated disease or disorder

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ACS OMEGA (2017) VOL.2, PP.2055-2064, JPN6022039259, ISSN: 0004877182 *
MOL. CANCER THER. (2016) VOL.15, NO.9, PP.1-12, JPN7022004460, ISSN: 0004877181 *

Also Published As

Publication number Publication date
EP3672980A1 (en) 2020-07-01
IL273772A (en) 2020-05-31
JP2023175693A (ja) 2023-12-12
MX2020004045A (es) 2020-07-20
EP3672980A4 (en) 2021-06-16
US20210238603A1 (en) 2021-08-05
WO2019079472A1 (en) 2019-04-25
CA3076924A1 (en) 2019-04-25
KR20200078538A (ko) 2020-07-01
CN111225922B (zh) 2023-07-18
US11008571B2 (en) 2021-05-18
US20200270606A1 (en) 2020-08-27
CN111225922A (zh) 2020-06-02
BR112020007443A2 (pt) 2020-10-20
AU2018350983A1 (en) 2020-04-09

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