AU2017248189B2 - Multimeric coding nucleic acid and uses thereof - Google Patents

Multimeric coding nucleic acid and uses thereof Download PDF

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AU2017248189B2
AU2017248189B2 AU2017248189A AU2017248189A AU2017248189B2 AU 2017248189 B2 AU2017248189 B2 AU 2017248189B2 AU 2017248189 A AU2017248189 A AU 2017248189A AU 2017248189 A AU2017248189 A AU 2017248189A AU 2017248189 B2 AU2017248189 B2 AU 2017248189B2
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protein
mcna
lna
ome
mrnas
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AU2017248189A1 (en
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Daniel Crawford
Frank Derosa
Michael Heartlein
Shrirang KARVE
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Translate Bio Inc
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Translate Bio Inc
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Priority to AU2024202844A priority patent/AU2024202844A1/en
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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
    • C12N15/63—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
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
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    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1816—Erythropoietin [EPO]
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
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    • 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
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    • 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
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C12N15/09—Recombinant DNA-technology
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    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
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    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
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    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Gastroenterology & Hepatology (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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AU2017248189A 2016-04-08 2017-04-07 Multimeric coding nucleic acid and uses thereof Active AU2017248189B2 (en)

Priority Applications (2)

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AU2021206908A AU2021206908B2 (en) 2016-04-08 2021-07-23 Multimeric coding nucleic acid and uses thereof
AU2024202844A AU2024202844A1 (en) 2016-04-08 2024-05-01 Multimeric coding nucleic acid and uses thereof

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US201662320073P 2016-04-08 2016-04-08
US62/320,073 2016-04-08
PCT/US2017/026660 WO2017177169A1 (en) 2016-04-08 2017-04-07 Multimeric coding nucleic acid and uses thereof

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US (4) US10266843B2 (enExample)
EP (2) EP3440206B1 (enExample)
JP (1) JP7150608B6 (enExample)
KR (2) KR102475301B1 (enExample)
CN (1) CN109072223B (enExample)
AU (3) AU2017248189B2 (enExample)
CA (1) CA3020343A1 (enExample)
DK (1) DK3440206T3 (enExample)
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