PL3144389T3 - WYTWARZANIE I STOSOWANIE ZSYNTETYZOWANEGO IN VITRO ssRNA DO WPROWADZANIA DO SSACZYCH KOMÓREK W CELU INDUKCJI EFEKTU BIOLOGICZNEGO LUB BIOCHEMICZNEGO - Google Patents

WYTWARZANIE I STOSOWANIE ZSYNTETYZOWANEGO IN VITRO ssRNA DO WPROWADZANIA DO SSACZYCH KOMÓREK W CELU INDUKCJI EFEKTU BIOLOGICZNEGO LUB BIOCHEMICZNEGO

Info

Publication number
PL3144389T3
PL3144389T3 PL16186283T PL16186283T PL3144389T3 PL 3144389 T3 PL3144389 T3 PL 3144389T3 PL 16186283 T PL16186283 T PL 16186283T PL 16186283 T PL16186283 T PL 16186283T PL 3144389 T3 PL3144389 T3 PL 3144389T3
Authority
PL
Poland
Prior art keywords
vitro
induce
biological
introducing
making
Prior art date
Application number
PL16186283T
Other languages
English (en)
Inventor
Judith Meis
Anthony D. PERSON
Cynthia S. Chin
Jerome Jendrisak
Gary A. Dahl
Original Assignee
Cellscript, Llc
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 Cellscript, Llc filed Critical Cellscript, Llc
Publication of PL3144389T3 publication Critical patent/PL3144389T3/pl

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K33/00—Medicinal preparations containing inorganic active ingredients
    • A61K33/06—Aluminium, calcium or magnesium; Compounds thereof, e.g. clay
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/005—Enzyme inhibitors
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • C12N15/1003—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor
    • 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
    • C12N15/1096—Processes for the isolation, preparation or purification of DNA or RNA cDNA Synthesis; Subtracted cDNA library construction, e.g. RT, RT-PCR
    • 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
    • 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
    • C12N5/0618—Cells of the nervous system
    • C12N5/0619—Neurons
    • 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
    • C12N5/0658—Skeletal muscle cells, e.g. myocytes, myotubes, myoblasts
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/38—Nucleosides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • 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
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/70—Enzymes
    • C12N2501/73—Hydrolases (EC 3.)
    • 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
    • C12N2506/13—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from connective tissue cells, from mesenchymal cells
    • C12N2506/1307—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from connective tissue cells, from mesenchymal cells from adult fibroblasts
    • 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)
    • C12Y301/26—Endoribonucleases producing 5'-phosphomonoesters (3.1.26)
    • C12Y301/26003—Ribonuclease III (3.1.26.3)
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Rheumatology (AREA)
  • Neurosurgery (AREA)
  • Developmental Biology & Embryology (AREA)
  • Transplantation (AREA)
  • Immunology (AREA)
  • Analytical Chemistry (AREA)
  • Inorganic Chemistry (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
PL16186283T 2011-12-30 2012-12-31 WYTWARZANIE I STOSOWANIE ZSYNTETYZOWANEGO IN VITRO ssRNA DO WPROWADZANIA DO SSACZYCH KOMÓREK W CELU INDUKCJI EFEKTU BIOLOGICZNEGO LUB BIOCHEMICZNEGO PL3144389T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161582080P 2011-12-30 2011-12-30
US201161582050P 2011-12-30 2011-12-30
US201261651738P 2012-05-25 2012-05-25
EP12815983.7A EP2798064B1 (en) 2011-12-30 2012-12-31 Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect
EP16186283.4A EP3144389B1 (en) 2011-12-30 2012-12-31 Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect

Publications (1)

Publication Number Publication Date
PL3144389T3 true PL3144389T3 (pl) 2018-10-31

Family

ID=47559751

Family Applications (2)

