JP7038045B2 - ホスホロチオエート化オリゴデオキシヌクレオチドを含む化合物及び組成物、ならびにその使用方法 - Google Patents

ホスホロチオエート化オリゴデオキシヌクレオチドを含む化合物及び組成物、ならびにその使用方法 Download PDF

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JP7038045B2
JP7038045B2 JP2018519358A JP2018519358A JP7038045B2 JP 7038045 B2 JP7038045 B2 JP 7038045B2 JP 2018519358 A JP2018519358 A JP 2018519358A JP 2018519358 A JP2018519358 A JP 2018519358A JP 7038045 B2 JP7038045 B2 JP 7038045B2
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mir
cells
unsubstituted
inhibitor
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JP2018530589A5 (enExample
JP2018530589A (ja
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コルティレフスキ，マルチン・トマシュ
シフィデルスキ，ピオトル・マレク
マルクッチ，グイド
ジャン，ビン
クオ，ヤー－フーエイ
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City of Hope
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    • 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
    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
    • 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
    • A61K31/7004—Monosaccharides having only carbon, hydrogen and oxygen atoms
    • 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
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/704—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
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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]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/17—Immunomodulatory nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid

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  • Animal Behavior & Ethology (AREA)
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  • Physics & Mathematics (AREA)
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  • Oncology (AREA)
  • Hematology (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)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2018519358A 2015-10-15 2016-10-14 ホスホロチオエート化オリゴデオキシヌクレオチドを含む化合物及び組成物、ならびにその使用方法 Active JP7038045B2 (ja)

Priority Applications (1)

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JP2022033235A JP2022081575A (ja) 2015-10-15 2022-03-04 ホスホロチオエート化オリゴデオキシヌクレオチドを含む化合物及び組成物、ならびにその使用方法

Applications Claiming Priority (3)

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US201562242189P 2015-10-15 2015-10-15
US62/242,189 2015-10-15
PCT/US2016/057143 WO2017066639A1 (en) 2015-10-15 2016-10-14 Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof

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JP2018530589A JP2018530589A (ja) 2018-10-18
JP2018530589A5 JP2018530589A5 (enExample) 2019-11-21
JP7038045B2 true JP7038045B2 (ja) 2022-03-17

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JP2022033235A Pending JP2022081575A (ja) 2015-10-15 2022-03-04 ホスホロチオエート化オリゴデオキシヌクレオチドを含む化合物及び組成物、ならびにその使用方法

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US (4) US10801026B2 (enExample)
EP (2) EP3985116A1 (enExample)
JP (2) JP7038045B2 (enExample)
CN (1) CN108367021A (enExample)
CA (1) CA3001723A1 (enExample)
WO (1) WO2017066639A1 (enExample)

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CA2968531A1 (en) 2014-11-21 2016-05-26 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
EP3985116A1 (en) 2015-10-15 2022-04-20 City of Hope Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
WO2018039629A2 (en) 2016-08-25 2018-03-01 Northwestern University Micellar spherical nucleic acids from thermoresponsive, traceless templates
WO2018209270A1 (en) 2017-05-11 2018-11-15 Northwestern University Adoptive cell therapy using spherical nucleic acids (snas)
WO2019241766A1 (en) 2018-06-15 2019-12-19 City Of Hope Oligonucleotide-based proteolysis targeting chimera
WO2020041691A1 (en) * 2018-08-24 2020-02-27 City Of Hope Oligonucleotide inhibitors of nuclear factor kappa-light-chain-enhancer
US20220168331A1 (en) * 2019-02-18 2022-06-02 Duke University PRR-Activating and MicroRNA-Inhibiting Molecules and Methods of Using Same
US11801266B2 (en) * 2019-03-05 2023-10-31 City Of Hope Methods of using anti-MIR126 compounds
EP4065716A4 (en) * 2019-11-26 2024-06-12 City of Hope MICRORNA AS A THERAPEUTIC
US20250195666A1 (en) * 2021-05-20 2025-06-19 City Of Hope Mir-142 compounds and uses thereof

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US20120295963A1 (en) 2007-03-07 2012-11-22 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Agents, compositions and methods for treating pathologies in which regulating an ache-associated biological pathway is beneficial
US20140004154A1 (en) 2010-12-16 2014-01-02 Steve Pascolo Pharmaceutical Composition Consisting Of RNA Having Alkali Metal As Counter Ion And Formulated With Dications
JP2014527819A (ja) 2011-09-14 2014-10-23 ラナ セラピューティクス インコーポレイテッド 多量体オリゴヌクレオチド化合物

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WO2008116267A1 (en) 2007-03-26 2008-10-02 Crc For Asthma And Airways Ltd Therapeutic targets and molecules
US20140004154A1 (en) 2010-12-16 2014-01-02 Steve Pascolo Pharmaceutical Composition Consisting Of RNA Having Alkali Metal As Counter Ion And Formulated With Dications
JP2014527819A (ja) 2011-09-14 2014-10-23 ラナ セラピューティクス インコーポレイテッド 多量体オリゴヌクレオチド化合物

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JP2022081575A (ja) 2022-05-31
US20180312837A1 (en) 2018-11-01
CA3001723A1 (en) 2017-04-20
US20210115443A1 (en) 2021-04-22
CN108367021A (zh) 2018-08-03
US11591596B2 (en) 2023-02-28
US20230340479A1 (en) 2023-10-26
EP3985116A1 (en) 2022-04-20
JP2018530589A (ja) 2018-10-18
EP3364984B1 (en) 2021-11-17
EP3364984A1 (en) 2018-08-29
EP3364984A4 (en) 2019-09-25
US10801026B2 (en) 2020-10-13
US12227745B2 (en) 2025-02-18
US20250207135A1 (en) 2025-06-26
WO2017066639A1 (en) 2017-04-20

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