CA2971284C - Compositions for introducing nucleic acid into cells - Google Patents

Compositions for introducing nucleic acid into cells Download PDF

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Publication number
CA2971284C
CA2971284C CA2971284A CA2971284A CA2971284C CA 2971284 C CA2971284 C CA 2971284C CA 2971284 A CA2971284 A CA 2971284A CA 2971284 A CA2971284 A CA 2971284A CA 2971284 C CA2971284 C CA 2971284C
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CA
Canada
Prior art keywords
copolymer
nucleic acid
repeating units
mrna
composition
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CA2971284A
Other languages
English (en)
French (fr)
Other versions
CA2971284A1 (en
Inventor
Christian Dohmen
Christian Plank
Carsten Rudolph
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ethris GmbH
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
Publication of CA2971284A1 publication Critical patent/CA2971284A1/en
Application granted granted Critical
Publication of CA2971284C publication Critical patent/CA2971284C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • 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
    • 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
    • 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
    • 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
    • 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)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Polymers & Plastics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
CA2971284A 2014-12-19 2015-12-18 Compositions for introducing nucleic acid into cells Active CA2971284C (en)

Applications Claiming Priority (3)

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

Publications (2)

Publication Number Publication Date
CA2971284A1 CA2971284A1 (en) 2016-06-23
CA2971284C true CA2971284C (en) 2023-01-10

Family

ID=52292658

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2971284A Active CA2971284C (en) 2014-12-19 2015-12-18 Compositions for introducing nucleic acid into cells

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)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3034539A1 (en) 2014-12-19 2016-06-22 Ethris GmbH Compositions for introducing nucleic acid into cells
GB2560250A (en) 2015-06-30 2018-09-05 Ethris Gmbh ATP-Binding cassette family coding polyribonucleotides and formulations thereof
WO2018010815A1 (en) * 2016-07-15 2018-01-18 Biontech Rna Pharmaceuticals Gmbh Formulation for administration of rna
KR20200109310A (ko) * 2018-01-11 2020-09-22 비온테크 알엔에이 파마슈티컬스 게엠베하 Rna의 투여를 위한 제형
US20210137840A1 (en) 2018-04-25 2021-05-13 Ethris Gmbh Lipid-based formulations for the delivery of rna
US20230000990A1 (en) * 2019-12-17 2023-01-05 Universiteit 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
IL305353A (en) 2021-02-26 2023-10-01 Ethris Gmbh Formulations for aerosol formation and aerosols for the delivery of nucleic acid
WO2023049814A2 (en) * 2021-09-22 2023-03-30 Sirnaomics, Inc. Nanoparticle pharmaceutical compositions with reduced nanoparticle size and improved polydispersity index
CN118475371A (zh) * 2021-09-22 2024-08-09 圣诺制药公司 基于组氨酸-赖氨酸共聚物形成的改善的纳米颗粒制剂
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
WO2024042236A1 (en) 2022-08-26 2024-02-29 Ethris Gmbh Stable lipid or lipidoid nanoparticle suspensions
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
CN121729225A (zh) 2023-08-25 2026-03-24 埃泽瑞斯公司 具有增强药物应用的特定表面活性剂性质的稳定脂质和类脂质纳米颗粒制剂
WO2025064850A1 (en) 2023-09-22 2025-03-27 BioNTech SE Rna constructs with n-terminal degrons to enhance an immune response
FR3155424A1 (fr) 2023-11-15 2025-05-23 BioNTech SE Compositions immunogènes contre le sars-cov-2
WO2025106754A1 (en) 2023-11-15 2025-05-22 BioNTech SE Coronavirus vaccine
WO2026047603A1 (en) 2024-08-28 2026-03-05 BioNTech SE Sars-cov-2 immunogenic compositions
WO2026057626A1 (en) 2024-09-11 2026-03-19 BioNTech SE Peptidoglycan hydrolases having multiple enzymatically active domains
WO2026057625A1 (en) 2024-09-11 2026-03-19 BioNTech SE Peptidoglycan hydrolases having a chap domain with engineered active site
WO2026082899A2 (en) 2024-10-16 2026-04-23 BioNTech SE Rna for preventing or treating tuberculosis

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JPS5821590A (ja) 1981-07-31 1983-02-08 株式会社東芝 制御棒駆動水圧装置
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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
EP3242903B1 (en) 2021-07-28
JP2018502956A (ja) 2018-02-01
US11020487B2 (en) 2021-06-01
CA2971284A1 (en) 2016-06-23
RU2715227C2 (ru) 2020-02-26
WO2016097377A1 (en) 2016-06-23
JP6745272B2 (ja) 2020-08-26
BR112017012482A2 (pt) 2018-02-27
EP3034539A1 (en) 2016-06-22
EP3242903A1 (en) 2017-11-15
AU2015366220A1 (en) 2017-06-01
BR112017012482B1 (pt) 2021-10-19
AU2015366220B2 (en) 2019-09-19
DK3242903T3 (da) 2021-09-27
US20180236090A1 (en) 2018-08-23
KR20170097124A (ko) 2017-08-25
RU2017122022A3 (enExample) 2019-05-22
CN107001627A (zh) 2017-08-01
KR102142180B1 (ko) 2020-08-07
ES2887404T3 (es) 2021-12-22
RU2017122022A (ru) 2018-12-24
CN107001627B (zh) 2020-01-14

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