JP2023526277A - 核酸リガンドコンジュゲートおよび細胞への送達のためのその使用 - Google Patents

核酸リガンドコンジュゲートおよび細胞への送達のためのその使用 Download PDF

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JP2023526277A
JP2023526277A JP2022569012A JP2022569012A JP2023526277A JP 2023526277 A JP2023526277 A JP 2023526277A JP 2022569012 A JP2022569012 A JP 2022569012A JP 2022569012 A JP2022569012 A JP 2022569012A JP 2023526277 A JP2023526277 A JP 2023526277A
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nucleic acid
conjugate product
linker
polypeptide
conjugate
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JP2023526277A5 (https=
Inventor
ペコット，チャド
アザム，サルマ・エイチ．
バウワーズ，アルバート
フレミング，マシュー・シリル
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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
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Publication of JP2023526277A publication Critical patent/JP2023526277A/ja
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    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • 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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • 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
    • 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]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; 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)
JP2022569012A 2020-05-13 2021-05-12 核酸リガンドコンジュゲートおよび細胞への送達のためのその使用 Pending JP2023526277A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (2)

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JP2023526277A true JP2023526277A (ja) 2023-06-21
JP2023526277A5 JP2023526277A5 (https=) 2024-05-17

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JP2022569012A Pending JP2023526277A (ja) 2020-05-13 2021-05-12 核酸リガンドコンジュゲートおよび細胞への送達のためのその使用

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

Families Citing this family (3)

* 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
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

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH03500530A (ja) * 1987-06-11 1991-02-07 シンセティック　ジェネティクス 新規な両親媒性核酸接合体
JP2012500191A (ja) * 2008-08-15 2012-01-05 ジーイー・ヘルスケア・アクスイェ・セルスカプ 異形成の検出方法
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
US20180223284A1 (en) * 2017-02-06 2018-08-09 Mpeg La, Llc Multimeric oligonucleotides having decreased kidney clearance
WO2018152244A1 (en) * 2017-02-14 2018-08-23 Seqwell, Inc. Compositions and methods for sequencing nucleic acids
JP2018138588A (ja) * 2013-04-28 2018-09-06 秦剛 新規リンカー、その製造方法およびその応用
JP2019531697A (ja) * 2016-07-01 2019-11-07 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 標的化された細胞表面編集のための複合体

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008079973A2 (en) * 2006-12-21 2008-07-03 Centocor, Inc. Egfr binding peptides and uses thereof
WO2009002440A2 (en) * 2007-06-22 2008-12-31 Intradigm Corporation Compositions comprising human egfr-sirna and methods of use
RU2010107199A (ru) * 2007-07-31 2011-09-10 Дзе Джонс Хопкинс Юниверсити (Us) Конъюгат полипептид-нуклеиновая кислота для иммунопрофилактики или иммунотерапии для неопластических или инфекционных нарушений
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 (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH03500530A (ja) * 1987-06-11 1991-02-07 シンセティック　ジェネティクス 新規な両親媒性核酸接合体
JP2012500191A (ja) * 2008-08-15 2012-01-05 ジーイー・ヘルスケア・アクスイェ・セルスカプ 異形成の検出方法
JP2018138588A (ja) * 2013-04-28 2018-09-06 秦剛 新規リンカー、その製造方法およびその応用
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
JP2019531697A (ja) * 2016-07-01 2019-11-07 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 標的化された細胞表面編集のための複合体
US20180223284A1 (en) * 2017-02-06 2018-08-09 Mpeg La, Llc Multimeric oligonucleotides having decreased kidney clearance
WO2018152244A1 (en) * 2017-02-14 2018-08-23 Seqwell, Inc. Compositions and methods for sequencing nucleic acids

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ACS BIOMATER. SCI. ENG., vol. 6, JPN6025014870, January 2020 (2020-01-01), pages 1074 - 1089, ISSN: 0005807167 *
MOL. PHARMACEUTICS, vol. 11, JPN6025014871, 2014, pages 3242 - 3250, ISSN: 0005807168 *

Also Published As

Publication number Publication date
EP4149557A1 (en) 2023-03-22
WO2021231518A1 (en) 2021-11-18
CA3174467A1 (en) 2021-11-18
CN115697416A (zh) 2023-02-03
CN121775155A (zh) 2026-04-03
EP4149557A4 (en) 2025-01-15
US20230285579A1 (en) 2023-09-14

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