Application Number Title Priority Date Filing Date
PL16186283T PL3144389T3 (pl) 2011-12-30 2012-12-31 WYTWARZANIE I STOSOWANIE ZSYNTETYZOWANEGO IN VITRO ssRNA DO WPROWADZANIA DO SSACZYCH KOMÓREK W CELU INDUKCJI EFEKTU BIOLOGICZNEGO LUB BIOCHEMICZNEGO
PL18170777T PL3421601T3 (pl) 2011-12-30 2012-12-31 Wytwarzanie i stosowanie zsyntetyzowanego in vitro ssRNA do wprowadzania do ssaczych komórek w celu indukcji efektu biologicznego lub biochemicznego

Family Applications After (1)

Application Number Title Priority Date Filing Date
PL18170777T PL3421601T3 (pl) 2011-12-30 2012-12-31 Wytwarzanie i stosowanie zsyntetyzowanego in vitro ssRNA do wprowadzania do ssaczych komórek w celu indukcji efektu biologicznego lub biochemicznego

Country Status (13)

Country Link
US (3) US10201620B2 (pl)
EP (4) EP3421601B1 (pl)
AU (4) AU2012362113B2 (pl)
CA (1) CA2862377A1 (pl)
DK (3) DK2798064T3 (pl)
ES (2) ES2770314T3 (pl)
HK (1) HK1203559A1 (pl)
HR (1) HRP20200251T1 (pl)
HU (1) HUE047947T2 (pl)
LT (2) LT3144389T (pl)
PL (2) PL3144389T3 (pl)
SI (1) SI3421601T1 (pl)
WO (1) WO2013102203A1 (pl)

