CN118931907A - 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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Publication number
CN118931907A
CN118931907A CN202410700060.8A CN202410700060A CN118931907A CN 118931907 A CN118931907 A CN 118931907A CN 202410700060 A CN202410700060 A CN 202410700060A CN 118931907 A CN118931907 A CN 118931907A
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nucleotides
cell
acid compound
compound
nucleic acid
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Pending
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CN202410700060.8A
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English (en)
Chinese (zh)
Inventor
J·J·罗西
S·尹
N·哈比
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Apterna Ltd
City of Hope
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Apterna Ltd
City of Hope
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Publication of CN118931907A publication Critical patent/CN118931907A/zh
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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
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/16Aptamers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (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)
CN202410700060.8A 2017-08-11 2018-08-10 转铁蛋白受体(TfR)的RNA适体 Pending CN118931907A (zh)

Applications Claiming Priority (6)

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

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201880066428.2A Division CN111542326B (zh) 2017-08-11 2018-08-10 转铁蛋白受体(TfR)的RNA适体

Publications (1)

Publication Number Publication Date
CN118931907A true CN118931907A (zh) 2024-11-12

Family

ID=65271776

Family Applications (2)

Application Number Title Priority Date Filing Date
CN202410700060.8A Pending CN118931907A (zh) 2017-08-11 2018-08-10 转铁蛋白受体(TfR)的RNA适体
CN201880066428.2A Active CN111542326B (zh) 2017-08-11 2018-08-10 转铁蛋白受体(TfR)的RNA适体

Family Applications After (1)

Application Number Title Priority Date Filing Date
CN201880066428.2A Active CN111542326B (zh) 2017-08-11 2018-08-10 转铁蛋白受体(TfR)的RNA适体

Country Status (8)

Country Link
US (1) US11427826B2 (https=)
EP (1) EP3664814A4 (https=)
JP (1) JP7382919B2 (https=)
KR (1) KR20200068647A (https=)
CN (2) CN118931907A (https=)
AU (1) AU2018314236B2 (https=)
CA (1) CA3072575A1 (https=)
WO (1) WO2019033051A1 (https=)

Families Citing this family (6)

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Publication number Priority date Publication date Assignee Title
EP3953473A1 (en) * 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
WO2020245198A1 (en) 2019-06-04 2020-12-10 Apterna Limited APTAMERS AGAINST TRANSFERRIN RECEPTOR (TfR)
GB202019692D0 (en) 2020-12-14 2021-01-27 Apterna Ltd Aptamer-sirna fusions
IL308258A (en) 2021-05-05 2024-01-01 Immatics Biotechnologies Gmbh Bma031 antigen binding polypeptides
WO2026011165A1 (en) * 2024-07-03 2026-01-08 Creyon Bio, Inc. Transferrin receptor (tfr) aptamers and use thereof
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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Publication number Publication date
US20200172905A1 (en) 2020-06-04
CN111542326B (zh) 2024-06-04
KR20200068647A (ko) 2020-06-15
CA3072575A1 (en) 2019-02-14
EP3664814A4 (en) 2021-05-05
EP3664814A1 (en) 2020-06-17
US11427826B2 (en) 2022-08-30
WO2019033051A1 (en) 2019-02-14
CN111542326A (zh) 2020-08-14
AU2018314236A1 (en) 2020-03-19
JP7382919B2 (ja) 2023-11-17
AU2018314236B2 (en) 2025-02-06
JP2020532958A (ja) 2020-11-19

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