PL3112467T3 - Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek - Google Patents

Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek

Info

Publication number
PL3112467T3
PL3112467T3 PL16181434T PL16181434T PL3112467T3 PL 3112467 T3 PL3112467 T3 PL 3112467T3 PL 16181434 T PL16181434 T PL 16181434T PL 16181434 T PL16181434 T PL 16181434T PL 3112467 T3 PL3112467 T3 PL 3112467T3
Authority
PL
Poland
Prior art keywords
rna
preparations containing
containing purified
cell reprogramming
purified modified
Prior art date
Application number
PL16181434T
Other languages
English (en)
Inventor
Gary Dahl
Judith Meis
Anthony Person
Jerome Jendrisak
Katalin Karikó
Drew Weissman
Original Assignee
The Trustees Of The University Of Pennsylvania
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 The Trustees Of The University Of Pennsylvania filed Critical The Trustees Of The University Of Pennsylvania
Publication of PL3112467T3 publication Critical patent/PL3112467T3/pl

Links

Classifications

    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • 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
    • C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1816—Erythropoietin [EPO]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/14—Drugs for dermatological disorders for baldness or alopecia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/505—Erythropoietin [EPO]
    • 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
    • C12N15/117—Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0073—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen 1.14.13
    • C12N9/0075—Nitric-oxide synthase (1.14.13.39)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/13—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen (1.14.13)
    • C12Y114/13039—Nitric-oxide synthase (NADPH dependent) (1.14.13.39)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/17—Immunomodulatory nucleic acids
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/602—Sox-2
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/603—Oct-3/4
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/604—Klf-4
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/605—Nanog
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/606—Transcription factors c-Myc
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/608—Lin28
    • 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
    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/02—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from embryonic 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
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Transplantation (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pulmonology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Crystallography & Structural Chemistry (AREA)
PL16181434T 2009-12-07 2010-12-07 Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek PL3112467T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US26731209P 2009-12-07 2009-12-07
EP16181434.8A EP3112467B1 (en) 2009-12-07 2010-12-07 Rna preparations comprising purified modified rna for reprogramming cells
EP10836557.8A EP2510099B1 (en) 2009-12-07 2010-12-07 Rna preparations comprising purified modified rna for reprogramming cells

Publications (1)

Publication Number Publication Date
PL3112467T3 true PL3112467T3 (pl) 2018-07-31

Family

ID=44143383

Family Applications (3)

Application Number Title Priority Date Filing Date
PL16181434T PL3112467T3 (pl) 2009-12-07 2010-12-07 Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek
PL10836557T PL2510099T3 (pl) 2009-12-07 2010-12-07 Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek
PL17195966T PL3287525T3 (pl) 2009-12-07 2010-12-07 Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek

Family Applications After (2)

Application Number Title Priority Date Filing Date
PL10836557T PL2510099T3 (pl) 2009-12-07 2010-12-07 Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek
PL17195966T PL3287525T3 (pl) 2009-12-07 2010-12-07 Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek

Country Status (22)

Country Link
US (6) US8808982B2 (pl)
EP (4) EP3623474A1 (pl)
JP (6) JP2013512690A (pl)
KR (5) KR20240136456A (pl)
CN (3) CN114317612B (pl)
AU (1) AU2010328310B2 (pl)
BR (1) BR112012013875B8 (pl)
CA (2) CA3170391A1 (pl)
CY (1) CY1120237T1 (pl)
DK (2) DK3287525T3 (pl)
ES (2) ES2769129T3 (pl)
HK (1) HK1251259B (pl)
HR (2) HRP20180737T1 (pl)
HU (2) HUE036684T2 (pl)
IL (1) IL220219A0 (pl)
LT (2) LT3287525T (pl)
NO (2) NO3112467T3 (pl)
PL (3) PL3112467T3 (pl)
PT (1) PT3112467T (pl)
SG (2) SG181564A1 (pl)
SI (2) SI3112467T1 (pl)
WO (2) WO2011071931A2 (pl)

