PL2578685T3 - Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania - Google Patents

Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania

Info

Publication number
PL2578685T3
PL2578685T3 PL12008048T PL12008048T PL2578685T3 PL 2578685 T3 PL2578685 T3 PL 2578685T3 PL 12008048 T PL12008048 T PL 12008048T PL 12008048 T PL12008048 T PL 12008048T PL 2578685 T3 PL2578685 T3 PL 2578685T3
Authority
PL
Poland
Prior art keywords
methods
modified nucleosides
containing modified
rna containing
rna
Prior art date
Application number
PL12008048T
Other languages
English (en)
Inventor
Katalin Kariko
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 PL2578685T3 publication Critical patent/PL2578685T3/pl

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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
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K38/46—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
    • A61K48/0008—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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C12N15/67—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
    • 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
    • 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
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/40—Regulators of development
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/95—Protection of vectors from inactivation by agents such as antibodies or enzymes, e.g. using polymers

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Pulmonology (AREA)
  • Transplantation (AREA)
  • Developmental Biology & Embryology (AREA)
  • Analytical Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
PL12008048T 2005-08-23 2006-08-21 Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania PL2578685T3 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US71016405P 2005-08-23 2005-08-23
EP12008048.6A EP2578685B1 (en) 2005-08-23 2006-08-21 Rna containing modified nucleosides and methods of use thereof
PCT/US2006/032372 WO2007024708A2 (en) 2005-08-23 2006-08-21 Rna containing modified nucleosides and methods of use thereof
EP06813536A EP1979364A4 (en) 2005-08-23 2006-08-21 RNA CONTAINING MODIFIED NUCLEOSIDES AND METHODS OF USE THEREOF

Publications (1)

Publication Number Publication Date
PL2578685T3 true PL2578685T3 (pl) 2020-01-31

Family

ID=37772217

Family Applications (1)

Application Number Title Priority Date Filing Date
PL12008048T PL2578685T3 (pl) 2005-08-23 2006-08-21 Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania

Country Status (13)

Country Link
US (10) US8278036B2 (pl)
EP (5) EP4332227A1 (pl)
DK (1) DK2578685T3 (pl)
ES (2) ES2735531T3 (pl)
HR (1) HRP20230113T3 (pl)
HU (1) HUE043492T2 (pl)
LT (1) LT2578685T (pl)
PL (1) PL2578685T3 (pl)
PT (1) PT2578685T (pl)
RS (1) RS63964B1 (pl)
SI (1) SI3611266T1 (pl)
TR (1) TR201909609T4 (pl)
WO (1) WO2007024708A2 (pl)

