CN111225922B - β连环蛋白核酸抑制剂分子 - Google Patents

β连环蛋白核酸抑制剂分子 Download PDF

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Publication number
CN111225922B
CN111225922B CN201880067973.3A CN201880067973A CN111225922B CN 111225922 B CN111225922 B CN 111225922B CN 201880067973 A CN201880067973 A CN 201880067973A CN 111225922 B CN111225922 B CN 111225922B
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nucleic acid
seq
catenin
inhibitor molecule
acid inhibitor
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Expired - Fee Related
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CN201880067973.3A
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Chinese (zh)
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CN111225922A (zh
Inventor
S.加内什
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • 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
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/39558—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against tumor tissues, cells, antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules 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
    • A61K9/51—Nanocapsules; Nanoparticles
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [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
    • C07K16/2818—Immunoglobulins [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
    • C—CHEMISTRY; METALLURGY
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    • 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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Chemical & Material Sciences (AREA)
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  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • 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)
CN201880067973.3A 2017-10-18 2018-10-17 β连环蛋白核酸抑制剂分子 Expired - Fee Related CN111225922B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762573999P 2017-10-18 2017-10-18
US62/573999 2017-10-18
PCT/US2018/056317 WO2019079472A1 (en) 2017-10-18 2018-10-17 BETA-CATENIN NUCLEIC ACID INHIBITOR MOLECULE

Publications (2)

Publication Number Publication Date
CN111225922A CN111225922A (zh) 2020-06-02
CN111225922B true CN111225922B (zh) 2023-07-18

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CN201880067973.3A Expired - Fee Related CN111225922B (zh) 2017-10-18 2018-10-17 β连环蛋白核酸抑制剂分子

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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 (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105861503A (zh) * 2011-11-18 2016-08-17 阿尔尼拉姆医药品有限公司 修饰的RNAi试剂
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 (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2009336191B2 (en) * 2008-12-18 2017-08-24 Novo Nordisk A/S Extended dicer substrate agents and methods for the specific inhibition of gene expression
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
CN103068980B (zh) * 2010-08-02 2017-04-05 瑟纳治疗公司 使用短干扰核酸（siNA）的RNA干扰介导的联蛋白（钙粘蛋白关联蛋白质），β1（CTNNB1）基因表达的抑制
EP3865576A1 (en) * 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105861503A (zh) * 2011-11-18 2016-08-17 阿尔尼拉姆医药品有限公司 修饰的RNAi试剂
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 (1)

* Cited by examiner, † Cited by third party
Title
Direct pharmacological inhibition of β-catenin by RNA interference in tumors of diverse origin;Shanthi Ganesh等;《Mol Cancer Ther》;20160930;第15卷(第9期);第2014-2154页 *

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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
JP2021500026A (ja) 2021-01-07
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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