Families Citing this family (158)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
US9249423B2 (en) 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
US10155038B2 (en) 2007-02-02 2018-12-18 Yale University Cells prepared by transient transfection and methods of use thereof
WO2008097926A2 (en) * 2007-02-02 2008-08-14 Yale University Transient transfection with rna
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
US8808982B2 (en) * 2009-12-07 2014-08-19 Cellscript, Llc Compositions and methods for reprogramming eukaryotic cells
US8802438B2 (en) 2010-04-16 2014-08-12 Children's Medical Center Corporation Compositions, kits, and methods for making induced pluripotent stem cells using synthetic modified RNAs
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
EP2591114B1 (en) 2010-07-06 2016-06-08 GlaxoSmithKline Biologicals SA Immunisation of large mammals with low doses of rna
ES2646669T3 (es) 2010-07-06 2017-12-14 Glaxosmithkline Biologicals Sa Procedimientos de aumento de una respuesta inmunitaria mediante el suministro de ARN
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
LT4226941T (lt) 2010-08-31 2025-01-10 Glaxosmithkline Biologicals Sa Pegilintos liposomos, skirtos imunogeną koduojančios rnr pristatymui
EP2625189B1 (en) 2010-10-01 2018-06-27 ModernaTX, Inc. Engineered nucleic acids and methods of use thereof
WO2012051211A2 (en) 2010-10-11 2012-04-19 Novartis Ag Antigen delivery platforms
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
EP2729165B1 (en) 2011-07-06 2017-11-08 GlaxoSmithKline Biologicals SA Immunogenic combination compositions and uses thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
DE19216461T1 (de) * 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
AU2012347919B2 (en) * 2011-12-05 2017-02-02 Factor Bioscience Inc. Methods and products for transfecting cells
WO2013090186A1 (en) * 2011-12-14 2013-06-20 modeRNA Therapeutics Modified nucleic acids, and acute care uses thereof
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
CN104968354A (zh) * 2011-12-21 2015-10-07 现代治疗公司 增加器官或器官外植体的活力或寿命的方法
EP3144389B1 (en) 2011-12-30 2018-05-09 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
CN104411338A (zh) * 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
EP3561054B1 (en) * 2012-05-13 2023-07-05 Allele Biotechnology And Pharmaceuticals, Inc. Feeder-free derivation of human-induced pluripotent stem cells with synthetic messenger rna
EP2858679B2 (en) * 2012-06-08 2024-06-05 Translate Bio, Inc. Pulmonary delivery of mrna to non-lung target cells
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2014028429A2 (en) 2012-08-14 2014-02-20 Moderna Therapeutics, Inc. Enzymes and polymerases for the synthesis of rna
JP2015527084A (ja) 2012-09-07 2015-09-17 ジェネンテック， インコーポレイテッド 誘導肝細胞を作製するための方法及び組成物
US9717749B2 (en) 2012-10-16 2017-08-01 Massachusetts Institute Of Technology Production of stable non-polyadenylated RNAs
KR102121086B1 (ko) 2012-11-01 2020-06-09 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
EP4331620A3 (en) * 2012-12-07 2024-12-04 Translate Bio, Inc. Lipidic nanoparticles for mrna delivery
AU2014218667A1 (en) 2013-02-22 2015-10-08 The Board Of Trustees Of The Leland Stanford Junior University Compounds, compositions, methods, and kits relating to telomere extension
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
