DK3013964T3 - Sammensætninger til indføring af rna i celler - Google Patents

Sammensætninger til indføring af rna i celler Download PDF

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Publication number
DK3013964T3
DK3013964T3 DK14742474.1T DK14742474T DK3013964T3 DK 3013964 T3 DK3013964 T3 DK 3013964T3 DK 14742474 T DK14742474 T DK 14742474T DK 3013964 T3 DK3013964 T3 DK 3013964T3
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DK
Denmark
Prior art keywords
compositions
cells
introducing rna
rna
introducing
Prior art date
Application number
DK14742474.1T
Other languages
English (en)
Inventor
Christian Koch
Christian Dohmen
Christian Plank
Carsten Rudolph
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.)
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Application filed by Ethris Gmbh filed Critical Ethris Gmbh
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Publication of DK3013964T3 publication Critical patent/DK3013964T3/da

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    • 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
    • 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
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K47/58Medicinal 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 by reactions only involving carbon-to-carbon unsaturated bonds, e.g. poly[meth]acrylate, polyacrylamide, polystyrene, polyvinylpyrrolidone, polyvinylalcohol or polystyrene sulfonic acid resin
    • 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
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08FMACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F8/00Chemical modification by after-treatment
    • C08F8/30Introducing nitrogen atoms or nitrogen-containing groups
    • C08F8/32Introducing nitrogen atoms or nitrogen-containing groups by reaction with amines
    • 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
    • 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
    • C08G73/0213Preparatory process
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02PCLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00Technologies relating to chemical industry
    • Y02P20/50Improvements relating to the production of bulk chemicals
    • Y02P20/55Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Polymers & Plastics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Inorganic Chemistry (AREA)
DK14742474.1T 2013-06-28 2014-06-27 Sammensætninger til indføring af rna i celler DK3013964T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP13174390 2013-06-28
EP13181380 2013-08-22
PCT/EP2014/063756 WO2014207231A1 (en) 2013-06-28 2014-06-27 Compositions for introducing rna into cells

Publications (1)

Publication Number Publication Date
DK3013964T3 true DK3013964T3 (da) 2020-06-15

Family

ID=51224914

Family Applications (1)

Application Number Title Priority Date Filing Date
DK14742474.1T DK3013964T3 (da) 2013-06-28 2014-06-27 Sammensætninger til indføring af rna i celler

Country Status (13)

Country Link
US (1) US20170021036A1 (da)
EP (1) EP3013964B1 (da)
JP (1) JP6609246B2 (da)
KR (1) KR102285326B1 (da)
CN (1) CN105579584B (da)
AU (1) AU2014300980B2 (da)
BR (1) BR112015032225B1 (da)
CA (1) CA2916800C (da)
DK (1) DK3013964T3 (da)
EA (1) EA036400B1 (da)
ES (1) ES2810298T3 (da)
WO (1) WO2014207231A1 (da)
ZA (1) ZA201509111B (da)

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DK2718269T3 (da) 2011-06-08 2018-04-09 Translate Bio Inc Spaltelige lipider
RU2016138020A (ru) * 2014-02-26 2018-03-29 Этрис Гмбх Композиции для введения phk в желудочно-кишечный тракт
RU2771104C2 (ru) 2014-05-14 2022-04-26 Таргиммьюн Терапьютикс Аг Улучшенные полиэтилениминовые полиэтиленгликолевые векторы
JP2017534649A (ja) * 2014-11-10 2017-11-24 エスリス ゲーエムベーハーethris GmbH Bmpをコードするrnaの送達による骨形成誘導
DK3317411T3 (da) 2015-06-30 2021-03-15 Ethris Gmbh Atp-bindingskassettefamilie som koder polyribonukleotider og formuleringer deraf
BR112018011193A2 (pt) * 2015-12-01 2018-11-21 Spark Therapeutics Inc métodos escalonáveis para produzir vetor viral adeno-associado (aav) recombinante em sistema de cultura celular em suspensão isento de soro adequado para uso clínico
BR112018075479A2 (pt) * 2016-06-09 2019-03-19 Curevac Ag portadores híbridos para carga de ácido nucleico
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
EP3565605A1 (en) 2017-01-03 2019-11-13 ethris GmbH Ornithine transcarbamylase coding polyribonucleotides and formulations thereof
WO2019204451A1 (en) * 2018-04-17 2019-10-24 Carnegie Mellon University Enhanced lipid nanoparticle drug delivery using a negatively charged polymer
CN112153985B (zh) 2018-04-25 2024-03-01 埃泽瑞斯公司 用于颗粒制剂的防冻剂
CA3129912A1 (en) * 2019-02-14 2020-08-20 Ethris Gmbh Treatment of ciliopathies
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
IL302689A (en) 2020-11-04 2023-07-01 Ethris Gmbh Use of IFN-Lambda mRNA to treat viral infections
CA3209032A1 (en) 2021-02-26 2022-09-01 Ethris Gmbh Formulations for aerosol formation and aerosols for the delivery of nucleic acid
EP4333810A1 (en) * 2021-05-07 2024-03-13 Carnegie Mellon University Lipid nanoparticle-mediated mrna delivery to the pancreas
CN113876639B (zh) * 2021-11-12 2023-08-22 湖北省麦诗特生物科技有限公司 一种含有提高皮肤弹性脂质体的面霜组合物及其制备方法
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
WO2024121160A1 (en) 2022-12-05 2024-06-13 Ethris Gmbh Regulator(s) of energy homeostasis-encoding rna molecule(s) with increased translation efficiency

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Also Published As

Publication number Publication date
ZA201509111B (en) 2021-03-31
AU2014300980A1 (en) 2016-01-07
ES2810298T3 (es) 2021-03-08
CN105579584A (zh) 2016-05-11
CA2916800A1 (en) 2014-12-31
BR112015032225B1 (pt) 2022-05-17
EA036400B1 (ru) 2020-11-06
KR20160040524A (ko) 2016-04-14
CN105579584B (zh) 2020-08-28
EA201600044A1 (ru) 2016-06-30
BR112015032225A2 (pt) 2017-07-25
EP3013964A1 (en) 2016-05-04
US20170021036A1 (en) 2017-01-26
AU2014300980B2 (en) 2020-03-05
JP2016524898A (ja) 2016-08-22
WO2014207231A1 (en) 2014-12-31
EP3013964B1 (en) 2020-05-06
CA2916800C (en) 2022-10-25
JP6609246B2 (ja) 2019-11-20
KR102285326B1 (ko) 2021-08-04

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