Families Citing this family (85)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
LT2578685T (lt) 2005-08-23 2019-06-10 The Trustees Of The University Of Pennsylvania Rnr, apimančios modifikuotus nukleozidus ir jų panaudojimo būdai
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
RS57314B1 (sr) 2009-12-01 2018-08-31 Translate Bio Inc Isporuka irnk za povećanje ekspresije proteina i enzima u humanim genetskim oboljenjima
EP3112467B1 (en) 2009-12-07 2018-02-14 The Trustees of the University of Pennsylvania Rna preparations comprising purified modified rna for reprogramming cells
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
CA3162352A1 (en) 2010-10-01 2012-04-05 Modernatx, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
JP6184945B2 (ja) 2011-06-08 2017-08-23 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 ｍＲＮＡ送達のための脂質ナノ粒子組成物および方法
BR112014013664A2 (pt) 2011-12-05 2020-11-03 Factor Bioscience Inc. métodos e produtos para transfectar células
LT2791160T (lt) 2011-12-16 2022-06-10 Modernatx, Inc. Modifikuotos mrnr sudėtys
DK2798064T3 (en) 2011-12-30 2016-12-19 Cellscript Llc PRODUCTION AND USE OF IN VITRO synthesized ssRNA TO FEED IN MAMMALIAN CELLS FOR INDUCTION OF A BIOLOGICAL OR BIOCHEMICAL EFFECT
US9303079B2 (en) 2012-04-02 2016-04-05 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
CA2868996A1 (en) 2012-04-02 2013-10-10 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
BR122019025678B1 (pt) 2012-11-01 2023-04-18 Factor Bioscience Inc Composições que compreendem ácido nucleico que codifica uma proteína de edição gênica
ES2676470T3 (es) * 2012-11-09 2018-07-19 Biontech Rna Pharmaceuticals Gmbh Método para la expresión de ARN en células
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
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
TR201901310T4 (tr) 2013-03-14 2019-02-21 Translate Bio Inc Mesajcı RNA'nın saflaştırılması yöntemleri.
MX365409B (es) 2013-03-14 2019-05-31 Shire Human Genetic Therapies Composiciones de ácido ribonucleico mensajero del regulador transmembrana de fibrosis quística y métodos y usos relacionados.
SI3019619T1 (sl) 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
EP3026112B1 (en) 2013-07-26 2022-09-21 Kyoto Prefectural Public University Corporation Osteoblast and method for preparing same
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
JP2016538829A (ja) 2013-10-03 2016-12-15 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 低密度リポタンパク質受容体をコードするポリヌクレオチド
WO2015061500A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Mrna therapy for argininosuccinate synthetase deficiency
EA201992208A1 (ru) 2013-10-22 2020-07-31 Транслейт Био, Инк. ЛЕЧЕНИЕ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
RU2020104969A (ru) 2014-01-31 2020-03-13 Фэктор Байосайенс Инк. Способы и продукты для получения и доставки нуклеиновых кислот
CA2944800A1 (en) 2014-04-25 2015-10-29 Shire Human Genetic Therapies, Inc. Methods for purification of messenger rna
CA2976376A1 (en) 2015-02-13 2016-08-18 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
EP3324979B1 (en) 2015-07-21 2022-10-12 ModernaTX, Inc. Infectious disease vaccines
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
US10849920B2 (en) 2015-10-05 2020-12-01 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
PL3718565T3 (pl) 2015-10-22 2022-09-19 Modernatx, Inc. Szczepionki przeciwko wirusom układu oddechowego
WO2017070613A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Human cytomegalovirus vaccine
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
US11007213B2 (en) 2016-04-01 2021-05-18 University Of Iowa Research Foundation Metabolically stabilized double stranded mRNA
ES2900272T3 (es) 2016-04-22 2022-03-16 BioNTech SE Métodos para proporcionar ARN monocatenario
JP2019528284A (ja) 2016-08-17 2019-10-10 ファクター バイオサイエンス インコーポレイテッド 核酸産物およびその投与方法
AU2017326423B2 (en) 2016-09-14 2023-11-09 Modernatx, Inc. High purity RNA compositions and methods for preparation thereof
JOP20190009A1 (ar) 2016-09-21 2019-01-27 Alx Oncology Inc أجسام مضادة ضد بروتين ألفا منظم للإشارات وطرق استخدامها
WO2018073615A1 (en) 2016-10-21 2018-04-26 Longboat Explorers Ab Methods and compositions for generating hematopoietic cells
MA46584A (fr) 2016-10-21 2019-08-28 Modernatx Inc Vaccin contre le cytomégalovirus humain
WO2018106536A1 (en) * 2016-12-06 2018-06-14 The Regents Of The University Of California Methods for making and using dedifferentiated and stem-like human cells
WO2018107088A2 (en) 2016-12-08 2018-06-14 Modernatx, Inc. Respiratory virus nucleic acid vaccines
US11384352B2 (en) 2016-12-13 2022-07-12 Modernatx, Inc. RNA affinity purification
EP3582790A4 (en) 2017-02-16 2020-11-25 ModernaTX, Inc. VERY POWERFUL IMMUNOGENIC COMPOSITIONS
CA3054062A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Novel codon-optimized cftr mrna
MA47790A (fr) 2017-03-17 2021-05-05 Modernatx Inc Vaccins à base d'arn contre des maladies zoonotiques
TWI796329B (zh) 2017-04-07 2023-03-21 美商默沙東有限責任公司 抗－ilt4抗體及抗原結合片段
WO2018188730A1 (en) 2017-04-11 2018-10-18 Biontech Rna Pharmaceuticals Gmbh Rna for treatment of autoimmune diseases
WO2018213476A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
US11377643B2 (en) 2017-05-31 2022-07-05 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type III
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
US10034951B1 (en) * 2017-06-21 2018-07-31 New England Biolabs, Inc. Use of thermostable RNA polymerases to produce RNAs having reduced immunogenicity
EP3668971B1 (en) * 2017-08-18 2024-04-10 ModernaTX, Inc. Rna polymerase variants
JP7275111B2 (ja) 2017-08-31 2023-05-17 モデルナティエックス インコーポレイテッド 脂質ナノ粒子の生成方法
WO2019055807A1 (en) 2017-09-14 2019-03-21 Modernatx, Inc. RNA VACCINES AGAINST ZIKA VIRUS
EP3752603A1 (en) 2018-02-16 2020-12-23 The United States of America, as represented by the Secretary, Department of Health and Human Services Treatment of infections and immune dysregulation in patients with primary immune deficiencies using mrna-corrected autologous granulocytes, lymphocytes and/or natural killer cells
CN112040979A (zh) 2018-03-21 2020-12-04 Alx肿瘤生物技术公司 针对信号调控蛋白α的抗体和使用方法
EP3790561A4 (en) * 2018-05-07 2022-06-08 President and Fellows of Harvard College CELL POPULATIONS AND GENE EXPRESSION RELATED TO IN VITRO BETA CELL DIFFERENTIATION
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
US20210340498A1 (en) * 2018-09-07 2021-11-04 Cha Biotech Co., Ltd. Medium for direct differentiation of pluripotent stem cell-derived mesenchymal stem cell, method for preparing mesenchymal stem cell by using same, and mesenchymal stem cell prepared thereby
JP7445657B2 (ja) 2018-12-06 2024-03-07 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
AU2020224103A1 (en) 2019-02-20 2021-09-16 Modernatx, Inc. Rna polymerase variants for co-transcriptional capping
CA3129782A1 (en) * 2019-03-15 2020-09-24 Charles E. Murry Improved survival of human cells differentiated <i>in vitro</i> by prpf31 gene expression knockdown
WO2020223477A1 (en) * 2019-04-30 2020-11-05 Memorial Sloan-Kettering Cancer Center Non-invasive methods for selectively enriching pluripotent cells
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
EP4010481A4 (en) 2019-08-09 2023-09-20 Nutcracker Therapeutics, Inc. MICROFLUIDIC DEVICES AND METHODS OF USE THEREOF
KR20220104682A (ko) 2019-10-18 2022-07-26 암니오틱스 아베 양수로부터 양수 간엽 줄기 세포 및 이로부터 유도된 세포를 얻기 위한 프로세스 및 장치
WO2021097155A1 (en) * 2019-11-14 2021-05-20 Cornell University modRNAS THAT EXPRESS ANTI MULLERIAN HORMONE (AMH) AND USES THEREOF
EP4061400A4 (en) * 2019-11-20 2024-01-10 Univ Cornell GENERATION OF RENAL GLOMERULUS-SPECIFIC ENDOTHELIAL CELLS AND METHODS OF USE
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
EP4168544A1 (en) * 2020-06-19 2023-04-26 eTheRNA Immunotherapies NV Rna purification method
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
US20240102065A1 (en) 2021-01-27 2024-03-28 CureVac SE Method of reducing the immunostimulatory properties of in vitro transcribed rna
CN113444726B (zh) * 2021-06-28 2022-07-29 甘肃农业大学 一种与仔猪细菌性腹泻相关的lncRNA ALDB-898及其应用
CN115558664A (zh) * 2021-07-27 2023-01-03 上海兆维科技发展有限公司 一种rna的制备方法、合成蛋白质的方法以及转录反应液