RS57739B1 (sr) 2013-03-14 2018-12-31 Translate Bio Inc Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
US20160032316A1 (en) * 2013-03-14 2016-02-04 The Trustees Of The University Of Pennsylvania Purification and Purity Assessment of RNA Molecules Synthesized with Modified Nucleosides
JP6586075B2 (ja) 2013-03-14 2019-10-02 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａの精製方法
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014152031A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Ribonucleic acid purification
EP2971165A4 (en) 2013-03-15 2016-11-23 Moderna Therapeutics Inc DISSOLUTION OF DNA FRAGMENTS IN MRNA MANUFACTURING METHODS
US10590161B2 (en) 2013-03-15 2020-03-17 Modernatx, Inc. Ion exchange purification of mRNA
WO2014152027A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Manufacturing methods for production of rna transcripts
EP2996697B1 (en) 2013-05-15 2019-06-26 Robert Kruse Intracellular translation of circular rna
CA2917348A1 (en) 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
BR112016001487A2 (pt) * 2013-07-26 2017-08-29 Kyoto Prefectural Public Univ Corp Osteoblasto e método para preparar o mesmo
KR102758333B1 (ko) * 2013-08-28 2025-01-23 벡톤 디킨슨 앤드 컴퍼니 대량의 동시 단일 세포 분석
EP3041934A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
CN106413811A (zh) 2013-10-22 2017-02-15 夏尔人类遗传性治疗公司 精氨基琥珀酸合成酶缺乏症的mrna疗法
JP6506749B2 (ja) 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのｍＲＮＡ療法
WO2015062738A1 (en) 2013-11-01 2015-05-07 Curevac Gmbh Modified rna with decreased immunostimulatory properties
EP3062798B1 (en) 2013-11-01 2020-05-06 CureVac AG Modified rna with decreased immunostimulatory properties
KR101665165B1 (ko) * 2013-12-04 2016-10-12 단국대학교 산학협력단 신규한 rna 앱타머 및 그의 용도
EP3099801B1 (en) 2014-01-31 2020-03-18 Factor Bioscience Inc. Synthetic rna for use in the treatment of dystrophic epidermolysis bullosa
ES2895941T3 (es) * 2014-02-10 2022-02-23 Univ Leland Stanford Junior Activación de la inmunidad innata para una reprogramación nuclear mejorada de células somáticas con ARNm
PL4023249T3 (pl) 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
US9850269B2 (en) 2014-04-25 2017-12-26 Translate Bio, Inc. Methods for purification of messenger RNA
WO2015196128A2 (en) 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
US10407683B2 (en) 2014-07-16 2019-09-10 Modernatx, Inc. Circular polynucleotides
US20170204152A1 (en) 2014-07-16 2017-07-20 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2016077125A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
CA2976376A1 (en) 2015-02-13 2016-08-18 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
GB2560250A (en) 2015-06-30 2018-09-05 Ethris Gmbh ATP-Binding cassette family coding polyribonucleotides and formulations thereof
US10662409B2 (en) * 2015-09-09 2020-05-26 Trustees Of Tufts College Methods of generating neural stem cells
EP3350333B2 (en) 2015-09-17 2025-08-06 ModernaTX, Inc. Polynucleotides containing a stabilizing tail region
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
JP7001585B2 (ja) * 2015-09-23 2022-02-04 ファイザー・インク 細胞および細胞培養の方法
EP3359670B2 (en) 2015-10-05 2024-02-14 ModernaTX, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
US11446398B2 (en) 2016-04-11 2022-09-20 Obsidian Therapeutics, Inc. Regulated biocircuit systems
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
SI3445850T1 (sl) 2016-04-22 2021-12-31 BioNTech SE Postopki za zagotavljanje enoverižne RNA
