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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JP2022513718A (ja
JPWO2020118115A5 (https=
JP2022513718A5 (https=
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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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  • 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)
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
JPWO2020118115A5 JPWO2020118115A5 (https=) 2022-12-12
JP2022513718A5 JP2022513718A5 (https=) 2022-12-12
JP7445657B2 true JP7445657B2 (ja) 2024-03-07

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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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
KR20240006603A (ko) 2021-05-07 2024-01-15 헬릭스 나노테크놀로지스, 인코포레이티드 변형된 리보핵산 및 이의 용도
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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Publication number Publication date
ES2960692T3 (es) 2024-03-06
JP2022513718A (ja) 2022-02-09
US20260078356A1 (en) 2026-03-19
CA3122080A1 (en) 2020-06-11
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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