AU2017326535B2 - Cell-specific expression of modrna - Google Patents

Cell-specific expression of modrna Download PDF

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Publication number
AU2017326535B2
AU2017326535B2 AU2017326535A AU2017326535A AU2017326535B2 AU 2017326535 B2 AU2017326535 B2 AU 2017326535B2 AU 2017326535 A AU2017326535 A AU 2017326535A AU 2017326535 A AU2017326535 A AU 2017326535A AU 2017326535 B2 AU2017326535 B2 AU 2017326535B2
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modrna
cms
pkm2
expression
mir
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AU2017326535A1 (en
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Ajit MAGADUM
Lior ZANGI
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Icahn School of Medicine at Mount Sinai
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Icahn School of Medicine at Mount Sinai
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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
    • 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
    • A61K31/7084—Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N15/67—General methods for enhancing the 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657—Cardiomyocytes; Heart cells
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0676—Pancreatic cells

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Cardiology (AREA)
  • Cell Biology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Vascular Medicine (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Rheumatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2017326535A 2016-09-16 2017-09-18 Cell-specific expression of modrna Active AU2017326535B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2023270313A AU2023270313A1 (en) 2016-09-16 2023-11-23 Cell-specific expression of modRNA

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662395701P 2016-09-16 2016-09-16
US62/395,701 2016-09-16
PCT/US2017/052035 WO2018053414A1 (en) 2016-09-16 2017-09-18 Cell-specific expression of modrna

Related Child Applications (1)

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AU2023270313A Division AU2023270313A1 (en) 2016-09-16 2023-11-23 Cell-specific expression of modRNA

Publications (2)

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AU2017326535A1 AU2017326535A1 (en) 2019-04-18
AU2017326535B2 true AU2017326535B2 (en) 2023-08-24

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AU2017326535A Active AU2017326535B2 (en) 2016-09-16 2017-09-18 Cell-specific expression of modrna
AU2023270313A Pending AU2023270313A1 (en) 2016-09-16 2023-11-23 Cell-specific expression of modRNA

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Country Status (9)

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US (3) US11299749B2 (https=)
EP (2) EP3512567B1 (https=)
JP (2) JP7432362B2 (https=)
CN (1) CN110177578B (https=)
AU (2) AU2017326535B2 (https=)
BR (1) BR112019005144A2 (https=)
CA (1) CA3036710A1 (https=)
ES (1) ES2967788T3 (https=)
WO (1) WO2018053414A1 (https=)

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* Cited by examiner, † Cited by third party
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EP3512567B1 (en) * 2016-09-16 2023-11-01 Icahn School of Medicine at Mount Sinai Cell-specific expression of modrna
JP7440868B2 (ja) * 2018-03-30 2024-02-29 国立大学法人京都大学 細胞の製造方法
BR112022002365A2 (pt) 2019-08-09 2022-04-26 Nutcracker Therapeutics Inc Métodos e aparelhos para fabricar e para remover material de uma composição terapêutica
WO2021092440A1 (en) 2019-11-07 2021-05-14 Icahn School Of Medicine At Mount Sinai Synthetic modified rna and uses thereof
EP4058584A4 (en) * 2019-11-11 2023-12-20 Icahn School of Medicine at Mount Sinai GENE DELIVERY SYSTEMS FOR THE TREATMENT OF HEART FAILURE
EP4351623A1 (en) 2021-06-09 2024-04-17 Icahn School of Medicine at Mount Sinai Gene combination as a broad spectrum antiviral
EP4355882A2 (en) * 2021-06-15 2024-04-24 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
CN114085834A (zh) * 2021-11-16 2022-02-25 珠海中科先进技术研究院有限公司 一种癌细胞导向电路组及应用
EP4514965A1 (en) * 2022-04-27 2025-03-05 The Penn State Research Foundation Modrna-based cas endonuclease and base editor and uses thereof
US20230364267A1 (en) * 2022-05-13 2023-11-16 The Uab Research Foundation Pre-clinical modified rna approaches used in large animals for muscle and vascular regeneration
EP4282963A1 (en) * 2022-05-23 2023-11-29 Eberhard Karls Universität Tübingen, Medizinische Fakultät Nucleic acid modified biological cell with expansion-dependent gene expression
WO2024015729A1 (en) * 2022-07-11 2024-01-18 Icahn School Of Medicine At Mount Sinai Regulatory system for expression of a gene of interest in a target cell and method of use thereof
US20260035707A1 (en) * 2022-07-26 2026-02-05 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
CN116785501A (zh) * 2022-08-04 2023-09-22 上海交通大学医学院附属上海儿童医学中心 促进心脏组织修复的modRNA转染改造的细胞及其应用
WO2024151975A1 (en) 2023-01-13 2024-07-18 Igor Lednev Detecting degradation of a polynucleotide by raman spectroscopy
CN119120571B (zh) * 2024-09-11 2026-03-17 武汉科技大学 一种治疗hiv感染的靶向外泌体嵌合抗原受体分子的构建及其应用

