DK3242903T3 - Sammensætninger til at indføre nukleinsyre i celler - Google Patents

Sammensætninger til at indføre nukleinsyre i celler Download PDF

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Publication number
DK3242903T3
DK3242903T3 DK15823330.4T DK15823330T DK3242903T3 DK 3242903 T3 DK3242903 T3 DK 3242903T3 DK 15823330 T DK15823330 T DK 15823330T DK 3242903 T3 DK3242903 T3 DK 3242903T3
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DK
Denmark
Prior art keywords
compositions
cells
nucleic acid
introducing nucleic
introducing
Prior art date
Application number
DK15823330.4T
Other languages
Danish (da)
English (en)
Inventor
Christian Dohmen
Carsten Rudolph
Christian Plank
Original Assignee
Ethris Gmbh
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ethris Gmbh filed Critical Ethris Gmbh
Application granted granted Critical
Publication of DK3242903T3 publication Critical patent/DK3242903T3/da

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    • 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
    • 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
    • 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
    • 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
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08GMACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G73/00Macromolecular compounds obtained by reactions forming a linkage containing nitrogen with or without oxygen or carbon in the main chain of the macromolecule, not provided for in groups C08G12/00 - C08G71/00
    • C08G73/02Polyamines
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08GMACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G73/00Macromolecular compounds obtained by reactions forming a linkage containing nitrogen with or without oxygen or carbon in the main chain of the macromolecule, not provided for in groups C08G12/00 - C08G71/00
    • C08G73/02Polyamines
    • C08G73/0206Polyalkylene(poly)amines
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Polymers & Plastics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Compositions Of Macromolecular Compounds (AREA)
  • Macromolecular Compounds Obtained By Forming Nitrogen-Containing Linkages In General (AREA)
DK15823330.4T 2014-12-19 2015-12-18 Sammensætninger til at indføre nukleinsyre i celler DK3242903T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP14199439.2A EP3034539A1 (en) 2014-12-19 2014-12-19 Compositions for introducing nucleic acid into cells
PCT/EP2015/080669 WO2016097377A1 (en) 2014-12-19 2015-12-18 Compositions for introducing nucleic acid into cells

Publications (1)

Publication Number Publication Date
DK3242903T3 true DK3242903T3 (da) 2021-09-27

Family

ID=52292658

Family Applications (1)

Application Number Title Priority Date Filing Date
DK15823330.4T DK3242903T3 (da) 2014-12-19 2015-12-18 Sammensætninger til at indføre nukleinsyre i celler

Country Status (12)

Country Link
US (1) US11020487B2 (enExample)
EP (2) EP3034539A1 (enExample)
JP (1) JP6745272B2 (enExample)
KR (1) KR102142180B1 (enExample)
CN (1) CN107001627B (enExample)
AU (1) AU2015366220B2 (enExample)
BR (1) BR112017012482B1 (enExample)
CA (1) CA2971284C (enExample)
DK (1) DK3242903T3 (enExample)
ES (1) ES2887404T3 (enExample)
RU (1) RU2715227C2 (enExample)
WO (1) WO2016097377A1 (enExample)

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EP3034539A1 (en) 2014-12-19 2016-06-22 Ethris GmbH Compositions for introducing nucleic acid into cells
DE112016002962T5 (de) 2015-06-30 2018-05-24 Ethris Gmbh Familie der ATP-bindenden Kassetten-codierende Polyribonucleotide und Formulierungen derselben
WO2018010815A1 (en) * 2016-07-15 2018-01-18 Biontech Rna Pharmaceuticals Gmbh Formulation for administration of rna
CA3087442A1 (en) * 2018-01-11 2019-07-18 Biontech Rna Pharmaceuticals Gmbh Formulation for administration of rna
EP3784284B1 (en) 2018-04-25 2023-08-02 Ethris GmbH Lipid-based formulations for the delivery of rna
IL293911A (en) * 2019-12-17 2022-08-01 Univ Gent Non-viral vectors comprising polypropyleneimine
CN111249476B (zh) * 2020-02-19 2023-09-26 深圳厚存纳米药业有限公司 泊洛沙姆和/或泊洛沙胺与脂质组合中性复合物纳米粒
CN116887812A (zh) 2021-02-26 2023-10-13 埃泽瑞斯公司 用于气溶胶形成的制剂以及用于递送核酸的气溶胶
WO2023049814A2 (en) * 2021-09-22 2023-03-30 Sirnaomics, Inc. Nanoparticle pharmaceutical compositions with reduced nanoparticle size and improved polydispersity index
EP4404973A4 (en) * 2021-09-22 2025-10-15 Sirnaomics Inc IMPROVED NANOPARTICLE FORMULATIONS FORMED FROM HISTIDINE-LYSINE COPOLYMERS
CN116549626A (zh) * 2022-01-27 2023-08-08 深圳瑞吉生物科技有限公司 一种载核酸脂质纳米颗粒冻干制剂及其制备方法与应用
EP4327829A1 (en) 2022-08-26 2024-02-28 Ethris GmbH Stabilization of lipid or lipidoid nanoparticle suspensions
JP2025529925A (ja) 2022-08-26 2025-09-09 エスリス ゲーエムベーハー 安定な脂質またはリピドイドナノ粒子懸濁液
WO2024240962A1 (en) 2023-05-25 2024-11-28 Ethris Gmbh Gm-csf-encoding nucleic acids, pharmaceutical compositions, methods and uses thereof
CN116478410B (zh) * 2023-06-20 2023-09-12 觅投克(北京)生物医学技术有限公司 一种菊糖修饰的聚乙烯亚胺衍生物及其制备方法和应用
WO2025045767A1 (en) 2023-08-25 2025-03-06 Ethris Gmbh Stabilized lipid and lipidoid nanoparticle formulations with specific surfactant properties for enhanced pharmaceutical applications
WO2025064850A1 (en) 2023-09-22 2025-03-27 BioNTech SE Rna constructs with n-terminal degrons to enhance an immune response
WO2025106738A1 (en) 2023-11-15 2025-05-22 BioNTech SE Sars-cov-2 immunogenic compositions
TW202539697A (zh) 2023-11-15 2025-10-16 德商拜恩技術股份公司 冠狀病毒疫苗
WO2026047603A1 (en) 2024-08-28 2026-03-05 BioNTech SE Sars-cov-2 immunogenic compositions

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CA1223831A (en) 1982-06-23 1987-07-07 Dean Engelhardt Modified nucleotides, methods of preparing and utilizing and compositions containing the same
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Also Published As

Publication number Publication date
JP6745272B2 (ja) 2020-08-26
EP3242903A1 (en) 2017-11-15
AU2015366220A1 (en) 2017-06-01
CA2971284A1 (en) 2016-06-23
CN107001627B (zh) 2020-01-14
US11020487B2 (en) 2021-06-01
US20180236090A1 (en) 2018-08-23
RU2017122022A3 (enExample) 2019-05-22
CN107001627A (zh) 2017-08-01
BR112017012482A2 (pt) 2018-02-27
AU2015366220B2 (en) 2019-09-19
RU2017122022A (ru) 2018-12-24
ES2887404T3 (es) 2021-12-22
KR20170097124A (ko) 2017-08-25
JP2018502956A (ja) 2018-02-01
WO2016097377A1 (en) 2016-06-23
BR112017012482B1 (pt) 2021-10-19
CA2971284C (en) 2023-01-10
EP3242903B1 (en) 2021-07-28
EP3034539A1 (en) 2016-06-22
KR102142180B1 (ko) 2020-08-07
RU2715227C2 (ru) 2020-02-26

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