CA3122080A1 - Compositions and methods for treating ornithine transcarbamylase deficiency - Google Patents

Compositions and methods for treating ornithine transcarbamylase deficiency Download PDF

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Publication number
CA3122080A1
CA3122080A1 CA3122080A CA3122080A CA3122080A1 CA 3122080 A1 CA3122080 A1 CA 3122080A1 CA 3122080 A CA3122080 A CA 3122080A CA 3122080 A CA3122080 A CA 3122080A CA 3122080 A1 CA3122080 A1 CA 3122080A1
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CA
Canada
Prior art keywords
seq
mrna
otc
polynucleotide
protein
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3122080A
Other languages
English (en)
French (fr)
Inventor
Carlos G. PEREZ-GARCIA
Kiyoshi Tachikawa
Daiki MATSUDA
Padmanabh Chivukula
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
Original Assignee
Arcturus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Arcturus Therapeutics Inc filed Critical Arcturus Therapeutics Inc
Publication of CA3122080A1 publication Critical patent/CA3122080A1/en
Pending legal-status Critical Current

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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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
CA3122080A 2018-12-06 2019-12-05 Compositions and methods for treating ornithine transcarbamylase deficiency Pending CA3122080A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3122080A1 true CA3122080A1 (en) 2020-06-11

Family

ID=70972469

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3122080A Pending CA3122080A1 (en) 2018-12-06 2019-12-05 Compositions and methods for treating ornithine transcarbamylase deficiency

Country Status (9)

Country Link
US (3) US11685906B2 (https=)
EP (2) EP4299750A3 (https=)
JP (1) JP7445657B2 (https=)
AU (2) AU2019394996B2 (https=)
CA (1) CA3122080A1 (https=)
ES (1) ES2960692T3 (https=)
HU (1) HUE064076T2 (https=)
PL (1) PL3891274T3 (https=)
WO (1) WO2020118115A1 (https=)

Families Citing this family (6)

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JP7445657B2 (ja) 2018-12-06 2024-03-07 アークトゥラス・セラピューティクス・インコーポレイテッド 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
JP2022520148A (ja) * 2018-12-06 2022-03-29 アークトゥラス・セラピューティクス・インコーポレイテッド 修飾タンパク質および関連の処置方法
JP7832114B2 (ja) * 2020-03-03 2026-03-17 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法
WO2022204370A1 (en) * 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency
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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
ES2960692T3 (es) 2024-03-06
JP2022513718A (ja) 2022-02-09
JP7445657B2 (ja) 2024-03-07
US20260078356A1 (en) 2026-03-19
US20200181584A1 (en) 2020-06-11
US12398379B2 (en) 2025-08-26
EP3891274A4 (en) 2022-08-31
AU2019394996B2 (en) 2025-11-06
AU2026200747A1 (en) 2026-02-26
PL3891274T3 (pl) 2024-03-25
US20240002815A1 (en) 2024-01-04
AU2019394996A1 (en) 2021-07-29
EP3891274A1 (en) 2021-10-13
EP3891274B1 (en) 2023-10-18
HUE064076T2 (hu) 2024-02-28
EP3891274C0 (en) 2023-10-18
EP4299750A3 (en) 2024-07-10
EP4299750A2 (en) 2024-01-03
US11685906B2 (en) 2023-06-27
WO2020118115A1 (en) 2020-06-11

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