JP7445657B2 - 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 - Google Patents

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 Download PDF

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JP7445657B2
JP7445657B2 JP2021531945A JP2021531945A JP7445657B2 JP 7445657 B2 JP7445657 B2 JP 7445657B2 JP 2021531945 A JP2021531945 A JP 2021531945A JP 2021531945 A JP2021531945 A JP 2021531945A JP 7445657 B2 JP7445657 B2 JP 7445657B2
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seq
mrna
otc
sequence
protein
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JP2022513718A5 (enExample
JP2022513718A (ja
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ジー ペレス－ガルシア，カルロス
清 立川
大輝 松田
チブクラ，パドマナブ
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Arcturus Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
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  • Heart & Thoracic Surgery (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021531945A 2018-12-06 2019-12-05 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 Active JP7445657B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862776302P 2018-12-06 2018-12-06
US62/776,302 2018-12-06
PCT/US2019/064786 WO2020118115A1 (en) 2018-12-06 2019-12-05 Compositions and methods for treating ornithine transcarbamylase deficiency

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JP2022513718A JP2022513718A (ja) 2022-02-09
JP2022513718A5 JP2022513718A5 (enExample) 2022-12-12
JP7445657B2 true JP7445657B2 (ja) 2024-03-07

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US (3) US11685906B2 (enExample)
EP (2) EP3891274B1 (enExample)
JP (1) JP7445657B2 (enExample)
AU (2) AU2019394996B2 (enExample)
CA (1) CA3122080A1 (enExample)
ES (1) ES2960692T3 (enExample)
HU (1) HUE064076T2 (enExample)
PL (1) PL3891274T3 (enExample)
WO (1) WO2020118115A1 (enExample)

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EP4114470A4 (en) * 2020-03-03 2024-04-17 Arcturus Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
US20240189449A1 (en) * 2021-03-24 2024-06-13 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency
EP4334330A1 (en) 2021-05-07 2024-03-13 Helix Nanotechnologies, Inc. Modified ribonucleic acids and uses thereof
WO2023028469A2 (en) * 2021-08-23 2023-03-02 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells

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AU2026200747A1 (en) 2026-02-26
US20200181584A1 (en) 2020-06-11
US20240002815A1 (en) 2024-01-04
PL3891274T3 (pl) 2024-03-25
AU2019394996A1 (en) 2021-07-29
EP3891274A4 (en) 2022-08-31
EP3891274B1 (en) 2023-10-18
ES2960692T3 (es) 2024-03-06
CA3122080A1 (en) 2020-06-11
JP2022513718A (ja) 2022-02-09
EP4299750A3 (en) 2024-07-10
US11685906B2 (en) 2023-06-27
EP3891274C0 (en) 2023-10-18
EP3891274A1 (en) 2021-10-13
US12398379B2 (en) 2025-08-26
HUE064076T2 (hu) 2024-02-28
US20260078356A1 (en) 2026-03-19
AU2019394996B2 (en) 2025-11-06
WO2020118115A1 (en) 2020-06-11
EP4299750A2 (en) 2024-01-03

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