JP7382919B2 - トランスフェリン受容体（ＴｆＲ）に対するＲＮＡアプタマー - Google Patents

トランスフェリン受容体（ＴｆＲ）に対するＲＮＡアプタマー Download PDF

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JP7382919B2
JP7382919B2 JP2020507679A JP2020507679A JP7382919B2 JP 7382919 B2 JP7382919 B2 JP 7382919B2 JP 2020507679 A JP2020507679 A JP 2020507679A JP 2020507679 A JP2020507679 A JP 2020507679A JP 7382919 B2 JP7382919 B2 JP 7382919B2
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acid compound
nucleotides
ribonucleic acid
cell
moiety
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JP2020532958A (ja
JP2020532958A5 (enExample
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ロッシ，ジョン・ジェイ
ヨーン，ソラー
ハビブ，ナギー
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City of Hope
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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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/001—Preparation for luminescence or biological staining
    • A61K49/0013—Luminescence
    • A61K49/0017—Fluorescence in vivo
    • A61K49/005—Fluorescence in vivo characterised by the carrier molecule carrying the fluorescent agent
    • A61K49/0052—Small organic molecules
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—Aptamers
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
JP2020507679A 2017-08-11 2018-08-10 トランスフェリン受容体（ＴｆＲ）に対するＲＮＡアプタマー Active JP7382919B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762544220P 2017-08-11 2017-08-11
US62/544,220 2017-08-11
US201862671604P 2018-05-15 2018-05-15
US62/671,604 2018-05-15
PCT/US2018/046343 WO2019033051A1 (en) 2017-08-11 2018-08-10 RNA APTAMERS AGAINST THE TRANSFERRIN RECEPTOR (TFR)

Publications (3)

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JP2020532958A JP2020532958A (ja) 2020-11-19
JP2020532958A5 JP2020532958A5 (enExample) 2021-09-16
JP7382919B2 true JP7382919B2 (ja) 2023-11-17

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US (1) US11427826B2 (enExample)
EP (1) EP3664814A4 (enExample)
JP (1) JP7382919B2 (enExample)
KR (1) KR20200068647A (enExample)
CN (2) CN111542326B (enExample)
AU (1) AU2018314236B2 (enExample)
CA (1) CA3072575A1 (enExample)
WO (1) WO2019033051A1 (enExample)

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WO2020208361A1 (en) * 2019-04-12 2020-10-15 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220396794A1 (en) 2019-06-04 2022-12-15 Apterna Limited APTAMERS AGAINST TRANSFERRIN RECEPTOR (TfR)
GB202019692D0 (en) 2020-12-14 2021-01-27 Apterna Ltd Aptamer-sirna fusions
US12162940B2 (en) 2021-05-05 2024-12-10 Immatics Biotechnologies Gmbh BMA031 antigen binding polypeptides
CN118879700B (zh) * 2024-07-18 2025-11-07 四川大学 具有sirt1基因靶向激活功能的框架核酸药物的制备及其用途

Citations (2)

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WO2016061386A1 (en) 2014-10-15 2016-04-21 City Of Hope Rna aptamers against transferrin receptor (tfr)
WO2016127216A1 (en) 2015-02-11 2016-08-18 Deakin University Epcam aptamers and conjugates thereof

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AU2008234019B2 (en) 2007-04-03 2014-05-29 Amgen Research (Munich) Gmbh Cross-species-specific bispecific binders
PT2155783E (pt) 2007-04-03 2013-11-07 Amgen Res Munich Gmbh Domínio de ligação específico inter-espécies
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CA3072575A1 (en) 2019-02-14
AU2018314236B2 (en) 2025-02-06
US11427826B2 (en) 2022-08-30
US20200172905A1 (en) 2020-06-04
WO2019033051A1 (en) 2019-02-14
EP3664814A1 (en) 2020-06-17
JP2020532958A (ja) 2020-11-19
CN111542326A (zh) 2020-08-14
CN111542326B (zh) 2024-06-04
EP3664814A4 (en) 2021-05-05
KR20200068647A (ko) 2020-06-15
AU2018314236A1 (en) 2020-03-19
CN118931907A (zh) 2024-11-12

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