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DK3646854T3 - Sammensætninger til transfektion af mRNA i en celle og anvendelser deraf - Google Patents

Sammensætninger til transfektion af mRNA i en celle og anvendelser deraf Download PDF

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Publication number
DK3646854T3
DK3646854T3 DK18306417.9T DK18306417T DK3646854T3 DK 3646854 T3 DK3646854 T3 DK 3646854T3 DK 18306417 T DK18306417 T DK 18306417T DK 3646854 T3 DK3646854 T3 DK 3646854T3
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Denmark
Prior art keywords
transfection
mrna
compositions
cell
Prior art date
Application number
DK18306417.9T
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English (en)
Inventor
Fabrice Stock
Moreau Valérie Toussaint
Patrick Erbacher
Original Assignee
Polyplus Transfection
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Publication of DK3646854T3 publication Critical patent/DK3646854T3/da

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/0005Vertebrate antigens
    • A61K39/0011Cancer antigens
    • 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
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K47/56Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D233/00Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings
    • C07D233/54Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members
    • C07D233/56Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, attached to ring carbon atoms
    • C07D233/58Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, attached to ring carbon atoms with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, attached to ring nitrogen atoms
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53DNA (RNA) vaccination

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dispersion Chemistry (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Communicable Diseases (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
DK18306417.9T 2018-10-30 2018-10-30 Sammensætninger til transfektion af mRNA i en celle og anvendelser deraf DK3646854T3 (da)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP18306417.9A EP3646854B1 (en) 2018-10-30 2018-10-30 Compositions for transfecting mrna into a cell and their applications

Publications (1)

Publication Number Publication Date
DK3646854T3 true DK3646854T3 (da) 2022-09-26

Family

ID=64572258

Family Applications (1)

Application Number Title Priority Date Filing Date
DK18306417.9T DK3646854T3 (da) 2018-10-30 2018-10-30 Sammensætninger til transfektion af mRNA i en celle og anvendelser deraf

Country Status (13)

Country Link
US (1) US20210386841A1 (da)
EP (1) EP3646854B1 (da)
JP (1) JP7477504B2 (da)
KR (1) KR20210084511A (da)
CN (1) CN112930172B (da)
AU (1) AU2019373387B2 (da)
DK (1) DK3646854T3 (da)
ES (1) ES2927532T3 (da)
IL (1) IL282579B2 (da)
PL (1) PL3646854T3 (da)
PT (1) PT3646854T (da)
SG (1) SG11202103117RA (da)
WO (1) WO2020089342A1 (da)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230399390A1 (en) 2019-10-24 2023-12-14 Novago Therapeutics Ag Novel anti-nogo-a antibodies
JP2023527163A (ja) * 2020-05-19 2023-06-27 ラモット・アット・テル・アビブ・ユニバーシテイ・リミテッド 核酸送達用の脂質粒子およびその臨床応用
US20230242624A1 (en) 2020-06-02 2023-08-03 Neurimmune Ag HUMAN ANTIBODIES AGAINST SEVERE ACUTE RESPIRATORY SYNDROME CORONAVIRUS-2 (SARS-CoV-2)
WO2024003363A1 (en) * 2022-06-30 2024-01-04 Universiteit Gent Cationic amphiphilic compound-based nanoparticle compositions
CN119486721A (zh) 2022-07-08 2025-02-18 宝利普拉斯生物转染公司 包含基于咪唑鎓的阳离子脂质的脂质纳米颗粒

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5627159A (en) 1994-10-27 1997-05-06 Life Technologies, Inc. Enhancement of lipid cationic transfections in the presence of serum
FR2732895B1 (fr) * 1995-04-11 1997-05-16 Pasteur Merieux Serums Vacc Utilisation d'un compose amphipathique cationique comme agent de transfection, comme adjuvant de vaccin, ou comme medicament
EP1129064B1 (en) 1998-11-12 2008-01-09 Invitrogen Corporation Transfection reagents
US20040072769A1 (en) * 2002-09-16 2004-04-15 Yin James Qinwei Methods for design and selection of short double-stranded oligonucleotides, and compounds of gene drugs
EP1844772A1 (en) 2006-04-06 2007-10-17 Polyplus-Transfection SA Compositions for transfection of oligonucleotides active for gene silencing and their biological and therapeutical applications
EP2860255A1 (en) * 2013-10-14 2015-04-15 Technische Universität Graz Compositions comprising cationic and neutral lipids for transfecting nucleic acid molecules into eukaryotic cells
WO2016011203A1 (en) * 2014-07-15 2016-01-21 Life Technologies Corporation Compositions with lipid aggregates and methods for efficient delivery of molecules to cells
ES2969956T3 (es) * 2014-09-05 2024-05-23 Novartis Ag Lípidos y composiciones lipídicas para el suministro de agentes activos
WO2017083403A1 (en) * 2015-11-10 2017-05-18 Children's Research Institute, Children's National Medical Center Echinomycin formulation, method of making and method of use thereof

Also Published As

Publication number Publication date
PL3646854T3 (pl) 2023-01-02
IL282579B2 (en) 2024-11-01
SG11202103117RA (en) 2021-04-29
AU2019373387B2 (en) 2025-04-17
IL282579B1 (en) 2024-07-01
WO2020089342A1 (en) 2020-05-07
PT3646854T (pt) 2022-09-27
US20210386841A1 (en) 2021-12-16
CA3113877A1 (en) 2020-05-07
CN112930172A (zh) 2021-06-08
EP3646854B1 (en) 2022-08-03
AU2019373387A1 (en) 2021-04-29
KR20210084511A (ko) 2021-07-07
JP2022512983A (ja) 2022-02-07
ES2927532T3 (es) 2022-11-08
IL282579A (en) 2021-06-30
EP3646854A1 (en) 2020-05-06
JP7477504B2 (ja) 2024-05-01
CN112930172B (zh) 2023-12-22

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