IL263079B2 (en) 2016-05-18 2024-05-01 Modernatx Inc Polynucleotides encoding relaxin
EP3463383A4 (en) * 2016-06-03 2020-02-12 Stemgenics, Inc. DIRECT REPROGRAMMING OF A HUMAN SOMATIC CELL IN A DIFFERENTIATED (PREDETERMINED) CELL SELECTED WITH FUNCTIONALIZED NANOPARTICLES
WO2017223176A1 (en) 2016-06-24 2017-12-28 Modernatx, Inc. Methods and apparatus for filtration
CN106226524B (zh) * 2016-07-07 2018-10-09 福建省农业科学院食用菌研究所 一种食用菌dsRNA病毒的检测方法
IL308824A (en) 2016-08-17 2024-01-01 Factor Bioscience Inc Nucleic acid products and methods of administration thereof
CA3036831A1 (en) 2016-09-14 2018-03-22 Modernatx, Inc. High purity rna compositions and methods for preparation thereof
CA3036710A1 (en) * 2016-09-16 2018-03-22 Icahn School Of Medicine At Mount Sinai Cell-specific expression of modrna
MA46766A (fr) 2016-11-11 2019-09-18 Modernatx Inc Vaccin antigrippal
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
TW202428301A (zh) 2017-02-28 2024-07-16 法商賽諾菲公司 治療性rna
KR102746901B1 (ko) 2017-03-03 2024-12-26 옵시디안 테라퓨틱스, 인크. Cd19 조성물 및 면역요법을 위한 방법
CA3055200A1 (en) 2017-03-03 2018-09-07 Obsidian Therapeutics, Inc. Compositions and methods for immunotherapy
WO2018188730A1 (en) * 2017-04-11 2018-10-18 Biontech Rna Pharmaceuticals Gmbh Rna for treatment of autoimmune diseases
KR102746223B1 (ko) 2017-04-20 2024-12-27 이제네시스, 인크. 유전자 변형 동물의 생성 방법
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
US10034951B1 (en) 2017-06-21 2018-07-31 New England Biolabs, Inc. Use of thermostable RNA polymerases to produce RNAs having reduced immunogenicity
MA49914A (fr) 2017-08-18 2021-04-21 Modernatx Inc Procédés analytiques par hplc
EP3668979A4 (en) 2017-08-18 2021-06-02 Modernatx, Inc. METHOD OF HPLC ANALYSIS
CN107563149B (zh) * 2017-08-21 2020-10-23 上海派森诺生物科技股份有限公司 全长转录本的结构注释和比对结果评估方法
EP3675817A1 (en) 2017-08-31 2020-07-08 Modernatx, Inc. Methods of making lipid nanoparticles
KR102762959B1 (ko) * 2017-09-13 2025-02-06 트론-트란슬라셔날레 온콜로기 안 데어 유니버시태츠메디진 데어 요하네스 구텐베르크-유니버시태트 마인츠 게마인뉘치게 게엠베하 세포에서 rna 발현을 향상시키는 방법
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
KR20200131301A (ko) * 2018-03-13 2020-11-23 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 세포 노화의 역전을 위한 일시적 세포 재프로그래밍
WO2019241315A1 (en) 2018-06-12 2019-12-19 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
CN108676855B (zh) * 2018-07-24 2021-09-28 内蒙古赛科星家畜种业与繁育生物技术研究院有限公司 标准曲线法荧光定量pcr鉴定牛转基因拷贝数的方法及引物
KR102950141B1 (ko) 2018-08-24 2026-04-07 트랜슬레이트 바이오 인코포레이티드 전령 rna의 정제 방법
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
US11072808B2 (en) 2018-10-04 2021-07-27 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed RNA
AU2019355177A1 (en) 2018-10-04 2021-05-06 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed RNA
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
US12174174B2 (en) * 2018-10-31 2024-12-24 National Cancer Center Composition comprising material for regulating OCT4 modification to repress stemness
WO2020106946A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
WO2020168466A1 (en) * 2019-02-19 2020-08-27 Stemirna Therapeutics Co., Ltd. Modified nucleoside and synthetic methods thereof
CN113874502A (zh) 2019-03-11 2021-12-31 摩登纳特斯有限公司 补料分批体外转录方法
CA3145425A1 (en) 2019-07-03 2021-01-07 Factor Bioscience Inc. Cationic lipids and uses thereof
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
CN114269918A (zh) * 2019-08-14 2022-04-01 摩登纳特斯有限公司 用于纯化体外转录的下游产物的方法
