CA3074293A1 - Compositions and processes for targeted delivery, expression and modulation of coding ribonucleic acids in tissue - Google Patents

Compositions and processes for targeted delivery, expression and modulation of coding ribonucleic acids in tissue Download PDF

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Publication number
CA3074293A1
CA3074293A1 CA3074293A CA3074293A CA3074293A1 CA 3074293 A1 CA3074293 A1 CA 3074293A1 CA 3074293 A CA3074293 A CA 3074293A CA 3074293 A CA3074293 A CA 3074293A CA 3074293 A1 CA3074293 A1 CA 3074293A1
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sequence
mrna
cells
cell
mirna
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Pending
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CA3074293A
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English (en)
French (fr)
Inventor
Romain Micol
Slawomir Antoszczyk
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Combined Therapeutics Inc
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Publication of CA3074293A1 publication Critical patent/CA3074293A1/en
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/763—Herpes virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16632—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16643—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • Y—GENERAL 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
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • 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)
CA3074293A 2017-09-07 2018-09-06 Compositions and processes for targeted delivery, expression and modulation of coding ribonucleic acids in tissue Pending CA3074293A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB1714430.4 2017-09-07
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
US62/632,056 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
CA3074293A1 true CA3074293A1 (en) 2019-03-14

Family

ID=60117350

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3074293A Pending CA3074293A1 (en) 2017-09-07 2018-09-06 Compositions and processes for targeted delivery, expression and modulation of coding ribonucleic acids in tissue

Country Status (14)

Country Link
US (3) US11359212B2 (https=)
EP (2) EP4218770A3 (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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WO2019032628A1 (en) 2017-08-07 2019-02-14 The Regents Of The University Of California GENERATING PLATFORM FOR SAFE CELLULAR THERAPEUTIC AGENTS
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
JP7539723B2 (ja) 2020-02-21 2024-08-26 コンバインド セラピューティクス インコーポレイテッド コード化リボ核酸の器官保護発現及び調節のための組成物並びに方法
MX2023001187A (es) 2020-07-31 2023-04-04 Combined Therapeutics Inc Composiciones y métodos para una vacunación mejorada.
EP4334446A1 (en) 2021-05-03 2024-03-13 CureVac SE 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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ATE420160T1 (de) * 2003-06-18 2009-01-15 Genelux Corp Modifizierte rekombinante vacciniaviren, verwendungen davon
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JP7539723B2 (ja) * 2020-02-21 2024-08-26 コンバインド セラピューティクス インコーポレイテッド コード化リボ核酸の器官保護発現及び調節のための組成物並びに方法

Also Published As

Publication number Publication date
JP2024059656A (ja) 2024-05-01
US12195746B2 (en) 2025-01-14
AU2018328289B2 (en) 2025-07-10
MX2020008606A (es) 2020-09-21
JP2020533411A (ja) 2020-11-19
EP3678673B1 (en) 2023-01-25
RU2020112990A (ru) 2021-10-07
RU2020130763A3 (https=) 2022-03-22
US20250129383A1 (en) 2025-04-24
CN111212650A (zh) 2020-05-29
EP4218770A3 (en) 2023-08-09
US20200255863A1 (en) 2020-08-13
WO2019051100A1 (en) 2019-03-14
RU2020130763A (ru) 2022-03-21
RU2020112990A3 (https=) 2022-03-05
ES2942890T3 (es) 2023-06-07
KR20200104282A (ko) 2020-09-03
AU2018328289A1 (en) 2020-03-19
JP2023159160A (ja) 2023-10-31
PL3678673T3 (pl) 2023-07-10
US11359212B2 (en) 2022-06-14
PT3678673T (pt) 2023-04-24
GB201714430D0 (en) 2017-10-25
KR102741572B1 (ko) 2024-12-10
EP4218770A2 (en) 2023-08-02
US20220220506A1 (en) 2022-07-14
JP2024122983A (ja) 2024-09-10
EP3678673A1 (en) 2020-07-15
MX2020002568A (es) 2020-09-18

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