JP2022527569A - 治療剤の標的化送達のための多価リガンドクラスター - Google Patents

治療剤の標的化送達のための多価リガンドクラスター Download PDF

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JP2022527569A
JP2022527569A JP2021559455A JP2021559455A JP2022527569A JP 2022527569 A JP2022527569 A JP 2022527569A JP 2021559455 A JP2021559455 A JP 2021559455A JP 2021559455 A JP2021559455 A JP 2021559455A JP 2022527569 A JP2022527569 A JP 2022527569A
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シャオ，ペンチェン・パトリック
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ミトセラピューティクス・エルエルシー
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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
    • A61K47/54—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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H1/00—Processes for the preparation of sugar derivatives
    • C07H1/02—Phosphorylation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/18—Acyclic radicals, substituted by carbocyclic rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic 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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
JP2021559455A 2019-03-21 2020-03-19 治療剤の標的化送達のための多価リガンドクラスター Pending JP2022527569A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962821628P 2019-03-21 2019-03-21
US62/821,628 2019-03-21
US201962952607P 2019-12-23 2019-12-23
US62/952,607 2019-12-23
PCT/US2020/023603 WO2020191183A1 (en) 2019-03-21 2020-03-19 Multivalent ligand clusters for targeted delivery of therapeutic agents

Publications (2)

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JP2022527569A true JP2022527569A (ja) 2022-06-02
JP2022527569A5 JP2022527569A5 (https=) 2023-03-28

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JP2021559455A Pending JP2022527569A (ja) 2019-03-21 2020-03-19 治療剤の標的化送達のための多価リガンドクラスター

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Country Link
US (1) US20220175934A1 (https=)
EP (1) EP3923989A1 (https=)
JP (1) JP2022527569A (https=)
CN (1) CN114206389A (https=)
AU (1) AU2020242043A1 (https=)
CA (1) CA3133629A1 (https=)
WO (1) WO2020191183A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230092615A1 (en) 2020-02-21 2023-03-23 Mitotherapeutix Llc Compositions and methods for inhibiting expressing of methylation-controlled j-protein (mcj)
CN115850358B (zh) * 2021-03-08 2026-04-24 南京吉迈生物技术有限公司 GalNAc簇亚磷酰胺和靶向治疗性核苷
AU2022299173A1 (en) * 2021-06-24 2024-01-18 Eli Lilly And Company Novel therapeutic delivery moieties and uses thereof
EP4359530A1 (en) * 2021-06-24 2024-05-01 Eli Lilly and Company Novel rna therapeutics and uses thereof
EP4380624A1 (en) * 2021-09-23 2024-06-12 Shanghai Argo Biopharmaceutical Co., Ltd. Multivalent ligand clusters with diamine scaffold for targeted delivery of therapeutic agents
IL312811A (en) 2021-11-16 2024-07-01 Shanghai Argo Biopharmaceutical Co Ltd Composition and method for inhibiting angiotensinogen (agt) protein expression
CA3235672A1 (en) 2021-11-29 2023-06-01 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of hepatitis b virus (hbv) protein
TW202345865A (zh) 2022-01-24 2023-12-01 大陸商上海舶望製藥有限公司 抑制LPA(Apo(a))蛋白表達的組合物和方法
WO2024104386A1 (zh) * 2022-11-16 2024-05-23 南京明德新药研发有限公司 一类含七元杂环的三齿缀合基团
CN116535449B (zh) * 2023-03-22 2025-09-16 华东理工大学 一种糖基没食子酸类衍生物及其制备方法与在制备乳腺癌靶向诊疗材料中的应用
CN116854753B (zh) * 2023-06-01 2025-12-09 北京炫景瑞医药科技有限公司 一种化合物、缀合物、组合物以及它们的用途
GB202311334D0 (en) * 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016522683A (ja) * 2013-05-01 2016-08-04 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｈｂｖおよびｔｔｒ発現を調節するための組成物および方法
WO2017111137A1 (ja) * 2015-12-22 2017-06-29 味の素株式会社 オリゴヌクレオチドの製造方法
WO2017178656A1 (en) * 2016-04-14 2017-10-19 Roche Innovation Center Copenhagen A/S TRITYL-MONO-GalNAc COMPOUNDS AND THEIR USE
WO2017177326A1 (en) * 2016-04-11 2017-10-19 Protiva Biotherapeutics, Inc. Targeted nucleic acid conjugate compositions
WO2018044350A1 (en) * 2016-09-02 2018-03-08 Arrowhead Pharmaceuticals, Inc Targeting ligands

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US3854480A (en) 1969-04-01 1974-12-17 Alza Corp Drug-delivery system
US4675189A (en) 1980-11-18 1987-06-23 Syntex (U.S.A.) Inc. Microencapsulation of water soluble active polypeptides
US4452775A (en) 1982-12-03 1984-06-05 Syntex (U.S.A.) Inc. Cholesterol matrix delivery system for sustained release of macromolecules
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US5211657A (en) 1988-11-07 1993-05-18 The United States Government As Represented By The Secretary Of The Department Of Health And Human Services Laminin a chain deduced amino acid sequence, expression vectors and active synthetic peptides
US5133974A (en) 1989-05-05 1992-07-28 Kv Pharmaceutical Company Extended release pharmaceutical formulations
US5407686A (en) 1991-11-27 1995-04-18 Sidmak Laboratories, Inc. Sustained release composition for oral administration of active ingredient
US5736152A (en) 1995-10-27 1998-04-07 Atrix Laboratories, Inc. Non-polymeric sustained release delivery system

Patent Citations (5)

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Publication number Priority date Publication date Assignee Title
JP2016522683A (ja) * 2013-05-01 2016-08-04 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｈｂｖおよびｔｔｒ発現を調節するための組成物および方法
WO2017111137A1 (ja) * 2015-12-22 2017-06-29 味の素株式会社 オリゴヌクレオチドの製造方法
WO2017177326A1 (en) * 2016-04-11 2017-10-19 Protiva Biotherapeutics, Inc. Targeted nucleic acid conjugate compositions
WO2017178656A1 (en) * 2016-04-14 2017-10-19 Roche Innovation Center Copenhagen A/S TRITYL-MONO-GalNAc COMPOUNDS AND THEIR USE
WO2018044350A1 (en) * 2016-09-02 2018-03-08 Arrowhead Pharmaceuticals, Inc Targeting ligands

Also Published As

Publication number Publication date
AU2020242043A1 (en) 2021-10-14
CA3133629A1 (en) 2020-09-24
CN114206389A (zh) 2022-03-18
EP3923989A1 (en) 2021-12-22
US20220175934A1 (en) 2022-06-09
WO2020191183A1 (en) 2020-09-24

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