Families Citing this family (443)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2340532T3 (es) 2001-06-05 2010-06-04 Curevac Gmbh Arnm con un contenido g/c aumentado que codifica para un antigeno bacteriano y utilizacion del mismo.
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
DE10347710B4 (de) 2003-10-14 2006-03-30 Johannes-Gutenberg-Universität Mainz Rekombinante Impfstoffe und deren Verwendung
EP4332227A1 (en) 2005-08-23 2024-03-06 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
US20070054873A1 (en) * 2005-08-26 2007-03-08 Protiva Biotherapeutics, Inc. Glucocorticoid modulation of nucleic acid-mediated immune stimulation
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
WO2007051303A1 (en) * 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
US7915399B2 (en) * 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
JP5876637B2 (ja) * 2006-10-18 2016-03-02 マリーナ バイオテック，インコーポレイテッド ニックまたはギャップの入った核酸分子およびそれらの使用
DE102006051516A1 (de) * 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
DE102007001370A1 (de) * 2007-01-09 2008-07-10 Curevac Gmbh RNA-kodierte Antikörper
WO2008109377A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting vegf family gene expression and uses thereof
JP2010519912A (ja) * 2007-03-02 2010-06-10 エムディーアールエヌエー，インコーポレイテッド Ｒａｓ遺伝子の発現を抑制するための核酸化合物およびその使用
JP2010519906A (ja) * 2007-03-02 2010-06-10 エムディーアールエヌエー，インコーポレイテッド Ｅｒｂｂファミリー遺伝子の発現を抑制するための核酸化合物およびその使用
WO2009046739A1 (en) 2007-10-09 2009-04-16 Curevac Gmbh Composition for treating prostate cancer (pca)
WO2009127230A1 (en) * 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
WO2010088927A1 (en) * 2009-02-09 2010-08-12 Curevac Gmbh Use of pei for the improvement of endosomal release and expression of transfected nucleic acids, complexed with cationic or polycationic compounds
US10837020B2 (en) 2009-04-22 2020-11-17 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long RNA molecules
EP2421563B1 (en) * 2009-04-22 2017-04-12 Massachusetts Institute of Technology Innate immune suppression enables repeated delivery of long rna molecules
CA2766907A1 (en) * 2009-07-06 2011-01-13 Novartis Ag Self replicating rna molecules and uses thereof
EP3581197A1 (de) 2009-07-31 2019-12-18 ethris GmbH Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
DE102009050308A1 (de) 2009-10-22 2011-05-05 Ludwig-Maximilians-Universität München RNA mit einer Kombination aus unmodifizierten und modifizierten Nucleotiden zur Proteinexpression
WO2011026185A1 (en) * 2009-09-04 2011-03-10 Murdoch Childrens Research Institute An assay for monitoring a neurological condition
NZ716192A (en) 2009-12-01 2017-07-28 Shire Human Genetic Therapies Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
AU2015215938B2 (en) * 2009-12-07 2018-01-18 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
US8808982B2 (en) * 2009-12-07 2014-08-19 Cellscript, Llc Compositions and methods for reprogramming eukaryotic cells
EP2338520A1 (de) 2009-12-21 2011-06-29 Ludwig Maximilians Universität Konjugat mit Zielfindungsligand und dessen Verwendung
EP3072961A1 (en) * 2010-04-16 2016-09-28 Children's Medical Center Corporation Sustained polypeptide expression from synthetic, modified rnas and uses thereof
EP2590670B1 (en) 2010-07-06 2017-08-23 GlaxoSmithKline Biologicals SA Methods of raising an immune response by delivery of rna
PT2590676T (pt) 2010-07-06 2016-11-04 Glaxosmithkline Biologicals Sa Partículas de transferência de tipo virião para moléculas de arn auto-replicante
ES2586580T3 (es) 2010-07-06 2016-10-17 Glaxosmithkline Biologicals Sa Inmunización de mamíferos grandes con dosis bajas de ARN
PL2590626T3 (pl) 2010-07-06 2016-04-29 Glaxosmithkline Biologicals Sa Liposomy z lipidami o korzystnej wartości pka do dostarczania rna
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
WO2012019630A1 (en) 2010-08-13 2012-02-16 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded protein
ES2938866T3 (es) 2010-08-31 2023-04-17 Glaxosmithkline Biologicals Sa Liposomas pegilados para la administración de ARN que codifica para inmunógeno
WO2012045082A2 (en) * 2010-10-01 2012-04-05 Jason Schrum Engineered nucleic acids and methods of use thereof
JP2013544504A (ja) 2010-10-11 2013-12-19 ノバルティス アーゲー 抗原送達プラットフォーム
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
JP2014511694A (ja) 2011-04-03 2014-05-19 ザ ジェネラル ホスピタル コーポレーション ドゥーイング ビジネス アズ マサチューセッツ ジェネラル ホスピタル 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現
BR112013029490A2 (pt) * 2011-05-17 2019-09-24 Moderna Therapeutics Inc ácidos nucleicos projetados e métodos de uso dos mesmos para vertebrados não humanos
HUE062102T2 (hu) 2011-05-24 2023-09-28 BioNTech SE Individualizált vakcinák a rák ellen
DK2717893T3 (da) * 2011-06-08 2019-07-22 Translate Bio Inc Lipid nanopartikelsammensætninger og fremgangsmåder til mrna-levering
WO2013006838A1 (en) 2011-07-06 2013-01-10 Novartis Ag 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
EP3682905B1 (en) 2011-10-03 2021-12-01 ModernaTX, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
WO2013078199A2 (en) * 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
WO2013086008A1 (en) 2011-12-05 2013-06-13 Factor Bioscience Inc. Methods and products for transfecting cells
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
WO2013086354A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
DK2791160T3 (da) 2011-12-16 2022-05-30 Modernatx Inc Modificerede mrna-sammensætninger
WO2013096709A2 (en) * 2011-12-21 2013-06-27 modeRNA Therapeutics Methods of increasing the viability or longevity of an organ or organ explant
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
WO2013120499A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly (a) sequence or a polyadenylation signal for increasing the expression of an encoded pathogenic antigen
WO2013120500A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded tumour antigen
WO2013120497A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
WO2013120498A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded allergenic antigen or an autoimmune self-antigen
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
US9683233B2 (en) 2012-03-27 2017-06-20 Curevac Ag Artificial nucleic acid molecules for improved protein or peptide expression
US10080809B2 (en) * 2012-03-27 2018-09-25 Curevac Ag Artificial nucleic acid molecules comprising a 5′TOP UTR
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
DE18200782T1 (de) 2012-04-02 2021-10-21 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
CA2868391A1 (en) * 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
CA2872030A1 (en) * 2012-05-31 2013-12-05 Sorrento Therapeutics, Inc. Antigen binding proteins that bind pd-l1
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
CA2876155C (en) * 2012-06-08 2022-12-13 Ethris Gmbh Pulmonary delivery of mrna to non-lung target cells
EA037448B1 (ru) 2012-06-08 2021-03-30 Этрис Гмбх Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
JOP20200308A1 (ar) 2012-09-07 2017-06-16 Novartis Ag جزيئات إرتباط il-18
US20140087416A1 (en) 2012-09-07 2014-03-27 Genentech, Inc. Methods and compositions for producing induced hepatocytes
EP3786298A1 (en) 2012-11-01 2021-03-03 Factor Bioscience Inc. Methods and products for expressing proteins in cells
HRP20220607T1 (hr) * 2012-11-26 2022-06-24 Modernatx, Inc. Terminalno modificirana rna
WO2014082729A1 (en) 2012-11-28 2014-06-05 Biontech Ag Individualized vaccines for cancer
