JP6745272B2 - 核酸を細胞内へ導入するための組成物 - Google Patents

核酸を細胞内へ導入するための組成物 Download PDF

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Publication number
JP6745272B2
JP6745272B2 JP2017533008A JP2017533008A JP6745272B2 JP 6745272 B2 JP6745272 B2 JP 6745272B2 JP 2017533008 A JP2017533008 A JP 2017533008A JP 2017533008 A JP2017533008 A JP 2017533008A JP 6745272 B2 JP6745272 B2 JP 6745272B2
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Prior art keywords
copolymer
repeating units
nucleic acid
rna
composition
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Japanese (ja)
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JP2018502956A5 (enExample
JP2018502956A (ja
Inventor
ドーメン クリスチャン
ドーメン クリスチャン
プランク クリスチャン
プランク クリスチャン
ルドルフ カルステン
ルドルフ カルステン
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Ethris GmbH
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Ethris GmbH
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    • 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
    • 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
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal 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
    • A61K48/0025—Medicinal 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
    • A61K48/0041—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08G—MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G73/00—Macromolecular 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
    • C08G73/02—Polyamines
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08G—MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G73/00—Macromolecular 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
    • C08G73/02—Polyamines
    • C08G73/0206—Polyalkylene(poly)amines
    • 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

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)
JP2017533008A 2014-12-19 2015-12-18 核酸を細胞内へ導入するための組成物 Active JP6745272B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP14199439.2 2014-12-19
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 (3)

Publication Number Publication Date
JP2018502956A JP2018502956A (ja) 2018-02-01
JP2018502956A5 JP2018502956A5 (enExample) 2019-02-07
JP6745272B2 true JP6745272B2 (ja) 2020-08-26

Family

ID=52292658

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2017533008A Active JP6745272B2 (ja) 2014-12-19 2015-12-18 核酸を細胞内へ導入するための組成物

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)

Cited By (2)

* Cited by examiner, † Cited by third party
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US20230097985A1 (en) * 2021-09-22 2023-03-30 Sirnaomics, Inc. Nanoparticle formulations formed from histidine-lysine copolymers
WO2023049814A3 (en) * 2021-09-22 2023-06-01 Sirnaomics, Inc. Nanoparticle pharmaceutical compositions with reduced nanoparticle size and improved polydispersity index

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EP3034539A1 (en) 2014-12-19 2016-06-22 Ethris GmbH Compositions for introducing nucleic acid into cells
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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
JP7256824B2 (ja) 2018-04-25 2023-04-12 エスリス ゲーエムベーハー 粒子状製剤用の凍結保護剤
JP7863882B2 (ja) * 2019-12-17 2026-05-22 ウニヴェルズィテート ゲント ポリプロピレンイミンを含む非ウイルスベクター
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
MX2023009757A (es) 2021-02-26 2023-09-04 Ethris Gmbh Formulaciones para la formacion de aerosoles y aerosoles para la administracion de acido nucleico.
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
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WO2024240962A1 (en) 2023-05-25 2024-11-28 Ethris Gmbh Gm-csf-encoding nucleic acids, pharmaceutical compositions, methods and uses thereof
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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
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FR3155424A1 (fr) 2023-11-15 2025-05-23 BioNTech SE Compositions immunogènes contre le sars-cov-2
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
WO2026119817A1 (en) 2024-12-02 2026-06-11 BioNTech SE Agents and methods for targeted delivery of immune effector cells
WO2026125590A1 (en) 2024-12-13 2026-06-18 BioNTech SE Measuring nucleic acid integrity
WO2026125598A1 (en) 2024-12-13 2026-06-18 BioNTech SE Measuring nucleic acid integrity
WO2026148356A1 (en) 2025-01-06 2026-07-09 BioNTech SE Coronavirus and influenza immunogenic combinations
WO2026150132A1 (en) 2025-01-12 2026-07-16 BioNTech SE Combination therapy comprising an rna cancer vaccine and a multispecific binding agent targeting pd-1/pd-l1 and vegf/vegfr for cancer treatment
WO2026162682A1 (en) 2025-01-29 2026-08-06 BioNTech SE Compositions for delivery of plasmodium rh5 antigens and related methods
WO2026162552A1 (en) 2025-01-31 2026-08-06 BioNTech SE Agents and methods for targeted delivery to cells

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230097985A1 (en) * 2021-09-22 2023-03-30 Sirnaomics, Inc. Nanoparticle formulations formed from histidine-lysine copolymers
WO2023049814A3 (en) * 2021-09-22 2023-06-01 Sirnaomics, Inc. Nanoparticle pharmaceutical compositions with reduced nanoparticle size and improved polydispersity index

Also Published As

Publication number Publication date
EP3242903B1 (en) 2021-07-28
US20180236090A1 (en) 2018-08-23
KR102142180B1 (ko) 2020-08-07
BR112017012482B1 (pt) 2021-10-19
DK3242903T3 (da) 2021-09-27
RU2715227C2 (ru) 2020-02-26
WO2016097377A1 (en) 2016-06-23
JP2018502956A (ja) 2018-02-01
RU2017122022A3 (enExample) 2019-05-22
ES2887404T3 (es) 2021-12-22
KR20170097124A (ko) 2017-08-25
CN107001627B (zh) 2020-01-14
BR112017012482A2 (pt) 2018-02-27
RU2017122022A (ru) 2018-12-24
EP3242903A1 (en) 2017-11-15
US11020487B2 (en) 2021-06-01
CA2971284C (en) 2023-01-10
EP3034539A1 (en) 2016-06-22
AU2015366220A1 (en) 2017-06-01
CN107001627A (zh) 2017-08-01
AU2015366220B2 (en) 2019-09-19
CA2971284A1 (en) 2016-06-23

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