RU2715227C2 - Композиция для введения нуклеиновой кислоты в клетки - Google Patents

Композиция для введения нуклеиновой кислоты в клетки Download PDF

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RU2715227C2
RU2715227C2 RU2017122022A RU2017122022A RU2715227C2 RU 2715227 C2 RU2715227 C2 RU 2715227C2 RU 2017122022 A RU2017122022 A RU 2017122022A RU 2017122022 A RU2017122022 A RU 2017122022A RU 2715227 C2 RU2715227 C2 RU 2715227C2
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copolymer
nucleic acid
repeating units
rna
composition
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RU2017122022A3 (cg-RX-API-DMAC7.html
RU2017122022A (ru
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Кристиан Домен
Кристиан Планк
Карстен Рудольф
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Этрис Гмбх
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    • 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
    • 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
    • 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

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  • 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)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Polymers & Plastics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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)
RU2017122022A 2014-12-19 2015-12-18 Композиция для введения нуклеиновой кислоты в клетки RU2715227C2 (ru)

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 (3)

Publication Number Publication Date
RU2017122022A RU2017122022A (ru) 2018-12-24
RU2017122022A3 RU2017122022A3 (cg-RX-API-DMAC7.html) 2019-05-22
RU2715227C2 true RU2715227C2 (ru) 2020-02-26

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RU2017122022A RU2715227C2 (ru) 2014-12-19 2015-12-18 Композиция для введения нуклеиновой кислоты в клетки

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US (1) US11020487B2 (cg-RX-API-DMAC7.html)
EP (2) EP3034539A1 (cg-RX-API-DMAC7.html)
JP (1) JP6745272B2 (cg-RX-API-DMAC7.html)
KR (1) KR102142180B1 (cg-RX-API-DMAC7.html)
CN (1) CN107001627B (cg-RX-API-DMAC7.html)
AU (1) AU2015366220B2 (cg-RX-API-DMAC7.html)
BR (1) BR112017012482B1 (cg-RX-API-DMAC7.html)
CA (1) CA2971284C (cg-RX-API-DMAC7.html)
DK (1) DK3242903T3 (cg-RX-API-DMAC7.html)
ES (1) ES2887404T3 (cg-RX-API-DMAC7.html)
RU (1) RU2715227C2 (cg-RX-API-DMAC7.html)
WO (1) WO2016097377A1 (cg-RX-API-DMAC7.html)

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JP2018520155A (ja) 2015-06-30 2018-07-26 エスリス ゲーエムベーハーethris GmbH ポリリボヌクレオチドをコードするatp結合カセットファミリーおよびその製剤
WO2018010815A1 (en) * 2016-07-15 2018-01-18 Biontech Rna Pharmaceuticals Gmbh Formulation for administration of rna
BR112020013974A8 (pt) * 2018-01-11 2022-10-18 Tron Translationale Onkologie An Der Univ Der Johannes Gutenberg Univ Mainz Ggmbh Formulação para administração de rna
CN112153985B (zh) 2018-04-25 2024-03-01 埃泽瑞斯公司 用于颗粒制剂的防冻剂
CN119733057A (zh) * 2019-12-17 2025-04-01 根特大学 包含聚丙烯亚胺的非病毒载体
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
JP2024507394A (ja) 2021-02-26 2024-02-19 エスリス ゲーエムベーハー エアロゾル形成用の製剤および核酸を送達するためのエアロゾル
EP4404979A4 (en) * 2021-09-22 2025-10-08 Sirnaomics Inc PHARMACEUTICAL COMPOSITIONS OF NANOPARTICLES WITH REDUCED NANOPARTICLE SIZE AND IMPROVED POLYDISPERSITY INDEX
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
CN120129532A (zh) 2022-08-26 2025-06-10 埃泽瑞斯公司 稳定的脂质或类脂质纳米颗粒悬浮液
EP4327829A1 (en) 2022-08-26 2024-02-28 Ethris GmbH Stabilization of 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
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
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DK3242903T3 (da) 2021-09-27
JP2018502956A (ja) 2018-02-01
KR102142180B1 (ko) 2020-08-07
EP3034539A1 (en) 2016-06-22
CA2971284A1 (en) 2016-06-23
CA2971284C (en) 2023-01-10
BR112017012482B1 (pt) 2021-10-19
BR112017012482A2 (pt) 2018-02-27
CN107001627B (zh) 2020-01-14
AU2015366220A1 (en) 2017-06-01
EP3242903A1 (en) 2017-11-15
CN107001627A (zh) 2017-08-01
RU2017122022A3 (cg-RX-API-DMAC7.html) 2019-05-22
JP6745272B2 (ja) 2020-08-26
ES2887404T3 (es) 2021-12-22
KR20170097124A (ko) 2017-08-25
US20180236090A1 (en) 2018-08-23
AU2015366220B2 (en) 2019-09-19
EP3242903B1 (en) 2021-07-28
WO2016097377A1 (en) 2016-06-23
RU2017122022A (ru) 2018-12-24
US11020487B2 (en) 2021-06-01

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