EP3628335B1 (en) 2012-12-07 2023-11-08 Translate Bio, Inc. Lipidic nanoparticles for mrna delivery in the lungs
US20150315541A1 (en) * 2012-12-13 2015-11-05 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
WO2014113089A2 (en) * 2013-01-17 2014-07-24 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
MA38322B1 (fr) 2013-02-08 2018-09-28 Novartis Ag Anticorps anti-il-17a et leur utilisation dans le traitement de troubles auto-immuns et inflammatoires
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
US20160024181A1 (en) * 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
UA117008C2 (uk) * 2013-03-14 2018-06-11 Шир Хьюман Дженетік Терапіс, Інк. IN VITRO ТРАНСКРИБОВАНА мРНК ТА КОМПОЗИЦІЯ, ЩО ЇЇ МІСТИТЬ, ДЛЯ ЗАСТОСУВАННЯ В ЛІКУВАННІ МУКОВІСЦИДОЗУ В ССАВЦЯ
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
DK3467108T3 (da) 2013-03-14 2024-06-10 Translate Bio Inc Fremgangsmåder til oprensning af messenger-RNA
WO2014160243A1 (en) * 2013-03-14 2014-10-02 The Trustees Of The University Of Pennsylvania Purification and purity assessment of rna molecules synthesized with modified nucleosides
WO2014160284A1 (en) * 2013-03-14 2014-10-02 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of stroke
US9957515B2 (en) 2013-03-15 2018-05-01 Cibus Us Llc Methods and compositions for targeted gene modification
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP2983804A4 (en) 2013-03-15 2017-03-01 Moderna Therapeutics, Inc. Ion exchange purification of mrna
WO2014152027A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Manufacturing methods for production of rna transcripts
EP2971165A4 (en) 2013-03-15 2016-11-23 Moderna Therapeutics Inc DISSOLUTION OF DNA FRAGMENTS IN MRNA MANUFACTURING METHODS
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
DE102013005361A1 (de) * 2013-03-28 2014-10-02 Eberhard Karls Universität Tübingen Medizinische Fakultät Polyribonucleotid
CN115261411A (zh) 2013-04-04 2022-11-01 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
WO2014180490A1 (en) 2013-05-10 2014-11-13 Biontech Ag Predicting immunogenicity of t cell epitopes
WO2014186334A1 (en) * 2013-05-15 2014-11-20 Robert Kruse Intracellular translation of circular rna
ES2810298T3 (es) 2013-06-28 2021-03-08 Ethris Gmbh Composiciones para introducir ARN en células
PL3019619T3 (pl) 2013-07-11 2022-01-10 Modernatx, Inc. Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania
CA2919324A1 (en) 2013-07-26 2015-01-29 Kenta Yamamoto Osteoblast and method for preparing same
JP2016530294A (ja) 2013-09-03 2016-09-29 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． キメラポリヌクレオチド
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
WO2015051169A2 (en) 2013-10-02 2015-04-09 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
DE102013111099B4 (de) * 2013-10-08 2023-11-30 Eberhard Karls Universität Tübingen Medizinische Fakultät Permanente Genkorrektur mittels nukleotidmodifizierter messenger RNA
EA201992208A1 (ru) 2013-10-22 2020-07-31 Транслейт Био, Инк. ЛЕЧЕНИЕ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
EP3060303B1 (en) 2013-10-22 2018-11-14 Translate Bio, Inc. Mrna therapy for argininosuccinate synthetase deficiency
EP3060257B1 (en) 2013-10-22 2021-02-24 Translate Bio, Inc. Lipid formulations for delivery of messenger rna
WO2015065505A1 (en) * 2013-10-29 2015-05-07 Duke University Use of cyp27a1 inhibitors, statin, or lxr antagonists alone or in combination with conventional therapy for the treatment of breast cancer
WO2015062738A1 (en) 2013-11-01 2015-05-07 Curevac Gmbh Modified rna with decreased immunostimulatory properties
EP3542802A1 (en) 2013-11-01 2019-09-25 CureVac AG Modified rna with decreased immunostimulatory properties
WO2015077717A1 (en) 2013-11-25 2015-05-28 The Broad Institute Inc. Compositions and methods for diagnosing, evaluating and treating cancer by means of the dna methylation status
WO2015085147A1 (en) 2013-12-05 2015-06-11 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
US20160367638A1 (en) 2013-12-19 2016-12-22 Crystal BYERS LEPTIN mRNA COMPOSITIONS AND FORMULATIONS
AU2014368898B2 (en) 2013-12-20 2020-06-11 Dana-Farber Cancer Institute, Inc. Combination therapy with neoantigen vaccine
US11254951B2 (en) 2014-12-30 2022-02-22 Curevac Ag Artificial nucleic acid molecules
CN111304231A (zh) 2013-12-30 2020-06-19 库瑞瓦格股份公司 人工核酸分子
WO2015101415A1 (en) 2013-12-30 2015-07-09 Curevac Gmbh Artificial nucleic acid molecules
DK3690056T5 (da) 2014-01-31 2024-08-26 Factor Bioscience Inc Fremgangsmåder og produkter til fremstilling og indgivelse af nukleinsyre
AU2015218576B2 (en) 2014-02-24 2020-02-27 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration
AU2014384269A1 (en) 2014-02-26 2016-09-08 Ethris Gmbh Compositions for gastrointestinal administration of RNA
CN106455514A (zh) 2014-03-14 2017-02-22 希博斯美国有限公司 采用寡核苷酸介导的基因修复提高靶向基因修饰的效率的方法和组合物
CA2944128C (en) * 2014-04-01 2022-08-30 Institut National De La Sante Et De La Recherche Medicale (Inserm) Capped and uncapped rna molecules and block copolymers for intracellular delivery of rna
RU2746406C2 (ru) 2014-04-23 2021-04-13 МОДЕРНАТиЭкс, ИНК. Вакцины на основе нуклеиновых кислот
JP6571679B2 (ja) 2014-04-25 2019-09-04 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
JP6557722B2 (ja) 2014-05-30 2019-08-07 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 核酸の送達のための生分解性脂質
WO2015188933A1 (en) 2014-06-10 2015-12-17 Curevac Ag Methods and means for enhancing rna production
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP3157573A4 (en) 2014-06-19 2018-02-21 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
ES2964588T3 (es) 2014-06-24 2024-04-08 Translate Bio Inc Composiciones enriquecidas estereoquímicamente para la administración de ácidos nucleicos
PL3766916T3 (pl) 2014-06-25 2023-02-27 Acuitas Therapeutics Inc. Nowe lipidy i formulacje nanocząstek lipidowych do dostarczania kwasów nukleinowych
EP3161001A2 (en) 2014-06-25 2017-05-03 Novartis AG Antibodies specific for il-17a fused to hyaluronan binding peptide tags
JP2017522028A (ja) 2014-07-16 2017-08-10 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 環状ポリヌクレオチド
CA2955250A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20170210788A1 (en) * 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
JP6841753B2 (ja) 2014-09-15 2021-03-10 ザ チルドレンズ メディカル センター コーポレーション ヒストンｈ３−リジントリメチル化を除去することによって体細胞核移入（ｓｃｎｔ）効率を増加させるための方法および組成物
WO2016044745A1 (en) 2014-09-19 2016-03-24 Regeneron Pharmaceuticals, Inc. Chimeric antigen receptors
WO2016045732A1 (en) 2014-09-25 2016-03-31 Biontech Rna Pharmaceuticals Gmbh Stable formulations of lipids and liposomes
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
WO2016070166A2 (en) 2014-11-02 2016-05-06 Arcturus Therapeutics, Inc. Messenger una molecules and uses thereof
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
EP3461904A1 (en) 2014-11-10 2019-04-03 ModernaTX, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
WO2016077123A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Multiparametric nucleic acid optimization
ES2946969T3 (es) 2014-12-12 2023-07-28 CureVac SE Moléculas de ácido nucleico artificiales para una expresión proteica mejorada
EP3034539A1 (en) 2014-12-19 2016-06-22 Ethris GmbH Compositions for introducing nucleic acid into cells
US10975442B2 (en) 2014-12-19 2021-04-13 Massachusetts Institute Of Technology Molecular biomarkers for cancer immunotherapy
WO2016100977A1 (en) 2014-12-19 2016-06-23 The Broad Institute Inc. Methods for profiling the t-cel- receptor repertoire