Family Cites Families (30)

* 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
US5849546A (en) 1996-09-13 1998-12-15 Epicentre Technologies Corporation Methods for using mutant RNA polymerases with reduced discrimination between non-canonical and canonical nucleoside triphosphates
US20030186237A1 (en) 2001-02-14 2003-10-02 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
AU2003297557B2 (en) 2002-11-21 2009-02-26 Cellscript, Inc. Methods for using primers that encode one strand of a double-stranded promoter
EP2216415B2 (en) * 2003-08-01 2017-01-04 Life Technologies Corporation Methods for preparing short RNA molecules
US20050153333A1 (en) 2003-12-02 2005-07-14 Sooknanan Roy R. Selective terminal tagging of nucleic acids
LT2578685T (lt) 2005-08-23 2019-06-10 The Trustees Of The University Of Pennsylvania Rnr, apimančios modifikuotus nukleozidus ir jų panaudojimo būdai
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
WO2007120863A2 (en) * 2006-04-14 2007-10-25 Epicentre Technologies Kits and methods for generating 5' capped rna
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
JP2010528622A (ja) 2007-05-30 2010-08-26 ザ ジェネラル ホスピタル コーポレイション 体細胞から多能性細胞を生成する方法
CA2707436C (en) 2007-06-29 2014-01-28 Epicentre Technologies Corporation Copy dna and sense rna
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
EP2250252A2 (en) 2008-02-11 2010-11-17 Cambridge Enterprise Limited Improved reprogramming of mammalian cells, and the 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
US20100273220A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
EP3112467B1 (en) 2009-12-07 2018-02-14 The Trustees of the University of Pennsylvania Rna preparations comprising purified modified rna for reprogramming 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
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
WO2012148586A1 (en) 2011-03-15 2012-11-01 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
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
US9862926B2 (en) * 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
DK2798064T3 (en) 2011-12-30 2016-12-19 Cellscript Llc PRODUCTION AND USE OF IN VITRO synthesized ssRNA TO FEED IN MAMMALIAN CELLS FOR INDUCTION OF A BIOLOGICAL OR BIOCHEMICAL EFFECT