CN112390838A (zh) * 2019-08-14 2021-02-23 斯微(上海)生物科技有限公司 一种改性核苷及其合成方法
WO2021050989A1 (en) 2019-09-13 2021-03-18 Duke University Zika antibodies and their use
EP4031662A1 (en) 2019-09-20 2022-07-27 Translate Bio, Inc. Mrna encoding engineered cftr
US12559527B2 (en) 2019-10-23 2026-02-24 Duke University Compositions comprising V2 opt HIV envelopes
CN110894493A (zh) * 2019-10-28 2020-03-20 吉林大学 一种重编程间充质干细胞及其制备方法
WO2021096998A1 (en) * 2019-11-11 2021-05-20 Atsuo Ochi Method and a kit to reprogram somatic cells
JP2023507501A (ja) * 2019-12-23 2023-02-22 ヴェルサメブ アーゲー 遺伝子の発現を同時に調節するための組成物及び方法
WO2021149824A1 (ja) * 2020-01-24 2021-07-29 アイ ピース， インコーポレイテッド 人工多能性幹細胞の作製方法
US12194089B2 (en) 2020-02-04 2025-01-14 CureVac SE Coronavirus vaccine
KR20210116324A (ko) 2020-03-12 2021-09-27 기초과학연구원 게놈 서열 변이를 가지는 세포 사멸 유도용 조성물 및 상기 조성물을 이용한 세포 사멸 유도 방법
FI20215508A1 (en) 2020-04-09 2021-10-10 Niemelae Erik Johan Mimetic nanoparticles to prevent the spread of new coronaviruses and reduce the rate of infection
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
AU2021380845A1 (en) * 2020-11-13 2023-06-15 Egenesis, Inc. Cells, tissues, organs, and animals having one or more modified genes for enhanced xenograft survival and tolerance
US11918643B2 (en) 2020-12-22 2024-03-05 CureVac SE RNA vaccine against SARS-CoV-2 variants
JP2022104813A (ja) 2020-12-29 2022-07-11 アイ ピース，インコーポレイテッド リプログラミング因子を導入された細胞の培養方法
EP4274607A1 (en) 2021-01-11 2023-11-15 ModernaTX, Inc. Seasonal rna influenza virus vaccines
CN113408945B (zh) * 2021-07-15 2023-03-24 广西中烟工业有限责任公司 一种烤烟纯度的检测方法、装置、电子设备及存储介质
US20230042860A1 (en) * 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Polycistronic expression vectors
US20230044997A1 (en) 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs and methods of use for cell rejuvenation and for treatment
WO2023288288A1 (en) 2021-07-15 2023-01-19 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs with on/off mechanism for controlled expression and methods of use
CN114196676B (zh) * 2021-11-29 2023-07-18 中国农业科学院北京畜牧兽医研究所 Itga2基因在调控猪米色脂肪形成中的应用
CN114561381A (zh) * 2022-03-14 2022-05-31 桂林医学院 免疫mRNA及其制备方法和应用
CN114540387B (zh) * 2022-03-28 2024-04-26 仁景(苏州)生物科技有限公司 一种ires序列介导的非帽依赖型的基因表达载体及其应用
EP4356933A1 (en) 2022-09-07 2024-04-24 Eyegene, Inc. Composition for delivering modified nucleic acid-containing mrna
KR102785258B1 (ko) * 2022-09-07 2025-03-26 아이진 주식회사 변형핵산 함유 mRNA의 체내 전달용 조성물
EP4608442A1 (en) 2022-10-28 2025-09-03 GlaxoSmithKline Biologicals S.A. Nucleic acid based vaccine
CN115992146B (zh) * 2022-11-11 2025-04-18 上海交通大学医学院附属第九人民医院 经修饰的mRNA分子及相关产品和用途
WO2024129826A2 (en) * 2022-12-14 2024-06-20 Grann Pharmaceuticals Inc. Compositions and methods for modulating hsp70 activity
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025024442A1 (en) * 2023-07-26 2025-01-30 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with bsep deficiency
CN116898988A (zh) * 2023-08-15 2023-10-20 臻赫医药(杭州)有限公司 皮肤重编程因子制剂、生物材料及用途
WO2025038982A2 (en) * 2023-08-17 2025-02-20 Duke University Regulators of t cell exhaustion
WO2025144938A1 (en) 2023-12-26 2025-07-03 Emmune, Inc. Systems for nucleic acid transfer
CN117511947B (zh) * 2024-01-08 2024-03-29 艾斯拓康医药科技(北京)有限公司 一种优化的5`utr序列及其应用
KR20250110708A (ko) 2024-01-11 2025-07-21 서울대학교산학협력단 역분화 유전자의 간 특이적 전달을 통한 간 재생 방법
CN119307519B (zh) * 2024-07-22 2025-11-25 苏州剂泰医药科技有限公司 编码荧光素酶的mRNA及其应用

