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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JP2018502956A (ja
JP2018502956A5 (enExample
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)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Polymers & Plastics (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (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)
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
EP14199439.2A 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
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 (1)

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

Families Citing this family (17)

* 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
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
WO2019137999A1 (en) 2018-01-11 2019-07-18 Biontech Rna Pharmaceuticals Gmbh Formulation for administration of rna
AU2019258678B2 (en) 2018-04-25 2024-08-22 Ethris Gmbh Lipid-based formulations for the delivery of RNA
EP4077689A1 (en) * 2019-12-17 2022-10-26 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
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
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
GB202416789D0 (en) 2023-11-15 2025-01-01 BioNTech SE SARS-COV-2 immunogenic compositions
WO2025106754A1 (en) 2023-11-15 2025-05-22 BioNTech SE Coronavirus vaccine

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4711955A (en) 1981-04-17 1987-12-08 Yale University Modified nucleotides and methods of preparing and using same
JPS5821590A (ja) 1981-07-31 1983-02-08 株式会社東芝 制御棒駆動水圧装置
JPS58215490A (ja) * 1982-06-08 1983-12-14 Nippon Shokubai Kagaku Kogyo Co Ltd 石炭−水スラリ−用分散剤
CA1223831A (en) 1982-06-23 1987-07-07 Dean Engelhardt Modified nucleotides, methods of preparing and utilizing and compositions containing the same
US5792608A (en) 1991-12-12 1998-08-11 Gilead Sciences, Inc. Nuclease stable and binding competent oligomers and methods for their use
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US5656611A (en) * 1994-11-18 1997-08-12 Supratek Pharma Inc. Polynucleotide compositions
DE19527102A1 (de) * 1995-07-25 1997-01-30 Bayer Ag Neue Polyamine und ihre Verwendung in Lacken und Beschichtungen
US6017700A (en) 1995-08-04 2000-01-25 Bayer Corporation Cationic oligonucleotides, and related methods of synthesis and use
FR2739292B1 (fr) * 1995-09-28 1997-10-31 Rhone Poulenc Rorer Sa Composition pharmaceutique utile pour la transfection d'acides nucleiques et ses utilisations
CA2298061A1 (en) * 1997-08-01 1999-02-11 Supratek Pharma, Inc. Polynucleotide compositions
ES2219346T3 (es) 1999-06-25 2004-12-01 Christian Plank Combinaciones para la introduccion de acidos nucleicos en celulas.
DE19933024C2 (de) * 1999-07-15 2003-03-13 Medinnova Ges Med Innovationen Kationische Blockcopolymere
EP1297169B1 (en) 2000-06-26 2012-08-08 Ethris Gmbh Method for transfecting cells using a magnetic field
US20030215395A1 (en) * 2002-05-14 2003-11-20 Lei Yu Controllably degradable polymeric biomolecule or drug carrier and method of synthesizing said carrier
US20070269891A9 (en) 2003-01-13 2007-11-22 Yasunobu Tanaka Solid surface with immobilized degradable cationic polymer for transfecting eukaryotic cells
JP4535229B2 (ja) 2003-05-08 2010-09-01 国立大学法人 東京大学 ポリエチレングリコール−ポリカチオンブロック共重合体
EP1859812B1 (en) 2005-02-10 2014-01-01 The University of Tokyo Polycation chargeable polymer and use as carrier of nucleic acid
CA2611944A1 (en) 2005-06-15 2006-12-28 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
BRPI0619932A2 (pt) 2005-12-15 2011-10-25 Centre Nat Rech Scient moléculas de oligonucleotìdeo-oligocátion, método para a obtenção das mesmas, reagentes de fosforamidita, método para uso em biologia e diagnóstico, e, composições farmacêuticas
US7700541B2 (en) * 2006-04-06 2010-04-20 Nitto Denko Corporation Biodegradable cationic polymers
GB0613753D0 (en) * 2006-07-11 2006-08-23 Norwegian Radium Hospital Res Method
EP2061443A4 (en) * 2006-08-18 2013-07-24 Arrowhead Res Corp POLYCONJUGATE FOR IN VIVO ADMINISTRATION OF POLYNUCLEOTIDES
US20090042825A1 (en) * 2007-08-06 2009-02-12 Majed Matar Composition, method of preparation & application of concentrated formulations of condensed nucleic acids with a cationic lipopolymer
CN102027043A (zh) * 2008-03-14 2011-04-20 艾根股份有限公司 生物可降解的交联支链聚(亚烷基亚胺)
JP6087504B2 (ja) 2008-11-07 2017-03-01 マサチューセッツ インスティテュート オブ テクノロジー アミノアルコールリピドイドおよびその使用
US20110263025A1 (en) 2008-12-02 2011-10-27 University Of Utah Research Foundation Biodegradable polydisulfide amines for gene delivery
WO2011012316A2 (de) 2009-07-31 2011-02-03 Ludwig-Maximilians-Universität Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
WO2011154331A1 (en) 2010-06-10 2011-12-15 F. Hoffmann-La Roche Ag Polymers for delivery of nucleic acids
EP2763701B1 (en) 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
US9931418B2 (en) 2012-08-07 2018-04-03 Northeastern University Compositions for the delivery of RNA and drugs into cells
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
EP3034539A1 (en) 2014-12-19 2016-06-22 Ethris GmbH Compositions for introducing nucleic acid into cells

Cited By (1)

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

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