CN111542326B - 转铁蛋白受体(TfR)的RNA适体 - Google Patents

转铁蛋白受体(TfR)的RNA适体 Download PDF

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CN111542326B
CN111542326B CN201880066428.2A CN201880066428A CN111542326B CN 111542326 B CN111542326 B CN 111542326B CN 201880066428 A CN201880066428 A CN 201880066428A CN 111542326 B CN111542326 B CN 111542326B
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nucleotides
moiety
cell
compound
nucleic acid
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CN111542326A (zh
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J·J·罗西
S·尹
N·哈比
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Apterna Ltd
City of Hope
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City of Hope
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    • 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
    • C12N15/115Aptamers, 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
    • 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
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/001Preparation for luminescence or biological staining
    • A61K49/0013Luminescence
    • A61K49/0017Fluorescence in vivo
    • A61K49/005Fluorescence in vivo characterised by the carrier molecule carrying the fluorescent agent
    • A61K49/0052Small organic molecules
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/16Aptamers
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
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    • C12N2310/3519Fusion with another nucleic acid
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific

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  • 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)
CN201880066428.2A 2017-08-11 2018-08-10 转铁蛋白受体(TfR)的RNA适体 Active CN111542326B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202410700060.8A CN118931907A (zh) 2017-08-11 2018-08-10 转铁蛋白受体(TfR)的RNA适体

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)

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CN111542326A CN111542326A (zh) 2020-08-14
CN111542326B true CN111542326B (zh) 2024-06-04

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CN202410700060.8A Pending CN118931907A (zh) 2017-08-11 2018-08-10 转铁蛋白受体(TfR)的RNA适体

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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)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
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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

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WO2016127216A1 (en) * 2015-02-11 2016-08-18 Deakin University Epcam aptamers and conjugates thereof
WO2016161165A1 (en) * 2015-03-31 2016-10-06 City Of Hope Anti-cancer rna aptamers

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Publication number Publication date
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
EP3664814A4 (en) 2021-05-05
JP7382919B2 (ja) 2023-11-17
KR20200068647A (ko) 2020-06-15
AU2018314236A1 (en) 2020-03-19
CN118931907A (zh) 2024-11-12

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