ES2960692T3 - Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa - Google Patents

Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa Download PDF

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Publication number
ES2960692T3
ES2960692T3 ES19893199T ES19893199T ES2960692T3 ES 2960692 T3 ES2960692 T3 ES 2960692T3 ES 19893199 T ES19893199 T ES 19893199T ES 19893199 T ES19893199 T ES 19893199T ES 2960692 T3 ES2960692 T3 ES 2960692T3
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Spain
Prior art keywords
otc
mrna
seq
protein
polynucleotide
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Active
Application number
ES19893199T
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English (en)
Spanish (es)
Inventor
Carlos Perez-Garcia
Kiyoshi Tachikawa
Daiki Matsuda
Padmanabh Chivukula
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Arcturus Therapeutics Inc
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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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  • 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)
ES19893199T 2018-12-06 2019-12-05 Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa Active ES2960692T3 (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
ES2960692T3 true ES2960692T3 (es) 2024-03-06

Family

ID=70972469

Family Applications (1)

Application Number Title Priority Date Filing Date
ES19893199T Active ES2960692T3 (es) 2018-12-06 2019-12-05 Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa

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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)

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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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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
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
AU2019394996B2 (en) 2025-11-06
US20240002815A1 (en) 2024-01-04
EP3891274C0 (en) 2023-10-18

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