WO2016128060A1 (en) 2015-02-12 2016-08-18 Biontech Ag Predicting t cell epitopes useful for vaccination
AU2016218977C1 (en) 2015-02-13 2023-03-23 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
JP2018526321A (ja) * 2015-04-27 2018-09-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 適応免疫応答を誘導するためのヌクレオシド修飾ｒｎａ
RU2733754C2 (ru) 2015-05-20 2020-10-06 Те Брод Инститьют Инк. Общие неоантигены
TWI750122B (zh) 2015-06-09 2021-12-21 美商博德研究所有限公司 用於贅瘤疫苗之調配物及其製備方法
PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
DK3317415T3 (da) 2015-06-30 2019-06-24 Ethris Gmbh UTR'er, som forøger translationseffektiviteten for RNA-molekyler
WO2017011766A1 (en) 2015-07-16 2017-01-19 Cornell University Methods of enhancing translation ability of rna molecules treatments, and kits
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
US11434486B2 (en) 2015-09-17 2022-09-06 Modernatx, Inc. Polynucleotides containing a morpholino linker
ES2810701T5 (es) 2015-10-05 2024-07-11 Modernatx Inc Procedimientos para la administración terapéutica de medicamentos de ácido ribonucleico mensajero
WO2017059902A1 (en) 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
IL308174A (en) 2015-10-09 2024-01-01 Univ Southampton Gene expression modulation and dysregulated protein expression scanning
US11970710B2 (en) 2015-10-13 2024-04-30 Duke University Genome engineering with Type I CRISPR systems in eukaryotic cells
DK3362461T3 (da) 2015-10-16 2022-05-09 Modernatx Inc Mrna-cap-analoger med modificeret phosphatbinding
WO2017066797A1 (en) 2015-10-16 2017-04-20 Modernatx, Inc. Trinucleotide mrna cap analogs
US20190225644A1 (en) * 2015-10-16 2019-07-25 Modernatx, Inc. Mrna cap analogs and methods of mrna capping
PT3718565T (pt) 2015-10-22 2022-07-20 Modernatx Inc Vacinas contra vírus respiratórios
PE20181529A1 (es) 2015-10-22 2018-09-26 Modernatx Inc Vacunas de acido nucleico para el virus varicela-zoster (vzv)
EP3364981A4 (en) 2015-10-22 2019-08-07 ModernaTX, Inc. VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
WO2017075531A1 (en) 2015-10-28 2017-05-04 Acuitas Therapeutics, Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
US11389546B2 (en) 2015-12-09 2022-07-19 Modernatx, Inc. Heterologous UTR sequences for enhanced mRNA expression
MA43415A (fr) * 2015-12-09 2018-10-17 Modernatx Inc Arnm modifié codant pour une uridine diphosphate glucuronosyltransférase et utilisations associées
WO2017098468A1 (en) 2015-12-09 2017-06-15 Novartis Ag Label-free analysis of rna capping efficiency using rnase h, probes and liquid chromatography/mass spectrometry
DK3386484T3 (da) 2015-12-10 2022-07-04 Modernatx Inc Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
AU2016369612B2 (en) 2015-12-17 2023-06-01 Modernatx, Inc. Polynucleotides encoding methylmalonyl-CoA mutase
WO2017127750A1 (en) * 2016-01-22 2017-07-27 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
JP2019506170A (ja) 2016-02-09 2019-03-07 キブス ユーエス エルエルシー オリゴヌクレオチド介在性遺伝子修復を利用した標的遺伝子修飾の効率を上昇させるための方法および組成物
WO2017151623A1 (en) 2016-03-01 2017-09-08 Alexion Pharmaceuticals, Inc. Biodegradable activated polymers for therapeutic delivery
US11318197B2 (en) 2016-03-03 2022-05-03 Duke University Compositions and methods for inducing HIV-1 antibodies
WO2017152146A2 (en) 2016-03-03 2017-09-08 Duke University Compositions and methods for inducing hiv-1 antibodies
EP3426775A1 (en) * 2016-03-10 2019-01-16 Novartis AG Chemically modified messenger rna's
KR102312903B1 (ko) 2016-03-31 2021-10-15 에트리스 게엠베하 신규의 최소 utr 서열
WO2017173354A2 (en) * 2016-04-01 2017-10-05 University Of Iowa Research Foundation METABOLICALLY STABILIZED DOUBLE STRANDED mRNA
EP3443001A4 (en) 2016-04-11 2020-04-29 Obsidian Therapeutics, Inc. REGULATED BIOCIRCUIT SYSTEMS
WO2017180917A2 (en) 2016-04-13 2017-10-19 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
US20190346442A1 (en) 2016-04-18 2019-11-14 The Broad Institute, Inc. Improved hla epitope prediction
LT3458083T (lt) 2016-05-18 2023-02-10 Modernatx, Inc. Polinukleotidai, koduojantys interleukiną-12 (il12), ir jų naudojimas
MA45051A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant la relaxine
EP3458108A4 (en) * 2016-05-18 2020-04-22 ModernaTX, Inc. POLYNUCLEOTIDES FOR CODING THE TRANSMEMBRANE CONDUCTIVE REGULATOR OF CYSTIC FIBROSE FOR TREATING CYSTIC FIBROSE
EP3458107B1 (en) 2016-05-18 2024-03-13 ModernaTX, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
CN116458475A (zh) 2016-06-03 2023-07-21 瑞泽恩制药公司 表达外源末端脱氧核苷酸转移酶的非人动物
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
US10927383B2 (en) 2016-06-30 2021-02-23 Ethris Gmbh Cas9 mRNAs
JP2019520829A (ja) 2016-07-07 2019-07-25 ルビウス セラピューティクス， インコーポレイテッド 外来性ｒｎａを発現する治療的細胞系に関連する組成物及び方法
WO2018035377A1 (en) 2016-08-17 2018-02-22 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
SG11201901941YA (en) 2016-09-14 2019-04-29 Modernatx Inc High purity rna compositions and methods for preparation thereof
CA3039089A1 (en) 2016-10-03 2018-04-12 Duke University Methods to identify immunogens by targeting improbable mutations
US10662416B2 (en) 2016-10-14 2020-05-26 Precision Biosciences, Inc. Engineered meganucleases specific for recognition sequences in the hepatitis B virus genome
WO2018075980A1 (en) 2016-10-21 2018-04-26 Modernatx, Inc. Human cytomegalovirus vaccine
JP7204643B2 (ja) 2016-10-24 2023-01-16 オリオニス バイオサイエンシズ ビーブイ 標的変異インターフェロン－ガンマおよびその使用
EP3532097A1 (en) * 2016-10-27 2019-09-04 The Trustees Of The University Of Pennsylvania Nucleoside-modified rna for inducing an adaptive immune response
US10907165B2 (en) 2016-10-31 2021-02-02 Cornell University Methods of enhancing translation ability and stability of RNA molecules, treatments, and kits
EP3538068A1 (en) 2016-11-10 2019-09-18 Translate Bio, Inc. Improved ice-based lipid nanoparticle formulation for delivery of mrna
EP3538146A4 (en) 2016-11-11 2020-07-15 ModernaTX, Inc. INFLUENZA VACCINE
EP3555289A1 (en) 2016-12-13 2019-10-23 ModernaTX, Inc. Rna affinity purification
BR112019014314A2 (pt) 2017-01-11 2020-02-18 The Trustees Of The University Of Pennsylvania Composição, e, método de indução de uma resposta imune adaptativa contra vírus zika.
CN110325210A (zh) 2017-01-11 2019-10-11 小利兰·斯坦福大学托管委员会 Rspo替代物分子
US11549149B2 (en) 2017-01-24 2023-01-10 The Broad Institute, Inc. Compositions and methods for detecting a mutant variant of a polynucleotide
CA3050133A1 (en) 2017-01-26 2018-08-02 Surrozen, Inc. Tissue-specific wnt signal enhancing molecules and uses thereof
AU2018214556A1 (en) 2017-02-01 2019-08-15 Modernatx, Inc. Immunomodulatory therapeutic mRNA compositions encoding activating oncogene mutation peptides
WO2018141964A1 (en) 2017-02-06 2018-08-09 Orionis Biosciences Nv Targeted chimeric proteins and uses thereof
US10906985B2 (en) 2017-02-06 2021-02-02 Orionis Biosciences, Inc. Targeted engineered interferon and uses thereof
KR20190108167A (ko) * 2017-02-10 2019-09-23 성균관대학교산학협력단 Rna 간섭을 위한 긴 이중가닥 rna
CA3054062A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Novel codon-optimized cftr mrna
WO2018157153A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Large scale synthesis of messenger rna
US11407800B2 (en) 2017-02-28 2022-08-09 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof
AU2018229278A1 (en) 2017-02-28 2019-10-17 Sanofi Therapeutic RNA
US11576961B2 (en) 2017-03-15 2023-02-14 Modernatx, Inc. Broad spectrum influenza virus vaccine
EP3595713A4 (en) 2017-03-15 2021-01-13 ModernaTX, Inc. RESPIRATORY SYNCYTIAL VIRUS VACCINE
