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

Info

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
Authority
CA
Canada
Prior art keywords
sequence
mrna
cells
cell
mirna
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3074293A
Other languages
English (en)
French (fr)
Inventor
Romain MICOL
Slawomir ANTOSZCZYK
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Combined Therapeutics Inc
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 CA3074293A1 publication Critical patent/CA3074293A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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
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
GB1714430.4 2017-09-07
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 (enExample)
EP (2) EP4218770A3 (enExample)
JP (4) JP2020533411A (enExample)
KR (1) KR102741572B1 (enExample)
CN (1) CN111212650A (enExample)
AU (1) AU2018328289B2 (enExample)
CA (1) CA3074293A1 (enExample)
ES (1) ES2942890T3 (enExample)
GB (1) GB201714430D0 (enExample)
MX (2) MX2020002568A (enExample)
PL (1) PL3678673T3 (enExample)
PT (1) PT3678673T (enExample)
RU (1) RU2020112990A (enExample)
WO (1) WO2019051100A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020530454A (ja) 2017-08-07 2020-10-22 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 安全な細胞治療薬を生成するためのプラットフォーム
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
WO2022035621A1 (en) 2020-07-31 2022-02-17 Combined Therapeutics, Inc. Compositions and methods for improved vaccination
WO2022233880A1 (en) 2021-05-03 2022-11-10 Curevac Ag 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

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004529607A (ja) * 2000-09-15 2004-09-30 キュラジェン コーポレイション ヒトポリヌクレオチドおよびそれによってコードされるポリペプチド
EP2140879A1 (en) * 2001-05-09 2010-01-06 M'S Science Corporation Composition and method for treating cancer using herpes virus
SG179291A1 (en) * 2003-06-18 2012-04-27 Genelux Corp Modified recombinant vaccinia viruses and other microorganisms, uses thereof
EP1648914A4 (en) 2003-07-31 2009-12-16 Regulus Therapeutics Inc OLIGOMERIC COMPOUNDS AND COMPOSITIONS USEFUL FOR MODULATING SMALL NON-CODING RNA
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
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
WO2011101912A1 (ja) * 2010-02-19 2011-08-25 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
EP2763701B1 (en) * 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
KR20140102759A (ko) * 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물
CA2863799C (en) 2012-02-22 2020-09-01 Matthew J. FRIGAULT Compositions and methods for generating a persisting population of t cells useful for the treatment of cancer
CA2892529C (en) * 2012-11-26 2023-04-25 Moderna Therapeutics, Inc. Terminally modified rna
WO2015058069A1 (en) * 2013-10-18 2015-04-23 Moderna Therapeutics, Inc. Compositions and methods for tolerizing cellular systems
JP6588024B2 (ja) * 2013-10-28 2019-10-09 ユニヴァーシティ オヴ ピッツバーグ オヴ ザ コモンウェルス システム オヴ ハイアー エデュケーション 腫瘍溶解性hsvベクター
CN105848674A (zh) * 2013-11-11 2016-08-10 阿尔莫生物科技股份有限公司 将白细胞介素-10用于治疗疾病和病症的方法
US20170202979A1 (en) * 2014-07-17 2017-07-20 Modernatx, Inc. Terminal modifications of polynucleotides
US11351271B2 (en) * 2014-09-08 2022-06-07 Massachusetts Institute Of Technology RNA-based logic circuits with RNA binding proteins, aptamers and small molecules
WO2016049512A1 (en) * 2014-09-26 2016-03-31 University Of Massachusetts Rna-modulating agents
WO2016100812A1 (en) * 2014-12-19 2016-06-23 Moderna Therapeutics, Inc. Terminal modifications of polynucleotides
WO2017013419A1 (en) * 2015-07-20 2017-01-26 Virttu Biologics Limited Use of oncolytic herpes simplex virus, alone or in combination with immune checkpoint inhibitor, in the treatment of cancer
CA3001003A1 (en) * 2015-10-05 2017-04-13 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
CN108474003A (zh) * 2015-11-20 2018-08-31 俄勒冈健康与科学大学 包含微小rna识别元件的cmv载体
EP4089166A1 (en) * 2016-01-27 2022-11-16 Oncorus, Inc. Oncolytic viral vectors and uses thereof
RU2765874C2 (ru) 2016-10-26 2022-02-04 МОДЕРНАТиЭкс, ИНК. Матричные рибонуклеиновые кислоты для усиления иммунных ответов и способы их применения
EP3558356A2 (en) 2016-12-23 2019-10-30 CureVac AG Mers coronavirus vaccine
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
EP4008333A1 (en) 2018-02-19 2022-06-08 Combined Therapeutics, Inc. Compositions and methods for organ-protective expression and modulation of coding ribonucleic acids
IL295724A (en) * 2020-02-21 2022-10-01 Combined Therapeutics Inc Compositions and methods for organ-protective expression and modulation of coding ribonucleic acids

Also Published As

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

Similar Documents

Publication Publication Date Title
US12195746B2 (en) Compositions and processes for targeted delivery, expression and modulation of coding ribonucleic acids in tissue
US20200376142A1 (en) Compositions and methods for organ-protective expression and modulation of coding ribonucleic acids
KR102838785B1 (ko) 암호화 리보핵산의 기관 보호적 발현 및 조절을 위한 조성물 및 방법
RU2803294C2 (ru) Композиции и способы органно-защитной экспрессии и модуляции кодирующих рибонуклеиновых кислот
HK40022397B (en) Compositions and processes for targeted delivery, expression and modulation of coding ribonucleic acids in tissue
HK40022397A (en) Compositions and processes for targeted delivery, expression and modulation of coding ribonucleic acids in tissue
AU2025271119A1 (en) Compositions and methods for organ-protective expression and modulation of coding ribonucleic acids
HK40083636B (zh) 用於编码核糖核酸的器官保护性表达和调节的组合物及方法

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220921

EEER Examination request

Effective date: 20220921

EEER Examination request

Effective date: 20220921

EEER Examination request

Effective date: 20220921

EEER Examination request

Effective date: 20220921