AU2018324345B2 - Cationic lipid compositions for tissue-specific delivery - Google Patents

Cationic lipid compositions for tissue-specific delivery Download PDF

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Publication number
AU2018324345B2
AU2018324345B2 AU2018324345A AU2018324345A AU2018324345B2 AU 2018324345 B2 AU2018324345 B2 AU 2018324345B2 AU 2018324345 A AU2018324345 A AU 2018324345A AU 2018324345 A AU2018324345 A AU 2018324345A AU 2018324345 B2 AU2018324345 B2 AU 2018324345B2
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molar ratio
lipid
compositional molar
cationic lipid
helper
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AU2018324345A
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AU2018324345A1 (en
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Xavier De Mollerat Du Jeu
Shikha Mishra
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Life Technologies Corp
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Life Technologies Corp
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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
    • A61K47/69—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
    • A61K47/6917—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 lipoprotein vesicle, e.g. HDL or LDL proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/541—Organic ions forming an ion pair complex with the pharmacologically or 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pulmonology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
AU2018324345A 2017-08-31 2018-07-17 Cationic lipid compositions for tissue-specific delivery Active AU2018324345B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762552783P 2017-08-31 2017-08-31
US62/552,783 2017-08-31
PCT/US2018/042555 WO2019045897A1 (en) 2017-08-31 2018-07-17 CATIONIC LIPIDIC COMPOSITIONS FOR TISSUE-SPECIFIC ADMINISTRATION

Publications (2)

Publication Number Publication Date
AU2018324345A1 AU2018324345A1 (en) 2020-03-05
AU2018324345B2 true AU2018324345B2 (en) 2024-06-27

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AU2018324345A Active AU2018324345B2 (en) 2017-08-31 2018-07-17 Cationic lipid compositions for tissue-specific delivery

Country Status (8)

Country Link
US (3) US20190060482A1 (enExample)
EP (1) EP3675826B1 (enExample)
JP (2) JP7379325B2 (enExample)
CN (1) CN111065377A (enExample)
AU (1) AU2018324345B2 (enExample)
CA (1) CA3073851A1 (enExample)
LT (1) LT3675826T (enExample)
WO (1) WO2019045897A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11690921B2 (en) * 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
PL4223886T3 (pl) 2019-04-16 2025-03-31 Genfit Kompozycje i sposoby stabilizacji mikro-rna
CA3145425A1 (en) * 2019-07-03 2021-01-07 Factor Bioscience Inc. Cationic lipids and uses thereof
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
EP3865122A1 (en) * 2020-02-11 2021-08-18 Pantherna Therapeutics GmbH Lipid composition and use thereof for delivery of a therapeutically active agent to endothelium
CN119604312A (zh) 2022-06-30 2025-03-11 生命技术公司 用于体内递送的脂质组合物
EP4642434A2 (en) 2022-12-29 2025-11-05 Life Technologies Corporation Lipid compositions on the basis of ionizable lipids, use for delivery to immune cells
CN119997938A (zh) * 2023-06-05 2025-05-13 安徽医科大学第一附属医院 肺靶向纳米胶束、制备方法及应用
TW202543585A (zh) 2024-03-08 2025-11-16 美商健臻公司 脂質奈米粒子
WO2026030375A2 (en) 2024-07-30 2026-02-05 Genzyme Corporation Lipid nanoparticles and methods of manufacture and use thereof
CN118576568B (zh) * 2024-08-06 2024-11-26 北京悦康科创医药科技股份有限公司 一种药物组合物及其制备方法和应用

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US6028077A (en) 1997-04-15 2000-02-22 Rinehart; Kenneth L. Crambescidin compounds
NZ512244A (en) 1998-11-12 2003-12-19 Invitrogen Corp Polycationic transfection reagents for introducing anions into a cell
EP1341497A4 (en) 2000-11-02 2005-10-19 Smithkline Beecham Corp RECEPTOR ANTAGONIST LIPID CONJUGATES AND DISPOSABLE SUPPLEMENTS CONTAINING THEM
WO2003053408A1 (en) 2001-12-19 2003-07-03 Alza Corporation Liposomal tumor necrosis factor compositions
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
US20130189364A1 (en) 2004-07-09 2013-07-25 Robert Sabin Compositions and methods of potentiating adjuvant pharmaceuticals targeting latent viral infections
GB0418172D0 (en) 2004-08-13 2004-09-15 Ic Vec Ltd Vector
WO2009111088A2 (en) * 2008-01-02 2009-09-11 The Johns Hopkins University Antitumor immunization by liposomal delivery of vaccine to the spleen
AP2015008874A0 (en) * 2009-08-14 2015-11-30 Alnylam Pharmaceuticals Inc Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
CA3009891C (en) * 2009-12-23 2020-09-15 Novartis Ag Lipids, lipid compositions, and methods of using them
CN103380113B (zh) * 2010-11-15 2018-03-30 生命科技公司 含胺的转染试剂及其制备和使用方法
RU2013154295A (ru) * 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
GR1008018B (el) 2012-09-06 2013-10-29 Ιδρυμα Ιατροβιολογικων Ερευνων Ακαδημιας Αθηνων, Λιπιδικα συμπλοκα αποτελουμενα απο ανιονικα λυσο-λιπιδια και η εφαρμογη αυτων
CN103296033B (zh) 2013-05-28 2016-05-11 京东方科技集团股份有限公司 一种阵列基板及其制作方法
EP3019595A4 (en) * 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
BR112016013516A8 (pt) 2013-12-12 2020-05-19 Life Technologies Corp complexo de transfecção e seu uso para aumentar transporte de molécula de carga em células e para o tratamento de câncer e terapia gênica, bem como método in vitro para fornecer um poliânion a uma célula
EP3169310A1 (en) * 2014-07-15 2017-05-24 Life Technologies Corporation Compositions with lipid aggregates and methods for efficient delivery of molecules to cells
WO2016205691A1 (en) 2015-06-19 2016-12-22 Massachusetts Institute Of Technology Alkenyl substituted 2,5-piperazinediones and their use in compositions for delivering an agent to a subject or cell
CN107849567B (zh) * 2015-06-26 2024-07-23 苏州瑞博生物技术股份有限公司 一种siRNA、含有该siRNA的药物组合物和缀合物及它们的应用
EP3362102A1 (en) 2015-10-14 2018-08-22 Life Technologies Corporation Ribonucleoprotein transfection agents
US11690921B2 (en) * 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
WO2020051223A1 (en) * 2018-09-04 2020-03-12 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids

Also Published As

Publication number Publication date
JP7379325B2 (ja) 2023-11-14
CA3073851A1 (en) 2019-03-07
AU2018324345A1 (en) 2020-03-05
CN111065377A (zh) 2020-04-24
LT3675826T (lt) 2023-09-11
US20260108623A1 (en) 2026-04-23
EP3675826A1 (en) 2020-07-08
JP2023138526A (ja) 2023-10-02
JP2020532546A (ja) 2020-11-12
WO2019045897A1 (en) 2019-03-07
US20220001023A1 (en) 2022-01-06
US20190060482A1 (en) 2019-02-28
EP3675826B1 (en) 2023-05-31
US12521447B2 (en) 2026-01-13

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