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WO2012012739A2 (en) * 2010-07-22 2012-01-26 President And Fellows Of Harvard College Multiple input biologic classifier circuits for cells
WO2015141827A1 (ja) * 2014-03-20 2015-09-24 国立大学法人京都大学 心筋細胞の選別方法
US20150315541A1 (en) * 2012-12-13 2015-11-05 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
WO2016040395A1 (en) * 2014-09-08 2016-03-17 Massachusetts Institute Of Technology Rna-based logic circuits with rna binding proteins, aptamers and small molecules

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JPH08506008A (ja) 1992-11-18 1996-07-02 アーチ ディベロプメント コーポレイション 心筋及び血管平滑筋へのアデノウィルス介在遺伝子移入
US20090005336A1 (en) * 2007-05-08 2009-01-01 Zhiguo Wang Use of the microRNA miR-1 for the treatment, prevention, and diagnosis of cardiac conditions
KR20240136456A (ko) * 2009-12-07 2024-09-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 세포 리프로그래밍을 위한 정제된 변형 ｒｎａ를 포함하는 ｒｎａ 제제
EP2694660B1 (en) 2011-04-03 2018-08-08 The General Hospital Corporation Efficient protein expression in vivo using modified rna (mod-rna)
EP2584040A1 (en) * 2011-10-17 2013-04-24 Pharmahungary 2000 Kft. Compounds for treatment of ischemic injury
CA2915627A1 (en) * 2012-06-15 2013-12-19 The General Hospital Corporation Inhibitors of micrornas that regulate production of atrial natriuretic peptide (anp) as therapeutics and uses thereof
EP3512567B1 (en) * 2016-09-16 2023-11-01 Icahn School of Medicine at Mount Sinai Cell-specific expression of modrna

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WO2012012739A2 (en) * 2010-07-22 2012-01-26 President And Fellows Of Harvard College Multiple input biologic classifier circuits for cells
US20150315541A1 (en) * 2012-12-13 2015-11-05 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
WO2015141827A1 (ja) * 2014-03-20 2015-09-24 国立大学法人京都大学 心筋細胞の選別方法
WO2016040395A1 (en) * 2014-09-08 2016-03-17 Massachusetts Institute Of Technology Rna-based logic circuits with rna binding proteins, aptamers and small molecules

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Also Published As

Publication number Publication date
JP2023123520A (ja) 2023-09-05
EP3512567B1 (en) 2023-11-01
JP2019528736A (ja) 2019-10-17
CA3036710A1 (en) 2018-03-22
CN110177578B (zh) 2024-02-13
US12215336B2 (en) 2025-02-04
US20190203226A1 (en) 2019-07-04
EP3512567A4 (en) 2020-03-18
US20220220501A1 (en) 2022-07-14
EP4316587A3 (en) 2024-04-24
CN110177578A (zh) 2019-08-27
BR112019005144A2 (pt) 2019-06-04
AU2017326535A1 (en) 2019-04-18
US11299749B2 (en) 2022-04-12
US20250154523A1 (en) 2025-05-15
WO2018053414A1 (en) 2018-03-22
ES2967788T3 (es) 2024-05-03
EP4316587A2 (en) 2024-02-07
EP3512567A1 (en) 2019-07-24
JP7432362B2 (ja) 2024-02-16
EP3512567C0 (en) 2023-11-01
AU2023270313A1 (en) 2024-01-04

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