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5824497A (en) * 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
WO1998059076A1 (en) * 1997-06-25 1998-12-30 Promega Corporation Method of isolating rna
US20030083272A1 (en) * 1997-09-19 2003-05-01 Lahive & Cockfield, Llp Sense mrna therapy
WO2002065093A2 (en) 2001-02-14 2002-08-22 Baylor College Of Medicine Methods and compositions of amplifying rna
FR2822164B1 (fr) 2001-03-19 2004-06-18 Centre Nat Rech Scient Polypeptides derives des arn polymerases, et leurs utilisations
US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
US8137911B2 (en) 2001-05-22 2012-03-20 Cellscript, Inc. Preparation and use of single-stranded transcription substrates for synthesis of transcription products corresponding to target sequences
WO2004048594A2 (en) 2002-11-21 2004-06-10 Epicentre Technologies Preparation and use of single-stranded transcription substrates for synthesis of transcription products corresponding to target sequences
US9567591B2 (en) * 2003-05-15 2017-02-14 Mello Biotechnology, Inc. Generation of human embryonic stem-like cells using intronic RNA
US20050153333A1 (en) 2003-12-02 2005-07-14 Sooknanan Roy R. Selective terminal tagging of nucleic acids
EP1773857A4 (en) 2004-07-02 2009-05-13 Protiva Biotherapeutics Inc THE IMMUNE SYSTEM STIMULATING SIRNA MOLECULES AND APPLICATIONS THEREOF
PL2578685T3 (pl) * 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
US20070087437A1 (en) * 2005-10-14 2007-04-19 Jifan Hu Methods for rejuvenating cells in vitro and in vivo
EP2010659B1 (en) 2006-04-14 2014-06-18 CellScript, Inc. Kits and methods for generating 5' capped RNA
DE102006051516A1 (de) * 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
KR101107256B1 (ko) 2007-03-27 2012-01-19 삼성전자주식회사 모션 벡터에 기반한 적응적 프레임율 변환 방법 및 장치 및적응적 프레임율 변환 기능을 가지는 디스플레이 장치
EP2164951A2 (en) * 2007-05-30 2010-03-24 The General Hospital Corporation Methods of generating pluripotent cells from somatic cells
US8039214B2 (en) 2007-06-29 2011-10-18 Cellscript, Inc. Synthesis of tagged nucleic acids
EP2072618A1 (en) * 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
GB0801215D0 (en) * 2008-01-23 2008-02-27 Univ Sheffield Cell re-programming
US20100330677A1 (en) 2008-02-11 2010-12-30 Cambridge Enterprise Limited Improved Reprogramming of Mammalian Cells, and Cells Obtained
WO2009127230A1 (en) * 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
US8669048B2 (en) * 2008-06-24 2014-03-11 Parkinson's Institute Pluripotent cell lines and methods of use thereof
GB0905507D0 (en) 2009-03-31 2009-05-13 Dow Corning Organopol Ysiloxane Compositions Containing An Active Material
WO2010123501A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
CA2769670C (en) 2009-07-31 2018-10-02 Ethris Gmbh Rna with a combination of unmodified and modified nucleotides for protein expression
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
US8808982B2 (en) * 2009-12-07 2014-08-19 Cellscript, Llc Compositions and methods for reprogramming eukaryotic cells
US8802438B2 (en) * 2010-04-16 2014-08-12 Children's Medical Center Corporation Compositions, kits, and methods for making induced pluripotent stem cells using synthetic modified RNAs
WO2012019168A2 (en) * 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2625189B1 (en) 2010-10-01 2018-06-27 ModernaTX, Inc. Engineered nucleic acids and methods of use thereof
US8765414B2 (en) * 2011-03-15 2014-07-01 The Board Of Trustees Of The Leland Stanford Junior University GPCR fusion protein containing an N-terminal autonomously folding stable domain, and crystals of the same
AU2012236099A1 (en) * 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
WO2013003475A1 (en) 2011-06-27 2013-01-03 Cellscript, Inc. Inhibition of innate immune response
EP3144389B1 (en) 2011-12-30 2018-05-09 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect

