PL3678673T3 - Kompozycje i sposoby do ukierunkowanego dostarczania, ekspresji i modulowania kodujących kwasów rybonukleinowych w tkankach - Google Patents

Kompozycje i sposoby do ukierunkowanego dostarczania, ekspresji i modulowania kodujących kwasów rybonukleinowych w tkankach

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Publication number
PL3678673T3
PL3678673T3 PL18792572.2T PL18792572T PL3678673T3 PL 3678673 T3 PL3678673 T3 PL 3678673T3 PL 18792572 T PL18792572 T PL 18792572T PL 3678673 T3 PL3678673 T3 PL 3678673T3
Authority
PL
Poland
Prior art keywords
modulation
tissue
expression
compositions
processes
Prior art date
Application number
PL18792572.2T
Other languages
English (en)
Polish (pl)
Inventor
Romain MICOL
Slawomir ANTOSZCZYK
Original Assignee
Combined Therapeutics, Inc.
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.)
Filing date
Publication date
Application filed by Combined Therapeutics, Inc. filed Critical Combined Therapeutics, Inc.
Publication of PL3678673T3 publication Critical patent/PL3678673T3/pl

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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • 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
    • 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
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/763Herpes virus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61K48/005Medicinal 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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
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    • 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
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
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    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/16011Herpesviridae
    • C12N2710/16611Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16632Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/16011Herpesviridae
    • C12N2710/16611Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641Use of virus, viral particle or viral elements as a vector
    • C12N2710/16643Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian
    • 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
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Chemical & Material Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
PL18792572.2T 2017-09-07 2018-09-06 Kompozycje i sposoby do ukierunkowanego dostarczania, ekspresji i modulowania kodujących kwasów rybonukleinowych w tkankach PL3678673T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1714430.4A GB201714430D0 (en) 2017-09-07 2017-09-07 Compositions and processes for targeted delivery and expression and modulation of therapeutic components in tissue
US201862632056P 2018-02-19 2018-02-19
PCT/US2018/049772 WO2019051100A1 (en) 2017-09-07 2018-09-06 COMPOSITIONS AND METHODS FOR TARGETED CODING ADMINISTRATION, EXPRESSION AND MODULATION OF RIBONUCLEIC ACIDS IN TISSUE

Publications (1)

Publication Number Publication Date
PL3678673T3 true PL3678673T3 (pl) 2023-07-10

Family

ID=60117350

Family Applications (1)

Application Number Title Priority Date Filing Date
PL18792572.2T PL3678673T3 (pl) 2017-09-07 2018-09-06 Kompozycje i sposoby do ukierunkowanego dostarczania, ekspresji i modulowania kodujących kwasów rybonukleinowych w tkankach

Country Status (14)

Country Link
US (3) US11359212B2 (https=)
EP (2) EP3678673B1 (https=)
JP (4) JP2020533411A (https=)
KR (1) KR102741572B1 (https=)
CN (1) CN111212650A (https=)
AU (1) AU2018328289B2 (https=)
CA (1) CA3074293A1 (https=)
ES (1) ES2942890T3 (https=)
GB (1) GB201714430D0 (https=)
MX (2) MX2020002568A (https=)
PL (1) PL3678673T3 (https=)
PT (1) PT3678673T (https=)
RU (1) RU2020112990A (https=)
WO (1) WO2019051100A1 (https=)

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* Cited by examiner, † Cited by third party
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AU2018316166A1 (en) 2017-08-07 2020-02-06 The Regents Of The University Of California Platform for generating safe cell therapeutics
GB201714430D0 (en) * 2017-09-07 2017-10-25 Micol Romain Compositions and processes for targeted delivery and expression and modulation of therapeutic components in tissue
IL295724A (en) 2020-02-21 2022-10-01 Combined Therapeutics Inc Compositions and methods for organ-protective expression and modulation of coding ribonucleic acids
IL300247A (en) 2020-07-31 2023-03-01 Combined Therapeutics Inc Compositions and methods for improving vaccination
CA3171589A1 (en) 2021-05-03 2022-11-03 Moritz THRAN Improved nucleic acid sequence for cell type specific expression
CN113444744A (zh) * 2021-06-28 2021-09-28 北京鼎成肽源生物技术有限公司 一种用于基因编辑NK细胞的mRNA模板及构建方法、mRNA的体外转录方法和应用
CN114107347B (zh) * 2021-11-24 2022-10-28 中国人民解放军空军军医大学 一种基于具有按需抗炎功能、装载炎症响应型mRNA的工程化外泌体及其构建方法和应用

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Also Published As

Publication number Publication date
RU2020112990A (ru) 2021-10-07
MX2020002568A (es) 2020-09-18
EP3678673B1 (en) 2023-01-25
AU2018328289B2 (en) 2025-07-10
CN111212650A (zh) 2020-05-29
JP2023159160A (ja) 2023-10-31
KR20200104282A (ko) 2020-09-03
JP2024059656A (ja) 2024-05-01
RU2020130763A (ru) 2022-03-21
RU2020130763A3 (https=) 2022-03-22
EP3678673A1 (en) 2020-07-15
ES2942890T3 (es) 2023-06-07
RU2020112990A3 (https=) 2022-03-05
US11359212B2 (en) 2022-06-14
MX2020008606A (es) 2020-09-21
US20220220506A1 (en) 2022-07-14
US12195746B2 (en) 2025-01-14
EP4218770A2 (en) 2023-08-02
AU2018328289A1 (en) 2020-03-19
GB201714430D0 (en) 2017-10-25
JP2024122983A (ja) 2024-09-10
US20250129383A1 (en) 2025-04-24
CA3074293A1 (en) 2019-03-14
JP2020533411A (ja) 2020-11-19
WO2019051100A1 (en) 2019-03-14
PT3678673T (pt) 2023-04-24
US20200255863A1 (en) 2020-08-13
KR102741572B1 (ko) 2024-12-10
EP4218770A3 (en) 2023-08-09

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