CN112996568A - 用于调节mir-10b活性的微小rna化合物和方法 - Google Patents

用于调节mir-10b活性的微小rna化合物和方法 Download PDF

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Publication number
CN112996568A
CN112996568A CN201980074354.1A CN201980074354A CN112996568A CN 112996568 A CN112996568 A CN 112996568A CN 201980074354 A CN201980074354 A CN 201980074354A CN 112996568 A CN112996568 A CN 112996568A
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nucleosides
certain embodiments
compound
subscript
modified oligonucleotide
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Chinese (zh)
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C·R·阿勒顿
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Regulus Therapeutics Inc
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Regulus Therapeutics Inc
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4164—1,3-Diazoles
    • A61K31/4188—1,3-Diazoles condensed with other heterocyclic ring systems, e.g. biotin, sorbinil
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K9/08—Solutions
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • 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
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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
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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 nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CN201980074354.1A 2018-11-13 2019-11-12 用于调节mir-10b活性的微小rna化合物和方法 Pending CN112996568A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862760546P 2018-11-13 2018-11-13
US62/760,546 2018-11-13
PCT/US2019/060841 WO2020102142A1 (en) 2018-11-13 2019-11-12 Microrna compounds and methods for modulating mir-10b activity

Publications (1)

Publication Number Publication Date
CN112996568A true CN112996568A (zh) 2021-06-18

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US (1) US20220096517A1 (enExample)
EP (1) EP3880310A1 (enExample)
JP (1) JP2022506958A (enExample)
KR (1) KR20210091732A (enExample)
CN (1) CN112996568A (enExample)
AU (1) AU2019381681A1 (enExample)
CA (1) CA3117981A1 (enExample)
MA (1) MA54226A (enExample)
TW (1) TW202031268A (enExample)
WO (1) WO2020102142A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11236337B2 (en) 2016-11-01 2022-02-01 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
US12319913B2 (en) 2018-10-31 2025-06-03 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11584932B2 (en) 2016-11-01 2023-02-21 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110224286A1 (en) * 2008-08-19 2011-09-15 Maine Institute For Human Genetics And Health MICRO RNA (MiRNA) AND NEUROFIBROMATOSIS TYPE 1: A ROLE IN DIAGNOSIS AND THERAPY
CN102802637A (zh) * 2009-06-12 2012-11-28 桑塔里斯制药公司 新的有力的抗apob反义化合物
US20130289093A1 (en) * 2012-04-25 2013-10-31 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-21 activity
WO2018106566A1 (en) * 2016-12-05 2018-06-14 Regulus Therapeutics Inc. Modified oligonucleotides for treatment of polycystic kidney disease

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3144493A1 (en) 2006-10-03 2008-04-10 Arbutus Biopharma Corporation Lipid containing formulations
US9364495B2 (en) * 2009-10-20 2016-06-14 Roche Innovation Center Copenhagen A/S Oral delivery of therapeutically effective LNA oligonucleotides
JP6153932B2 (ja) * 2011-09-07 2017-06-28 株式会社スリー・ディー・マトリックス 骨肉腫のためのマイクロｒｎａに基づく方法およびアッセイ

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110224286A1 (en) * 2008-08-19 2011-09-15 Maine Institute For Human Genetics And Health MICRO RNA (MiRNA) AND NEUROFIBROMATOSIS TYPE 1: A ROLE IN DIAGNOSIS AND THERAPY
CN102802637A (zh) * 2009-06-12 2012-11-28 桑塔里斯制药公司 新的有力的抗apob反义化合物
US20130289093A1 (en) * 2012-04-25 2013-10-31 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-21 activity
WO2018106566A1 (en) * 2016-12-05 2018-06-14 Regulus Therapeutics Inc. Modified oligonucleotides for treatment of polycystic kidney disease

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
G. GABRIAL Y ET AL.: "Human Glioma Growth is Controlled by MicroRNA-10b", CANCER RESEARCH, vol. 71, no. 10, pages 3565 - 3572 *
NADIYA M TEPLYUK ET AL.: "Therapeutic potential of targeting microRNA-10b in established intracranial glioblastoma:first stemps toward the clinic", EMBO MOLECULAR MEDICINE, vol. 8, no. 3, pages 268 - 287, XP055664156, DOI: 10.15252/emmm.201505495 *
YI LIU ET AL.: "MicroRNA-10b overexpression promotes non-small cell lung cancer cell proliferation and invasion", EUROPEAN JOURNAL OF MEDICAL RESEARCH, vol. 18, no. 1, pages 41, XP021168958, DOI: 10.1186/2047-783X-18-41 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11236337B2 (en) 2016-11-01 2022-02-01 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
US12319913B2 (en) 2018-10-31 2025-06-03 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer

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WO2020102142A1 (en) 2020-05-22
US20220096517A1 (en) 2022-03-31
AU2019381681A1 (en) 2021-05-27
CA3117981A1 (en) 2020-05-22
JP2022506958A (ja) 2022-01-17
TW202031268A (zh) 2020-09-01
KR20210091732A (ko) 2021-07-22
MA54226A (fr) 2021-09-22
EP3880310A1 (en) 2021-09-22

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