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HRP20220796T1 - Ribonukleinske kiseline koje sadrže n1-metil-pseudouracil i njihove uporabe - Google Patents

Ribonukleinske kiseline koje sadrže n1-metil-pseudouracil i njihove uporabe Download PDF

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Publication number
HRP20220796T1
HRP20220796T1 HRP20220796TT HRP20220796T HRP20220796T1 HR P20220796 T1 HRP20220796 T1 HR P20220796T1 HR P20220796T T HRP20220796T T HR P20220796TT HR P20220796 T HRP20220796 T HR P20220796T HR P20220796 T1 HRP20220796 T1 HR P20220796T1
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Croatia
Prior art keywords
mrna
binding partner
methyl
major groove
nucleotides
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HRP20220796TT
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English (en)
Inventor
Jason P. SCHRUM
Suhaib Siddiqi
Kenechi Ejebe
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ModernaTX, Inc.
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Application filed by ModernaTX, Inc. filed Critical ModernaTX, Inc.
Publication of HRP20220796T1 publication Critical patent/HRP20220796T1/hr

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    • 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
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    • 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
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
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    • C—CHEMISTRY; METALLURGY
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
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    • C07—ORGANIC CHEMISTRY
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2887—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD20
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • C12N15/09—Recombinant DNA-technology
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    • C12N5/0602—Vertebrate cells
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    • C12P21/00—Preparation of peptides or proteins
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/558—Immunoassay; Biospecific binding assay; Materials therefor using diffusion or migration of antigen or antibody
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    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
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    • 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
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    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U

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Claims (7)

1. Postupak sinteze mRNA, sadrži korake: a) osiguravanje komplementarne deoksiribonukleinske kiseline (cDNA) koja kodira protein od interesa; b) odabir nukleotida za koji je poznato da poremeti vezanje glavnog utora partnera koji se veže s nukleinskom kiselinom, pri čemu nukleotid ima smanjen afinitet vezanja prema glavnom utoru partnera koji se veže; c) dovođenje u kontakt priložene cDNA i odabranog nukleotida s RNA polimerazom pod uvjetima da se mRNA sintetizira, pri čemu nukleotid sadrži N1-metil-pseudouridin, i pri čemu je 100% nukleotida koji sadrže uracil u RNA zamijenjeno nukleotidima koji sadrže N1-metil-pseudouridin.
2. Postupak prema zahtjevu 1, naznačen time što je glavni utor partnera koji se veže odabran iz skupine koja se sastoji od receptora nalik na Toll (TLR) 3, TLR7, TLR8, gena I inducibilnog retinojskom kiselinom (RIG-I), gena povezanog s diferencijacijom melanoma 5 (MDA5) i laboratorij za genetiku i fiziologiju 2 (LGP2).
3. mRNA u kojoj je 100% nukleotida koji sadrže uracil u mRNA zamijenjeno nukleotidima koji sadrže N1-metil-pseudouridin.
4. mRNA prema zahtjevu 3, naznačena time što je mRNA duga najmanje 300 nukleotida.
5. mRNA prema bilo kojem od zahtjeva 3 ili 4 sadrži rep poliA.
6. mRNA prema bilo kojem od zahtjeva 3-5, naznačena time što glavni utor partnera koji se veže je RIG-I, MDA5 ili LGP2.
7. mRNA prema bilo kojem od zahtjeva 3-5, naznačena time što glavni utor partnera koji se veže je TLR3, TLR7 ili TLR8.
HRP20220796TT 2010-10-01 2011-10-03 Ribonukleinske kiseline koje sadrže n1-metil-pseudouracil i njihove uporabe HRP20220796T1 (hr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US40441310P 2010-10-01 2010-10-01
EP19177059.3A EP3590949B1 (en) 2010-10-01 2011-10-03 Ribonucleic acids containing n1-methyl-pseudouracils and uses thereof

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HRP20220796T1 true HRP20220796T1 (hr) 2022-10-14

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HRP20220796TT HRP20220796T1 (hr) 2010-10-01 2011-10-03 Ribonukleinske kiseline koje sadrže n1-metil-pseudouracil i njihove uporabe

Country Status (24)

Country Link
US (11) US20120237975A1 (hr)
EP (8) EP2857499A1 (hr)
JP (1) JP2013543381A (hr)
CN (3) CN104531812A (hr)
AU (2) AU2011308496A1 (hr)
BR (1) BR112013007862A2 (hr)
CA (3) CA2813466A1 (hr)
DE (1) DE19177059T1 (hr)
DK (1) DK3590949T3 (hr)
ES (3) ES2737960T3 (hr)
HR (1) HRP20220796T1 (hr)
HU (1) HUE058896T2 (hr)
IL (1) IL225493A0 (hr)
LT (1) LT3590949T (hr)
MX (1) MX2013003681A (hr)
NZ (1) NZ608972A (hr)
PL (1) PL3590949T3 (hr)
PT (1) PT3590949T (hr)
RS (1) RS63430B1 (hr)
RU (1) RU2013120302A (hr)
SG (2) SG10201508149TA (hr)
SI (1) SI3590949T1 (hr)
WO (2) WO2012045082A2 (hr)
ZA (2) ZA201303161B (hr)

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