US11752206B2 (en) 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
US11045540B2 (en) 2017-03-15 2021-06-29 Modernatx, Inc. Varicella zoster virus (VZV) vaccine
MA47790A (fr) 2017-03-17 2021-05-05 Modernatx Inc Vaccins à base d'arn contre des maladies zoonotiques
MA48047A (fr) 2017-04-05 2020-02-12 Modernatx Inc Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée
WO2018188730A1 (en) 2017-04-11 2018-10-18 Biontech Rna Pharmaceuticals Gmbh Rna for treatment of autoimmune diseases
WO2018191657A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
CA3061326A1 (en) 2017-04-27 2018-11-01 The Trustees Of The University Of Pennsylvania Nucleoside-modified mrna-lipid nanoparticle lineage vaccine for hepatitis c virus
CA3061612A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
CA3062591A1 (en) 2017-05-08 2018-11-15 Gritstone Oncology, Inc. Alphavirus neoantigen vectors
EP3624824B1 (en) 2017-05-16 2024-07-10 Translate Bio, Inc. Codon-optimized mrna encoding cftr for use in treating cystic fibrosis
EP3630964A4 (en) 2017-05-31 2021-03-03 Ultragenyx Pharmaceutical Inc. THERAPEUTIC AGENTS FOR GLYCOGEN STORAGE DISEASE TYPE III
AU2018281280A1 (en) 2017-06-07 2020-01-16 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
US20200131498A1 (en) 2017-06-14 2020-04-30 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase
MA49395A (fr) * 2017-06-14 2020-04-22 Modernatx Inc Polynucléotides codant pour le facteur viii de coagulation
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
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
EP3424524A3 (en) * 2017-07-04 2019-02-27 CureVac AG Cancer rna-vaccine
WO2019010304A1 (en) * 2017-07-05 2019-01-10 Wisconsin Alumni Research Foundation MICROPARTICLES COATED WITH MINERALS FOR GENES ADMINISTRATION IN CHRONIC WOUND THERAPY
EP3655040A1 (en) 2017-07-21 2020-05-27 Modernatx, Inc. Modified mrna encoding a propionyl-coa carboxylase and uses thereof
MA49684A (fr) 2017-07-24 2020-06-03 Modernatx Inc Arnm modifié codant une glucose-6-phosphatase et utilisations associées
US11639329B2 (en) 2017-08-16 2023-05-02 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036000A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
CN111246854A (zh) 2017-08-17 2020-06-05 宾夕法尼亚大学理事会 编码单纯疱疹病毒糖蛋白的修饰mrna疫苗及其用途
US11912982B2 (en) 2017-08-18 2024-02-27 Modernatx, Inc. Methods for HPLC analysis
EP3668971B8 (en) 2017-08-18 2024-05-29 ModernaTX, Inc. Rna polymerase variants
EP3668977A4 (en) 2017-08-18 2021-04-21 Modernatx, Inc. HPLC ANALYTICAL PROCESSES
GB2599884B (en) 2017-08-25 2022-08-31 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
US10653767B2 (en) 2017-09-14 2020-05-19 Modernatx, Inc. Zika virus MRNA vaccines
WO2019070730A1 (en) 2017-10-02 2019-04-11 Duke University MOSAIC ENVELOPES OF HIV-1 TO INDUCE ADCC RESPONSES
WO2019087113A1 (en) 2017-11-01 2019-05-09 Novartis Ag Synthetic rnas and methods of use
EP3714047A2 (en) 2017-11-22 2020-09-30 ModernaTX, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
WO2019104152A1 (en) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US20200325532A1 (en) 2017-12-15 2020-10-15 Novartis Ag Polya tail length analysis of rna by mass spectrometry
EP3735271A4 (en) 2018-01-04 2022-06-15 Iconic Therapeutics, Inc. ANTI-TISSUE FACTOR ANTIBODIES, ANTIBODY DRUG CONJUGATES AND RELATED METHODS
CN111819285B (zh) 2018-01-09 2024-08-09 希博斯美国有限公司 防碎基因和突变
MA54676A (fr) 2018-01-29 2021-11-17 Modernatx Inc Vaccins à base d'arn contre le vrs
CA3090406A1 (en) 2018-02-05 2019-08-08 Orionis Biosciences, Inc. Fibroblast binding agents and use thereof
EP3749373A1 (en) 2018-02-07 2020-12-16 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
WO2019158583A1 (en) 2018-02-13 2019-08-22 Ethris Gmbh Polyribonucleotide containing deuterated nucleotides
CA3093823A1 (en) * 2018-03-13 2019-09-19 The Board Of Trustees Of The Leland Stanford Junior University Transient cellular reprogramming for reversal of cell aging
MX2020010795A (es) 2018-04-12 2021-01-08 Prec Biosciences Inc Nucleasas modificadas genéticamente optimizadas que tienen especificidad para el gen de la región constante alfa del receptor de linfocitos t humanos.
US11142750B2 (en) 2018-04-12 2021-10-12 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
ES2960939T3 (es) 2018-04-25 2024-03-07 Ethris Gmbh Agentes crioprotectores para formulaciones particuladas
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
US20210363547A1 (en) 2018-05-06 2021-11-25 Emendobio Inc. Differential knockout of an allele of a heterozygous elane gene
EP3806888B1 (en) 2018-06-12 2024-01-31 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
CN108956566B (zh) * 2018-07-06 2020-10-02 军事科学院军事医学研究院环境医学与作业医学研究所 基于切刻内切酶和杂交链式反应双重放大的上转换荧光检测多氯联苯试剂盒及检测方法
CN118421617A (zh) 2018-08-24 2024-08-02 川斯勒佰尔公司 用于纯化信使rna的方法
JP2022500436A (ja) * 2018-09-13 2022-01-04 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 糖原病を処置するためのグルコース−６−ホスファターゼをコードするポリヌクレオチド
EP3863645A4 (en) * 2018-09-13 2022-11-16 ModernaTX, Inc. MODIFIED mRNA FOR THE TREATMENT OF FAMILY PROGRESSIVE INTRAHEPATIC CHOLESTASIS DISORDERS
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
US20220152225A1 (en) * 2018-09-27 2022-05-19 Modernatx, Inc. Polynucleotides encoding arginase 1 for the treatment of arginase deficiency
US20210382068A1 (en) 2018-10-02 2021-12-09 Dana-Farber Cancer Institute, Inc. Hla single allele lines
EP3870600A1 (en) 2018-10-24 2021-09-01 Obsidian Therapeutics, Inc. Er tunable protein regulation
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
CN111303283A (zh) 2018-12-12 2020-06-19 上海君实生物医药科技股份有限公司 抗il-17a抗体及其应用
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
EP3898661A1 (en) 2018-12-21 2021-10-27 Precision BioSciences, Inc. Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria
KR20210138569A (ko) 2019-01-11 2021-11-19 아퀴타스 테라퓨틱스 인크. 활성제의 지질 나노입자 전달을 위한 지질
WO2020163379A1 (en) 2019-02-05 2020-08-13 Emendobio Inc. Crispr compositions and methods for promoting gene editing of ribosomal protein s19 (rps19) gene
WO2020168466A1 (en) 2019-02-19 2020-08-27 Stemirna Therapeutics Co., Ltd. Modified nucleoside and synthetic methods thereof
EP3927821A4 (en) 2019-02-20 2023-01-25 ModernaTX, Inc. RNA POLYMERASE VARIANTS FOR CO-STRANSCRIPTION CAPTING
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
WO2020190750A1 (en) 2019-03-15 2020-09-24 Modernatx, Inc. Hiv rna vaccines
US20230072532A1 (en) * 2019-03-25 2023-03-09 Flagship Pioneering Innovations Vi, Llc Compositions comprising modified circular polyribonucleotides and uses thereof
CA3135799A1 (en) 2019-04-03 2020-10-08 Precision Biosciences, Inc. Genetically-modified immune cells comprising a microrna-adapted shrna (shrnamir)
US20220204994A1 (en) 2019-04-05 2022-06-30 Precision Biosciences, Inc. Methods of preparing populations of genetically-modified immune cells
US20230190819A1 (en) * 2019-05-11 2023-06-22 Youngsuk Yi Compositions and methods containing exosomes
JP7457733B2 (ja) 2019-05-30 2024-03-28 グリットストーン バイオ インコーポレイテッド 改変アデノウイルス
EP4001308A4 (en) 2019-07-15 2023-08-23 Shanghai Junshi Biosciences Co., Ltd. ANTI-TIGIT ANTIBODIES AND THEIR APPLICATION
US20220273715A1 (en) 2019-07-25 2022-09-01 Precision Biosciences, Inc. Compositions and methods for sequential stacking of nucleic acid sequences into a genomic locus
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
WO2021030268A2 (en) 2019-08-09 2021-02-18 Nutcracker Therapeutics, Inc. Microfluidic apparatus and methods of use thereof
