PT3678673T - Composições e processos para aministração dirigida, expressão e modulação de ácidos robonucleicos codificantes num tecido - Google Patents

Composições e processos para aministração dirigida, expressão e modulação de ácidos robonucleicos codificantes num tecido

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Publication number
PT3678673T
PT3678673T PT187925722T PT18792572T PT3678673T PT 3678673 T PT3678673 T PT 3678673T PT 187925722 T PT187925722 T PT 187925722T PT 18792572 T PT18792572 T PT 18792572T PT 3678673 T PT3678673 T PT 3678673T
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PT
Portugal
Prior art keywords
modulation
tissue
expression
compositions
processes
Prior art date
Application number
PT187925722T
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English (en)
Portuguese (pt)
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 PT3678673T publication Critical patent/PT3678673T/pt

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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/63Introduction 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
    • 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
    • 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
    • C12N2710/16632Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • CCHEMISTRY; METALLURGY
    • 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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  • Health & Medical Sciences (AREA)
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  • 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)
PT187925722T 2017-09-07 2018-09-06 Composições e processos para aministração dirigida, expressão e modulação de ácidos robonucleicos codificantes num tecido PT3678673T (pt)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
PT3678673T true PT3678673T (pt) 2023-04-24

Family

ID=60117350

Family Applications (1)

Application Number Title Priority Date Filing Date
PT187925722T PT3678673T (pt) 2017-09-07 2018-09-06 Composições e processos para aministração dirigida, expressão e modulação de ácidos robonucleicos codificantes num tecido

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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* Cited by examiner, † Cited by third party
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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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EP2213300B1 (en) * 2001-05-09 2015-03-25 Takara Bio, Inc. Composition and method for treating cancer using herpes virus
ATE420160T1 (de) * 2003-06-18 2009-01-15 Genelux Corp Modifizierte rekombinante vacciniaviren, verwendungen davon
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
WO2009012263A2 (en) 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
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JPWO2011101912A1 (ja) * 2010-02-19 2013-06-17 国立大学法人 東京大学 組み換えヘルペスウイルス及び組換えヘルペスウイルスを含む医薬組成物
DE19216461T1 (de) * 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und 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
CA3074293A1 (en) 2019-03-14
US11359212B2 (en) 2022-06-14
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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