CN102985548B - 用于递送治疗分子的包含阳离子两亲物和辅脂质的组合物 - Google Patents

用于递送治疗分子的包含阳离子两亲物和辅脂质的组合物 Download PDF

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CN102985548B
CN102985548B CN201080031986.9A CN201080031986A CN102985548B CN 102985548 B CN102985548 B CN 102985548B CN 201080031986 A CN201080031986 A CN 201080031986A CN 102985548 B CN102985548 B CN 102985548B
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nucleic acid
composition
solution
cationic lipid
lipid
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CN102985548A (zh
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崔坤元
梁东
大卫·斯威德勒
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • A61K47/69—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0014—Skin, i.e. galenical aspects of topical compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
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  • Wood Science & Technology (AREA)
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  • Molecular Biology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Pulmonology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CN201080031986.9A 2009-05-16 2010-05-16 用于递送治疗分子的包含阳离子两亲物和辅脂质的组合物 Active CN102985548B (zh)

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Application Number Priority Date Filing Date Title
US17896209P 2009-05-16 2009-05-16
US61/178,962 2009-05-16
PCT/US2010/035049 WO2010135207A1 (en) 2009-05-16 2010-05-16 Compositions comprising cationic amphiphiles and colipids for delivering therapeutics molecules

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CN102985548A CN102985548A (zh) 2013-03-20
CN102985548B true CN102985548B (zh) 2016-10-26

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US (1) US9080186B2 (enExample)
EP (1) EP2430168B1 (enExample)
JP (1) JP5675784B2 (enExample)
CN (1) CN102985548B (enExample)
AU (1) AU2010249881B2 (enExample)
CA (1) CA2762095A1 (enExample)
WO (1) WO2010135207A1 (enExample)

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WO2024027809A1 (zh) 2022-08-05 2024-02-08 厦门甘宝利生物医药有限公司 一种抑制apoc3基因表达的rna抑制剂及其应用
WO2024088190A1 (zh) 2022-10-24 2024-05-02 厦门甘宝利生物医药有限公司 一种抑制lpa基因表达的rna抑制剂及其应用
WO2025113592A1 (zh) 2023-12-01 2025-06-05 厦门甘宝利生物医药有限公司 一种抑制补体系统基因表达的rna抑制剂及其应用

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US10253315B2 (en) 2012-08-30 2019-04-09 Commissariat A L'energie Atomique Et Aux Energies Alternatives Method for transfection of nucleic acids into eukaryotic cells in 3D scaffold
FR2994849B1 (fr) * 2012-08-30 2015-01-02 Commissariat Energie Atomique Formulation pour la delivrance de sequences nucleotidiques susceptibles de moduler des mecanismes endogenes d'arn interferents
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KR101671864B1 (ko) * 2014-08-29 2016-11-04 세종대학교 산학협력단 요오드화 오일을 함유하는 양이온성 에멀젼 및 바이러스 벡터의 유전자 전달 증진을 위한 이의 용도
CN104922141B (zh) * 2015-05-28 2016-05-25 厦门成坤生物技术有限公司 一种用于治疗乙型病毒性肝炎的siRNA组合物
CN105079021A (zh) * 2015-08-03 2015-11-25 厦门成坤生物技术有限公司 包含m10c1化合物与活性成分的组合物及其制备方法
HRP20220156T1 (hr) 2015-09-17 2022-04-15 Modernatx, Inc. Spojevi i pripravci za unutarstaničnu isporuku terapeutskih sredstava
RS63051B1 (sr) 2015-12-22 2022-04-29 Modernatx Inc Jedinjenja i kompozicije za intracelularnu isporuku agenasa
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WO2024027809A1 (zh) 2022-08-05 2024-02-08 厦门甘宝利生物医药有限公司 一种抑制apoc3基因表达的rna抑制剂及其应用
WO2024088190A1 (zh) 2022-10-24 2024-05-02 厦门甘宝利生物医药有限公司 一种抑制lpa基因表达的rna抑制剂及其应用
WO2025113592A1 (zh) 2023-12-01 2025-06-05 厦门甘宝利生物医药有限公司 一种抑制补体系统基因表达的rna抑制剂及其应用

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US20120289584A1 (en) 2012-11-15
AU2010249881B2 (en) 2015-01-22
EP2430168A4 (en) 2013-11-27
JP5675784B2 (ja) 2015-02-25
CN102985548A (zh) 2013-03-20
EP2430168A1 (en) 2012-03-21
WO2010135207A1 (en) 2010-11-25
US9080186B2 (en) 2015-07-14
AU2010249881A1 (en) 2012-01-19
CA2762095A1 (en) 2010-11-25
EP2430168B1 (en) 2016-12-21
JP2012526858A (ja) 2012-11-01

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