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
DE102019122014A1 (de) 2019-08-15 2021-02-18 Technische Universität Darmstadt Reduktion der Knochenresorption, insbesondere bei chronischen Gelenkerkrankungen
WO2021035054A1 (en) 2019-08-20 2021-02-25 Precision Biosciences, Inc. Lymphodepletion dosing regimens for cellular immunotherapies
WO2021055609A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
CN114728176A (zh) 2019-10-09 2022-07-08 川斯勒佰尔公司 信使rna的组合物、方法和使用
JP2022553042A (ja) 2019-10-21 2022-12-21 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の組成物、方法および使用
AU2020370751A1 (en) 2019-10-24 2022-06-09 Novago Therapeutics Ag Novel anti-Nogo-A antibodies
US20220411479A1 (en) 2019-10-30 2022-12-29 Precision Biosciences, Inc. Cd20 chimeric antigen receptors and methods of use for immunotherapy
WO2021113765A1 (en) 2019-12-06 2021-06-10 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the hepatitis b virus genome
CA3160096A1 (en) 2019-12-06 2021-06-10 Bruce J. Mccreedy Jr. Methods for cancer immunotherapy
CA3165388A1 (en) 2019-12-20 2021-06-24 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
EP4076393A2 (en) 2019-12-20 2022-10-26 Translate Bio, Inc. Rectal delivery of messenger rna
WO2021138447A1 (en) 2019-12-31 2021-07-08 Elixirgen Therapeutics, Inc. Temperature-based transient delivery of nucleic acids and proteins to cells and tissues
WO2021142245A1 (en) 2020-01-10 2021-07-15 Translate Bio, Inc. Compounds, pharmaceutical compositions and methods for modulating expression of muc5b in lung cells and tissues
US20240277830A1 (en) 2020-02-04 2024-08-22 CureVac SE Coronavirus vaccine
AU2021218671A1 (en) 2020-02-10 2022-10-06 Translate Bio, Inc. Methods and compositions for messenger RNA purification
IL295669A (en) 2020-02-18 2022-10-01 Translate Bio Inc Improved processes for in vitro messenger rna transcription
KR20230035217A (ko) 2020-02-24 2023-03-13 인스티튜트 오브 마이크로바이올로지, 차이니즈 아카데미 오브 사이언스즈 2019 신규 코로나바이러스의 인간화 모노클로날 항체 및 그의 용도
AU2021226578A1 (en) 2020-02-25 2022-10-20 Translate Bio, Inc. Improved processes of preparing mRNA-loaded lipid nanoparticles
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
MX2022013254A (es) 2020-04-22 2023-01-24 BioNTech SE Vacuna contra el coronavirus.
IL297962A (en) 2020-05-07 2023-01-01 Translate Bio Inc Optimized nucleotide sequences encoding sars-cov-2 antigens
AU2021268028A1 (en) 2020-05-07 2023-01-19 Translate Bio, Inc. Generation of optimized nucleotide sequences
WO2021226463A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy
KR20230022409A (ko) 2020-05-11 2023-02-15 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머
WO2021231259A1 (en) 2020-05-11 2021-11-18 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
CA3172171A1 (en) 2020-05-12 2021-11-18 Victor Bartsevich Treatment of retinitis pigmentosa using improved engineered meganucleases
WO2021231901A1 (en) 2020-05-15 2021-11-18 Translate Bio, Inc. Lipid nanoparticle formulations for mrna delivery
US20230242624A1 (en) 2020-06-02 2023-08-03 Neurimmune Ag HUMAN ANTIBODIES AGAINST SEVERE ACUTE RESPIRATORY SYNDROME CORONAVIRUS-2 (SARS-CoV-2)
US20240059771A1 (en) 2020-07-13 2024-02-22 Shanghai Junshi Biosciences Co., Ltd. Anti-cldn-18.2 antibody and use thereof
EP4179112A2 (en) 2020-07-13 2023-05-17 The Board of Trustees of the Leland Stanford Junior University Systems and methods to assess rna stability
AU2021308681A1 (en) 2020-07-16 2023-03-09 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
EP4201958A4 (en) 2020-07-21 2024-09-04 Shanghai Junshi Biosciences Co Ltd ANTI-CTLA-4 ANTIBODY AND ITS USE
MX2023001556A (es) 2020-08-05 2023-03-08 Dragonfly Therapeutics Inc Anticuerpos que se dirigen a egfr y uso de los mismos.
WO2022032196A2 (en) 2020-08-06 2022-02-10 Gritstone Bio, Inc. Multiepitope vaccine cassettes
EP4192958A4 (en) * 2020-08-07 2024-08-28 Univ Hong Kong Science & Tech COMPOSITIONS AND METHODS FOR INCREASING PROTEIN EXPRESSION
WO2022035793A1 (en) 2020-08-10 2022-02-17 Precision Biosciences, Inc. Antibodies and fragments specific for b-cell maturation antigen and uses thereof
EP4200412A1 (en) 2020-08-21 2023-06-28 Precision BioSciences, Inc. Engineered meganucleases having specificity for a recognition sequence in the transthyretin gene
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
US11492611B2 (en) 2020-08-31 2022-11-08 The Board Of Trustees Of The Leland Stanford Junior University Systems and methods for producing RNA constructs with increased translation and stability
WO2022076562A1 (en) 2020-10-06 2022-04-14 Translate Bio, Inc. Improved process and formulation of lipid nanoparticles
WO2022076547A1 (en) 2020-10-07 2022-04-14 Precision Biosciences, Inc. Lipid nanoparticle compositions
US20220110884A1 (en) 2020-10-12 2022-04-14 Translate Bio, Inc. Process of preparing mrna-loaded lipid nanoparticles
US20220133631A1 (en) 2020-10-12 2022-05-05 Translate Bio, Inc. Process of preparing ice-based lipid nanoparticles
US20230233706A1 (en) * 2020-10-13 2023-07-27 The Trustees Of The University Of Pennsylvania In vivo targeting of CD4+-T cells for mRNA therapeutics
CA3198726A1 (en) 2020-10-15 2022-04-21 Translate Bio, Inc. Large scale synthesis of messenger rna
WO2022090752A1 (en) 2020-10-26 2022-05-05 Pécsi Tudományegyetem Vaccine platform
GB202017649D0 (en) 2020-11-09 2020-12-23 Autolus Ltd Polypeptide
WO2022099194A1 (en) 2020-11-09 2022-05-12 Translate Bio, Inc. Improved compositions for delivery of codon-optimized mrna
CA3172481A1 (en) 2020-11-12 2022-05-19 Precision Biosciences, Inc. Engineered meganucleases having specificity for recognition sequences in the dystrophin gene
WO2022106860A1 (en) 2020-11-20 2022-05-27 Pécsi Tudományegyetem Recombinant peptides for use in therapy
US20220287966A1 (en) 2020-11-25 2022-09-15 Translate Bio, Inc. Stable Liquid Lipid Nanoparticle Formulations
EP4251170A1 (en) 2020-11-25 2023-10-04 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
JP2024502210A (ja) 2020-12-22 2024-01-17 キュアバック エスイー ＳＡＲＳ－ＣｏＶ－２バリアントに対するＲＮＡワクチン
WO2022150616A1 (en) 2021-01-08 2022-07-14 Precision Biosciences, Inc. Engineered meganucleases having specificity for a recognition sequence in the hydroxyacid oxidase 1 gene
CN116981692A (zh) 2021-01-14 2023-10-31 翻译生物公司 递送mRNA编码的抗体的方法和组合物
WO2022165111A1 (en) 2021-01-28 2022-08-04 Precision Biosciences, Inc. Modulation of tgf beta signaling in genetically-modified eukaryotic cells
AU2022214429A1 (en) 2021-02-01 2023-09-14 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
AU2022219332A1 (en) 2021-02-09 2023-09-21 Shanghai Junshi Biosciences Co., Ltd. Anti-cd112r antibody and use thereof
US20240226337A9 (en) * 2021-02-16 2024-07-11 The Board Of Trustees Of The Leland Stanford Junior University Systems and Methods to Enhance RNA Stability and Translation and Uses Thereof
JP2024510415A (ja) 2021-03-01 2024-03-07 スキロム ゲゼルシャフト ミット ベシュレンクテル ハフツング ｉＲｈｏｍ２に対するヒト化抗体
US20220323553A1 (en) * 2021-03-08 2022-10-13 The Trustees Of The University Of Pennsylvania Epigenomic editing and reactivation of targets for the treatment of Fragile X syndrome
WO2022204549A1 (en) 2021-03-25 2022-09-29 Translate Bio, Inc. Optimized nucleotide sequences encoding the extracellular domain of human ace2 protein or a portion thereof
WO2022207652A1 (en) 2021-03-29 2022-10-06 Scirhom Gmbh Methods of treatment using protein binders to irhom2 epitopes
WO2022214664A1 (en) 2021-04-09 2022-10-13 Philogen S.P.A. Improved interferon-gamma mutant