Also Published As

Publication number Publication date
AU2010328310B2 (en) 2015-05-21
WO2011071931A2 (en) 2011-06-16
SG10201408129XA (en) 2015-02-27
DK3112467T3 (en) 2018-04-23
US20140315988A1 (en) 2014-10-23
JP7763277B2 (ja) 2025-10-31
HRP20200246T1 (hr) 2020-05-29
WO2011071936A3 (en) 2011-10-13
JP7152541B2 (ja) 2022-10-12
KR102703156B1 (ko) 2024-09-06
JP2019162125A (ja) 2019-09-26
DK3287525T3 (da) 2020-01-20
CA2783032A1 (en) 2011-06-16
BR112012013875A2 (pt) 2015-11-03
ES2769129T3 (es) 2020-06-24
US20210024895A1 (en) 2021-01-28
EP3287525A1 (en) 2018-02-28
US20180265848A1 (en) 2018-09-20
CN114317612B (zh) 2024-10-29
AU2010328310A1 (en) 2012-06-28
SI3287525T1 (sl) 2020-03-31
CN107090436B (zh) 2021-11-30
PL3287525T3 (pl) 2020-05-18
KR101878502B1 (ko) 2018-07-13
JP2021078516A (ja) 2021-05-27
CN102947450A (zh) 2013-02-27
JP2024041917A (ja) 2024-03-27
CA2783032C (en) 2022-08-30
HUE036684T2 (hu) 2018-07-30
EP3287525B1 (en) 2019-10-23
BR112012013875B8 (pt) 2020-06-23
KR20230035422A (ko) 2023-03-13
SG181564A1 (en) 2012-07-30
CN114317612A (zh) 2022-04-12
CA3170391A1 (en) 2011-06-16
NO2510099T3 (pl) 2018-01-06
US20110143436A1 (en) 2011-06-16
US20160251629A1 (en) 2016-09-01
JP6842495B2 (ja) 2021-03-17
EP2510099A2 (en) 2012-10-17
EP3112467A1 (en) 2017-01-04
EP3112467B1 (en) 2018-02-14
KR20240136456A (ko) 2024-09-13
BR112012013875B1 (pt) 2020-04-14
EP3623474A1 (en) 2020-03-18
US11739300B2 (en) 2023-08-29
EP2510099B1 (en) 2017-08-09
HRP20180737T1 (hr) 2018-06-29
NO3112467T3 (pl) 2018-07-14
LT3287525T (lt) 2020-02-10
SI3112467T1 (en) 2018-06-29
WO2011071936A2 (en) 2011-06-16
EP2510099A4 (en) 2014-06-11
CN102947450B (zh) 2016-11-23
KR102171849B1 (ko) 2020-10-30
IL220219A0 (en) 2012-07-31
JP2016171804A (ja) 2016-09-29
PL2510099T3 (pl) 2018-01-31
US8808982B2 (en) 2014-08-19
ES2665681T3 (es) 2018-04-26
US10006007B2 (en) 2018-06-26
KR102505097B1 (ko) 2023-03-02
JP7475406B2 (ja) 2024-04-26
WO2011071931A3 (en) 2011-11-17
KR20180081836A (ko) 2018-07-17
CY1120237T1 (el) 2019-07-10
JP2022185047A (ja) 2022-12-13
LT3112467T (lt) 2018-06-25
KR20200124331A (ko) 2020-11-02
US20160369243A1 (en) 2016-12-22
JP2013512690A (ja) 2013-04-18
US9371544B2 (en) 2016-06-21
US11028370B2 (en) 2021-06-08
CN107090436A (zh) 2017-08-25
PT3112467T (pt) 2018-04-16
HK1251259B (en) 2020-07-03
HUE047165T2 (hu) 2020-04-28
KR20130009944A (ko) 2013-01-24

Similar Documents

Publication Publication Date Title
PL3112467T3 (pl) Preparaty RNA zawierające oczyszczony zmodyfikowany RNA do przeprogramowywania komórek
IL254144A0 (en) Methods for reprogramming cells and uses thereof
HUE062860T2 (hu) Magas koncentrációjú gyógyszerészeti készítmények
PL2634242T3 (pl) Ulepszony sposób hodowli komórek
FI20106121A7 (fi) Soluviljelymateriaali
HUE041436T2 (hu) Tal-effektor-közvetített DNS-módosítás
DK3192865T3 (da) Enkelt pluripotent stamcelle-kultur
BRPI1010035A2 (pt) Células deficientes em fucosilação
DK2411028T3 (da) Terapi til fremme af cellevækst
DK3495498T3 (da) Genekspressionsanalyse i enkeltceller
DK2344682T3 (da) Elektrode for elektrolysecelle
IL244941A (en) Insulin preparations containing methionine
EP2681306A4 (en) CELL CULTURE SYSTEM
PL2379085T3 (pl) Preparat farmaceutyczny zawierający supernatant z hodowli komórek jednojądrzastych
DK2598514T3 (da) Proteinoprensning
HUE036071T2 (hu) Macitentánt tartalmazó terápiás készítmények
EP2516615A4 (en) IMPROVED BIOREACTORS
IL225785A0 (en) Heterocyclic compounds, compositions comprising the same and uses thereof
EP3524672C0 (en) REPROGRAMMING CANCER CELLS
EP2475430A4 (en) GRANULATED ANTHELMINTIC PREPARATIONS AND OUTPUT SYSTEMS THEREFOR
IL220376A (en) Polycyclic heterocyclic compounds, process for their preparation, intermediates obtained in said process and pharmaceutical compositions comprising said compounds
EP2271739A4 (en) PRODUCT FOR CELL CULTURE
AU2024266827A1 (en) RNA preparations comprising purified modified RNA for reprogramming cells
DK2401556T3 (da) Planart elektrodesystem
FI20095795A0 (fi) Menetelmä biologiseen puhdistamiseen