Also Published As

Publication number Publication date
EP3677678C0 (en) 2024-01-31
AU2021240304A1 (en) 2021-10-28
EP3421601A1 (en) 2019-01-02
EP3421601B1 (en) 2019-12-04
HK1203559A1 (en) 2015-10-30
ES2676600T3 (es) 2018-07-23
SI3421601T1 (sl) 2020-03-31
US10201620B2 (en) 2019-02-12
AU2012362113A1 (en) 2014-08-14
AU2018264152A1 (en) 2018-12-06
LT3421601T (lt) 2020-01-10
PL3421601T3 (pl) 2020-06-01
CA2862377A1 (en) 2013-07-04
US20220257795A1 (en) 2022-08-18
EP3144389A1 (en) 2017-03-22
HRP20200251T1 (hr) 2020-05-29
EP3677678A1 (en) 2020-07-08
US20140328825A1 (en) 2014-11-06
AU2018264152B2 (en) 2021-07-01
ES2770314T3 (es) 2020-07-01
EP3144389B1 (en) 2018-05-09
LT3144389T (lt) 2018-08-10
DK2798064T3 (en) 2016-12-19
EP2798064A1 (en) 2014-11-05
HUE047947T2 (hu) 2020-05-28
US20190321490A1 (en) 2019-10-24
WO2013102203A1 (en) 2013-07-04
DK3421601T3 (da) 2020-02-24
EP3677678B1 (en) 2024-01-31
DK3144389T3 (en) 2018-07-16
US11135314B2 (en) 2021-10-05
AU2017251767B2 (en) 2018-08-16
AU2017251767A1 (en) 2017-11-16
EP2798064B1 (en) 2016-08-31
AU2012362113B2 (en) 2017-08-03

Similar Documents

Publication Publication Date Title
EP3677678C0 (en) PREPARATION AND USE OF IN VITRO SYNTHESIZED SSRNA FOR INTRODUCTION INTO MAMMAL CELLS TO INDUCE A BIOLOGICAL OR BIOCHEMICAL ACTION
IL281686A (en) Expansion of stem cells in hollow fiber bioreactors
IL288509A (en) Mammalian cell culture
HK1251617A1 (zh) 表達cd40l的哺乳動物細胞及其用途
EP2891712A4 (en) METHOD OF INDUCING THE DIFFERENTIATION OF PLURIPOTENT STEM CELLS IN VITRICULAR MYOCYTES IN VITRO
EP2811011A4 (en) CELL CULTURE KIT AND METHOD FOR USING THE CELL CULTURE KIT
SG2014013676A (en) Therapeutics using adipose cells and cell secretions
IL214529A0 (en) Bioreactor for the cultivation of mammalian cells and methods of use thereof
EP2671945A4 (en) METHOD OF BREEDING HUMAN PLURIPOTENTER STEM CELLS
EP2700707A4 (en) CELL CULTURE METHOD AND CELL CULTURE KIT
IL227516A0 (en) A method for preserving cells and cell cultures
GB201201645D0 (en) Improvements in and relating to biological sample collection
EP2699672A4 (en) METHOD FOR PRODUCING VIRUSES IN CULTURED CELLS
GB201200327D0 (en) Improvements in and relating to living walls
IL225348A0 (en) A method for obtaining a cell population rich in desired cells