WO2022225918A1 (en) 2021-04-19 2022-10-27 Translate Bio, Inc. Improved compositions for delivery of mrna
WO2022226303A1 (en) 2021-04-22 2022-10-27 Precision Biosciences, Inc. Engineered meganucleases that target human mitochondrial genomes
EP4326861A1 (en) 2021-04-22 2024-02-28 Precision Biosciences, Inc. Engineered meganucleases that target human mitochondrial genomes
KR20240004662A (ko) 2021-04-29 2024-01-11 트랜슬레이트 바이오 인코포레이티드 폴리 a 테일 길이를 측정하는 방법
AU2022267356A1 (en) * 2021-04-30 2023-11-23 The Trustees Of The University Of Pennsylvania Compositions and methods for targeting lipid nanoparticle therapeutics to stem cells
WO2022234003A1 (en) 2021-05-07 2022-11-10 Avacta Life Sciences Limited Cd33 binding polypeptides with stefin a protein
WO2022235838A1 (en) 2021-05-07 2022-11-10 Helix Nanotechnologies, Inc. Modified ribonucleic acids and uses thereof
EP4351623A1 (en) 2021-06-09 2024-04-17 Icahn School of Medicine at Mount Sinai Gene combination as a broad spectrum antiviral
WO2023278754A1 (en) 2021-07-01 2023-01-05 Translate Bio, Inc. Compositions for delivery of mrna
KR20240051112A (ko) 2021-07-01 2024-04-19 인답타 세라뷰틱스 인코포레이티드 조작된 자연 살해(nk) 세포 및 관련 방법
CA3227103A1 (en) 2021-07-30 2023-02-02 Matthew P. GEMBERLING Compositions and methods for modulating expression of frataxin (fxn)
CA3227105A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4388090A1 (en) 2021-08-16 2024-06-26 Translate Bio, Inc. Screening codon-optimized nucleotide sequences
CN116059391A (zh) 2021-08-26 2023-05-05 上海君实生物医药科技股份有限公司 抗cldn-18.2抗体药物偶联物及其用途
TW202332694A (zh) 2021-10-07 2023-08-16 英商阿凡克塔生命科學公司 血清半衰期延長之ｐｄ－ｌ１結合多肽
EP4413038A1 (en) 2021-10-07 2024-08-14 Avacta Life Sciences Limited Pd-l1 binding affimers
EP4419660A2 (en) 2021-10-19 2024-08-28 Precision BioSciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
AU2022371430A1 (en) 2021-10-19 2024-05-30 Precision Biosciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
WO2023068931A1 (en) 2021-10-21 2023-04-27 Curevac Netherlands B.V. Cancer neoantigens
EP4401789A1 (en) 2021-10-22 2024-07-24 BioNTech SE Oligosaccharide complexes and uses
EP4402149A1 (en) 2021-10-22 2024-07-24 BioNTech SE Disulfide oligosaccharide compounds and complexes
EP4285932A1 (en) 2022-05-30 2023-12-06 BioNTech SE Oligosaccharide complexes and uses
EP4285933A1 (en) 2022-05-30 2023-12-06 BioNTech SE Oligosaccharide complexes and uses
WO2023067126A1 (en) 2021-10-22 2023-04-27 BioNTech SE Oligosaccharide compounds and complexes
WO2023067125A1 (en) 2021-10-22 2023-04-27 BioNTech SE Oligosaccharide complexes and uses
EP4286003A1 (en) 2022-05-30 2023-12-06 BioNTech SE Oligosaccharide compounds and complexes
EP4169534A1 (en) 2021-10-22 2023-04-26 BioNTech SE Oligosaccharide complexes and uses
EP4169579A1 (en) 2021-10-22 2023-04-26 BioNTech SE Disulfide oligosaccharide compounds and complexes
EP4169580A1 (en) 2021-10-22 2023-04-26 BioNTech SE Oligosaccharide compounds and complexes
EP4401838A1 (en) 2021-10-22 2024-07-24 BioNTech SE Oligosaccharide compounds and complexes
EP4286394A1 (en) 2022-05-30 2023-12-06 BioNTech SE Oligosaccharide compounds and complexes
EP4186528A1 (en) 2021-11-30 2023-05-31 BioNTech SE Oligosaccharide complexes and uses
EP4286004A1 (en) 2022-05-30 2023-12-06 BioNTech SE Disulfide oligosaccharide compounds and complexes
EP4169578A1 (en) 2021-10-22 2023-04-26 BioNTech SE Oligosaccharide compounds and complexes
WO2023081767A1 (en) 2021-11-05 2023-05-11 Precision Biosciences, Inc. Methods for immunotherapy
WO2023083434A1 (en) * 2021-11-09 2023-05-19 BioNTech SE Rna encoding peptidoglycan hydrolase and use thereof for treating bacterial infection
CN118591398A (zh) 2021-11-10 2024-09-03 翻译生物公司 用于治疗原发性纤毛运动不良症的组合物和方法
US20230220106A1 (en) 2021-12-08 2023-07-13 Dragonfly Therapeutics, Inc. Antibodies targeting 5t4 and uses thereof
EP4202045A1 (en) 2021-12-22 2023-06-28 Universität Hamburg Synthetic transfer rna with modified nucleotides
TW202346348A (zh) 2022-01-10 2023-12-01 美商再生元醫藥公司 作為基因治療所遞送之bbb靶向的gaa在龐貝氏症小鼠模型中治療cns及肌肉
AU2023209012A1 (en) 2022-01-20 2024-09-05 Shanghai Junshi Biosciences Co., Ltd. Anti-cd3 and anti-cd20 bispecific antibody and use thereof
WO2023145755A1 (ja) 2022-01-25 2023-08-03 公益財団法人川崎市産業振興財団 経皮投与用のｒｎａを含む組成物および当該組成物の投与方法
WO2023143444A1 (zh) 2022-01-28 2023-08-03 上海君实生物医药科技股份有限公司 抗dkk1抗体、其药物组合物及用途
WO2023183921A2 (en) 2022-03-24 2023-09-28 Nature's Toolbox, Inc. Methods and compositions for the enzymatic production of pseudouridine triphosphate
WO2023218243A1 (en) 2022-05-12 2023-11-16 Avacta Life Sciences Limited Lag-3/pd-l1 binding fusion proteins
US12064479B2 (en) 2022-05-25 2024-08-20 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2023232747A1 (en) 2022-05-30 2023-12-07 BioNTech SE Complexes for delivery of nucleic acids
TW202405175A (zh) 2022-06-07 2024-02-01 美商斯奎柏治療公司 用於靶向pcsk9的組合物及方法
TW202413643A (zh) 2022-06-07 2024-04-01 美商斯奎柏治療公司 用於靶向pcsk9的組合物及方法
WO2023250511A2 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
US11878055B1 (en) 2022-06-26 2024-01-23 BioNTech SE Coronavirus vaccine
WO2024007020A1 (en) 2022-06-30 2024-01-04 Indapta Therapeutics, Inc. Combination of engineered natural killer (nk) cells and antibody therapy and related methods
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
WO2024015881A2 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
WO2024023246A1 (en) 2022-07-28 2024-02-01 Philogen S.P.A. Antibody binding to pd1
WO2024033362A1 (en) 2022-08-08 2024-02-15 Atb Therapeutics Humanized antibodies against cd79b
US20240067969A1 (en) 2022-08-19 2024-02-29 Tune Therapeutics, Inc. Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
AU2023274159A1 (en) 2022-09-07 2024-03-21 Eyegene Inc. COMPOSITION FOR IN-VIVO DELIVERING mRNA CONTAINING MODIFIED NUCLEOTIDE
WO2024064642A2 (en) 2022-09-19 2024-03-28 Tune Therapeutics, Inc. Compositions, systems, and methods for modulating t cell function
TW202417510A (zh) 2022-09-20 2024-05-01 美商威特拉公司 用ｃ３ｂ抗體治療補體介導之疾病及病症
WO2024068674A1 (en) 2022-09-26 2024-04-04 BioNTech SE Nucleic acid complexes and uses thereof
WO2024086827A2 (en) 2022-10-20 2024-04-25 Repertoire Immune Medicines, Inc. Cd8 t cell targeted il2
WO2024084462A1 (en) 2022-10-21 2024-04-25 BioNTech SE Nucleic acid complexes and uses thereof
WO2024112652A1 (en) 2022-11-21 2024-05-30 Translate Bio, Inc. Compositions of dry powder formulations of messenger rna and methods of use thereof
WO2024126809A1 (en) 2022-12-15 2024-06-20 Sanofi Mrna encoding influenza virus-like particle
WO2024133515A1 (en) 2022-12-20 2024-06-27 Sanofi Rhinovirus mrna vaccine
WO2024133884A2 (en) 2022-12-23 2024-06-27 Sanofi Optimized tailing of messenger rna
WO2024141786A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Multitarget vaccines and therapeutics
WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
WO2024148167A1 (en) 2023-01-05 2024-07-11 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for the human t cell receptor alpha constant region gene
WO2024151975A1 (en) 2023-01-13 2024-07-18 Igor Lednev Detecting degradation of a polynucleotide by raman spectroscopy
WO2024153793A1 (en) 2023-01-20 2024-07-25 Astrazeneca Ab Nucleic acid molecules
WO2024153794A1 (en) 2023-01-20 2024-07-25 Astrazeneca Ab Vaccine
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024170684A1 (en) 2023-02-15 2024-08-22 Sanofi Screening codon-optimized nucleotide sequences
WO2024184489A1 (en) 2023-03-07 2024-09-12 Sanofi Manufacture of messenger rna with kp34 polymerase

