BR112017012482B1 - Composição, usos de uma composição, e, métodos para liberar um ácido nucleico a uma célula ou tecido alvo e para a produção da composição - Google Patents

Composição, usos de uma composição, e, métodos para liberar um ácido nucleico a uma célula ou tecido alvo e para a produção da composição Download PDF

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Publication number
BR112017012482B1
BR112017012482B1 BR112017012482-3A BR112017012482A BR112017012482B1 BR 112017012482 B1 BR112017012482 B1 BR 112017012482B1 BR 112017012482 A BR112017012482 A BR 112017012482A BR 112017012482 B1 BR112017012482 B1 BR 112017012482B1
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BR
Brazil
Prior art keywords
copolymer
repeating units
nucleic acid
composition
mrna
Prior art date
Application number
BR112017012482-3A
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English (en)
Portuguese (pt)
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BR112017012482A2 (pt
Inventor
Christian Dohmen
Christian Plank
Carsten Rudolph
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Ethris Gmbh
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Publication date
Application filed by Ethris Gmbh filed Critical Ethris Gmbh
Publication of BR112017012482A2 publication Critical patent/BR112017012482A2/pt
Publication of BR112017012482B1 publication Critical patent/BR112017012482B1/pt

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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
    • 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
    • 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
    • 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
    • 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)
BR112017012482-3A 2014-12-19 2015-12-18 Composição, usos de uma composição, e, métodos para liberar um ácido nucleico a uma célula ou tecido alvo e para a produção da composição BR112017012482B1 (pt)

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 (2)

Publication Number Publication Date
BR112017012482A2 BR112017012482A2 (pt) 2018-02-27
BR112017012482B1 true BR112017012482B1 (pt) 2021-10-19

Family

ID=52292658

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112017012482-3A BR112017012482B1 (pt) 2014-12-19 2015-12-18 Composição, usos de uma composição, e, métodos para liberar um ácido nucleico a uma célula ou tecido alvo e para a produção da composição

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
ES2960936T3 (es) 2018-04-25 2024-03-07 Ethris Gmbh Formulaciones a base de lípidos para el suministro de ARN
CN114829615B (zh) * 2019-12-17 2025-01-10 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
EP4297722A1 (en) 2021-02-26 2024-01-03 Ethris GmbH Formulations for aerosol formation and aerosols for the delivery of nucleic acid
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
WO2024042236A1 (en) 2022-08-26 2024-02-29 Ethris Gmbh Stable lipid or lipidoid nanoparticle suspensions
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
WO2025106754A1 (en) 2023-11-15 2025-05-22 BioNTech SE Coronavirus vaccine
CA3254064A1 (en) 2023-11-15 2025-05-21 BioNTech SE Immunogenic compositions against SARS-CoV-2

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Also Published As

Publication number Publication date
JP2018502956A (ja) 2018-02-01
CN107001627B (zh) 2020-01-14
WO2016097377A1 (en) 2016-06-23
CN107001627A (zh) 2017-08-01
AU2015366220B2 (en) 2019-09-19
CA2971284A1 (en) 2016-06-23
CA2971284C (en) 2023-01-10
RU2715227C2 (ru) 2020-02-26
DK3242903T3 (da) 2021-09-27
KR102142180B1 (ko) 2020-08-07
EP3034539A1 (en) 2016-06-22
JP6745272B2 (ja) 2020-08-26
RU2017122022A3 (enExample) 2019-05-22
AU2015366220A1 (en) 2017-06-01
EP3242903A1 (en) 2017-11-15
RU2017122022A (ru) 2018-12-24
US11020487B2 (en) 2021-06-01
US20180236090A1 (en) 2018-08-23
KR20170097124A (ko) 2017-08-25
BR112017012482A2 (pt) 2018-02-27
EP3242903B1 (en) 2021-07-28
ES2887404T3 (es) 2021-12-22

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Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
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