AU2019394996B2 - Compositions and methods for treating ornithine transcarbamylase deficiency - Google Patents

Compositions and methods for treating ornithine transcarbamylase deficiency

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Publication number
AU2019394996B2
AU2019394996B2 AU2019394996A AU2019394996A AU2019394996B2 AU 2019394996 B2 AU2019394996 B2 AU 2019394996B2 AU 2019394996 A AU2019394996 A AU 2019394996A AU 2019394996 A AU2019394996 A AU 2019394996A AU 2019394996 B2 AU2019394996 B2 AU 2019394996B2
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Australia
Prior art keywords
seq
otc
mrna
sequence
protein
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AU2019394996A
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English (en)
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AU2019394996A1 (en
Inventor
Padmanabh Chivukula
Daiki MATSUDA
Carlos G. PEREZ-GARCIA
Kiyoshi Tachikawa
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Arcturus Therapeutics Inc
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Arcturus Therapeutics Inc
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Publication of AU2019394996A1 publication Critical patent/AU2019394996A1/en
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Publication of AU2019394996B2 publication Critical patent/AU2019394996B2/en
Priority to AU2026200747A priority Critical patent/AU2026200747A1/en
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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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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Cardiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • 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)
AU2019394996A 2018-12-06 2019-12-05 Compositions and methods for treating ornithine transcarbamylase deficiency Active AU2019394996B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2026200747A AU2026200747A1 (en) 2018-12-06 2026-02-02 Compositions and methods for treating ornithine transcarbamylase deficiency

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2026200747A Division AU2026200747A1 (en) 2018-12-06 2026-02-02 Compositions and methods for treating ornithine transcarbamylase deficiency

Publications (2)

Publication Number Publication Date
AU2019394996A1 AU2019394996A1 (en) 2021-07-29
AU2019394996B2 true AU2019394996B2 (en) 2025-11-06

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Family Applications (2)

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AU2019394996A Active AU2019394996B2 (en) 2018-12-06 2019-12-05 Compositions and methods for treating ornithine transcarbamylase deficiency
AU2026200747A Pending AU2026200747A1 (en) 2018-12-06 2026-02-02 Compositions and methods for treating ornithine transcarbamylase deficiency

Family Applications After (1)

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AU2026200747A Pending AU2026200747A1 (en) 2018-12-06 2026-02-02 Compositions and methods for treating ornithine transcarbamylase deficiency

Country Status (9)

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US (3) US11685906B2 (enExample)
EP (2) EP4299750A3 (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)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019394996B2 (en) 2018-12-06 2025-11-06 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
EP3891175A4 (en) * 2018-12-06 2022-11-09 Arcturus Therapeutics, Inc. MODIFIED PROTEINS AND RELATED TREATMENT METHODS
US12351834B2 (en) 2020-03-03 2025-07-08 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
WO2022204370A1 (en) * 2021-03-24 2022-09-29 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
US20250127927A1 (en) * 2021-08-23 2025-04-24 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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WO2017218524A1 (en) * 2016-06-13 2017-12-21 Rana Therapeutics, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency

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Also Published As

Publication number Publication date
EP4299750A2 (en) 2024-01-03
US11685906B2 (en) 2023-06-27
WO2020118115A1 (en) 2020-06-11
CA3122080A1 (en) 2020-06-11
JP2022513718A (ja) 2022-02-09
ES2960692T3 (es) 2024-03-06
EP3891274B1 (en) 2023-10-18
PL3891274T3 (pl) 2024-03-25
US12398379B2 (en) 2025-08-26
EP3891274A4 (en) 2022-08-31
US20200181584A1 (en) 2020-06-11
HUE064076T2 (hu) 2024-02-28
EP3891274A1 (en) 2021-10-13
US20260078356A1 (en) 2026-03-19
AU2026200747A1 (en) 2026-02-26
EP4299750A3 (en) 2024-07-10
JP7445657B2 (ja) 2024-03-07
AU2019394996A1 (en) 2021-07-29
US20240002815A1 (en) 2024-01-04
EP3891274C0 (en) 2023-10-18

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