Family Cites Families (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5807718A (en) 1994-12-02 1998-09-15 The Scripps Research Institute Enzymatic DNA molecules
EP0795029A1 (en) * 1994-12-08 1997-09-17 Pabio Chemical labelling of objects
CA2207593A1 (en) * 1994-12-13 1996-06-20 John Gustofson Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
AU9319398A (en) * 1997-09-19 1999-04-05 Sequitur, Inc. Sense mrna therapy
US6127535A (en) * 1997-11-05 2000-10-03 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into oligonucleotides
JP2002500010A (ja) * 1997-12-23 2002-01-08 カイロン コーポレイション ヒト遺伝子および遺伝子発現産物ｉ
US6066625A (en) 1998-02-03 2000-05-23 Methylgene, Inc. Optimized antisense oligonucleotides complementary to DNA methyltransferase sequences
WO2000027340A2 (en) * 1998-11-12 2000-05-18 The Children's Medical Center Corporation USE OF t-RNA AND FRAGMENTS FOR INHIBITING ANGIOGENESIS AND COMPOSITIONS THEREOF
WO2001002608A1 (en) * 1999-07-06 2001-01-11 Isis Pharmaceuticals, Inc. Oligonucleotides incorporating both 2-aminoadenine and 5-substituted pyrimidines
US20040086880A1 (en) * 1999-07-20 2004-05-06 Sampson Jeffrey R Method of producing nucleic acid molecules with reduced secondary structure
US20040229271A1 (en) * 2000-05-19 2004-11-18 Williams Richard B. Compositions and methods for the identification and selection of nucleic acids and polypeptides
WO2005072087A2 (en) * 2003-12-12 2005-08-11 Proteonova, Inc. System and methods for nucleic acid and polypeptide selection
WO2003044194A1 (en) * 2001-11-16 2003-05-30 Proteonova, Inc. Selection and evolution of nucleic acids and polypeptides
SE0003002D0 (sv) * 2000-08-22 2000-08-22 Primert Ab New sequences
FR2822164B1 (fr) 2001-03-19 2004-06-18 Centre Nat Rech Scient Polypeptides derives des arn polymerases, et leurs utilisations
US20030171253A1 (en) * 2001-04-19 2003-09-11 Averil Ma Methods and compositions relating to modulation of A20
US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
ES2340532T3 (es) * 2001-06-05 2010-06-04 Curevac Gmbh Arnm con un contenido g/c aumentado que codifica para un antigeno bacteriano y utilizacion del mismo.
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
DE10229872A1 (de) 2002-07-03 2004-01-29 Curevac Gmbh Immunstimulation durch chemisch modifizierte RNA
US9567591B2 (en) 2003-05-15 2017-02-14 Mello Biotechnology, Inc. Generation of human embryonic stem-like cells using intronic RNA
GB0316089D0 (en) 2003-07-09 2003-08-13 Xo Bioscience Ltd Differentiation method
DE10335833A1 (de) 2003-08-05 2005-03-03 Curevac Gmbh Transfektion von Blutzellen mit mRNA zur Immunstimulation und Gentherapie
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
CA2572439A1 (en) 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory sirna molecules and uses therefor
DE102005023170A1 (de) 2005-05-19 2006-11-23 Curevac Gmbh Optimierte Formulierung für mRNA
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
EP4332227A1 (en) 2005-08-23 2024-03-06 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
US20070087437A1 (en) 2005-10-14 2007-04-19 Jifan Hu Methods for rejuvenating cells in vitro and in vivo
DE102006007433A1 (de) 2006-02-17 2007-08-23 Curevac Gmbh Adjuvanz in Form einer Lipid-modifizierten Nukleinsäure
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
WO2008151058A2 (en) 2007-05-30 2008-12-11 The General Hospital Corporation Methods of generating pluripotent cells from somatic cells
WO2009006438A2 (en) 2007-06-29 2009-01-08 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
US20100330677A1 (en) 2008-02-11 2010-12-30 Cambridge Enterprise Limited Improved Reprogramming of Mammalian Cells, and Cells Obtained
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
WO2010008486A2 (en) 2008-06-24 2010-01-21 Parkinsons 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
EP2421563B1 (en) 2009-04-22 2017-04-12 Massachusetts Institute of Technology Innate immune suppression enables repeated delivery of long rna molecules
EP3581197A1 (de) 2009-07-31 2019-12-18 ethris GmbH Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
US8808982B2 (en) 2009-12-07 2014-08-19 Cellscript, Llc Compositions and methods for reprogramming eukaryotic cells
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
EP3072961A1 (en) 2010-04-16 2016-09-28 Children's Medical Center Corporation Sustained polypeptide expression from synthetic, modified rnas and uses thereof
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical 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
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

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