CN115697416A - 核酸配体偶联物及其用于向细胞递送的用途 - Google Patents

核酸配体偶联物及其用于向细胞递送的用途 Download PDF

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Publication number
CN115697416A
CN115697416A CN202180037230.3A CN202180037230A CN115697416A CN 115697416 A CN115697416 A CN 115697416A CN 202180037230 A CN202180037230 A CN 202180037230A CN 115697416 A CN115697416 A CN 115697416A
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Prior art keywords
nucleic acid
coupling product
linker
polypeptide
cancer
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CN202180037230.3A
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English (en)
Chinese (zh)
Inventor
C·佩科特
S·H·阿扎姆
A·鲍尔斯
M·C·费莱明
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University of North Carolina at Chapel Hill
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University of North Carolina at Chapel Hill
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Application filed by University of North Carolina at Chapel Hill filed Critical University of North Carolina at Chapel Hill
Priority to CN202610037779.7A priority Critical patent/CN121775155A/zh
Publication of CN115697416A publication Critical patent/CN115697416A/zh
Pending legal-status Critical Current

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    • 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
    • A61K47/62Medicinal 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 a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/08Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • 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
    • 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
    • C12N15/113Non-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
    • C12N15/1135Non-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 against oncogenes or tumor suppressor genes
    • 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
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/3513Protein; Peptide

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Oncology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
CN202180037230.3A 2020-05-13 2021-05-12 核酸配体偶联物及其用于向细胞递送的用途 Pending CN115697416A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202610037779.7A CN121775155A (zh) 2020-05-13 2021-05-12 核酸配体偶联物及其用于向细胞递送的用途

Applications Claiming Priority (3)

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US202063024142P 2020-05-13 2020-05-13
US63/024,142 2020-05-13
PCT/US2021/031903 WO2021231518A1 (en) 2020-05-13 2021-05-12 Nucleic acid ligand conjugates and use thereof for delivery to cells

Related Child Applications (1)

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CN202610037779.7A Division CN121775155A (zh) 2020-05-13 2021-05-12 核酸配体偶联物及其用于向细胞递送的用途

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CN115697416A true CN115697416A (zh) 2023-02-03

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CN202610037779.7A Pending CN121775155A (zh) 2020-05-13 2021-05-12 核酸配体偶联物及其用于向细胞递送的用途

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US (1) US20230285579A1 (https=)
EP (1) EP4149557A4 (https=)
JP (1) JP2023526277A (https=)
CN (2) CN115697416A (https=)
CA (1) CA3174467A1 (https=)
WO (1) WO2021231518A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117865868A (zh) * 2023-05-19 2024-04-12 湖南大学 一种功能化分子、其制备方法及其应用

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024102864A2 (en) * 2022-11-08 2024-05-16 Purdue Research Foundation Multivalent multispecific conjugates and related compositions and methods of use
EP4687988A2 (en) * 2023-03-28 2026-02-11 KIST (Korea Institute of Science and Technology) Therapeutic compounds for inhibiting and reducing the expression of cell surface proteins

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100151003A1 (en) * 2006-12-21 2010-06-17 Mohit Trikha Egfr binding peptides and uses thereof
CN102317303A (zh) * 2007-07-31 2012-01-11 约翰·霍普金斯大学 用于肿瘤或感染性疾病的免疫预防或免疫治疗的多肽-核酸偶联物

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US4904582A (en) * 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
WO2009002440A2 (en) * 2007-06-22 2008-12-31 Intradigm Corporation Compositions comprising human egfr-sirna and methods of use
GB0814960D0 (en) * 2008-08-15 2008-09-24 Ge Healthcare As Method for detecting dysplasia
CN105722851A (zh) * 2013-04-28 2016-06-29 秦刚 一种新型的连接子及其制备方法和用途
WO2017127473A1 (en) * 2016-01-19 2017-07-27 The University Of North Carolina At Chapel Hill Methods and compositions using rna interference for inhibition of kras
CN109641038A (zh) * 2016-07-01 2019-04-16 小利兰·斯坦福大学理事会 靶向细胞表面编辑的共轭物
SG11201906728TA (en) * 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance
US20200002746A1 (en) * 2017-02-14 2020-01-02 Seqwell, Inc. Compositions and methods for sequencing nucleic acids
KR102353086B1 (ko) * 2018-09-07 2022-01-20 아주대학교산학협력단 신규 면역독소 제조방법
JP7839554B2 (ja) * 2020-04-07 2026-04-02 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル Krasの阻害のためのrna干渉およびアンチセンスオリゴヌクレオチドを使用する方法および組成物

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100151003A1 (en) * 2006-12-21 2010-06-17 Mohit Trikha Egfr binding peptides and uses thereof
CN102317303A (zh) * 2007-07-31 2012-01-11 约翰·霍普金斯大学 用于肿瘤或感染性疾病的免疫预防或免疫治疗的多肽-核酸偶联物

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KOLDO URBIOLA ET AL.: "Novel PAMAM-PEG-Peptide Conjugates for siRNA Delivery Targeted to th e Transferrin and Epidermal Growth Factor Receptors", J PERS MED., 9 January 2018 (2018-01-09) *
孙立春 等: "受体靶向多肽载体抗肿瘤药物", 上海医药, 10 January 2015 (2015-01-10) *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117865868A (zh) * 2023-05-19 2024-04-12 湖南大学 一种功能化分子、其制备方法及其应用
CN117865868B (zh) * 2023-05-19 2024-09-06 湖南大学 一种功能化分子、其制备方法及其应用

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Publication number Publication date
EP4149557A1 (en) 2023-03-22
WO2021231518A1 (en) 2021-11-18
CA3174467A1 (en) 2021-11-18
CN121775155A (zh) 2026-04-03
EP4149557A4 (en) 2025-01-15
US20230285579A1 (en) 2023-09-14
JP2023526277A (ja) 2023-06-21

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