JP2021505620A - アルギナーゼ１欠損症を治療するための方法及び組成物 - Google Patents

アルギナーゼ１欠損症を治療するための方法及び組成物 Download PDF

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JP2021505620A
JP2021505620A JP2020531164A JP2020531164A JP2021505620A JP 2021505620 A JP2021505620 A JP 2021505620A JP 2020531164 A JP2020531164 A JP 2020531164A JP 2020531164 A JP2020531164 A JP 2020531164A JP 2021505620 A JP2021505620 A JP 2021505620A
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arginase
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arginine
plasma
levels
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JP2021505620A5 (enExample
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スコット ダブリュー． ローリンソン，
スコット ダブリュー． ローリンソン，
アンソニー ジー． クイン，
アンソニー ジー． クイン，
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イーレイズ・インコーポレーテッド
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51—Lyases (4)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/03—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amidines (3.5.3)
    • C12Y305/03001—Arginase (3.5.3.1)

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020531164A 2017-12-05 2018-12-05 アルギナーゼ１欠損症を治療するための方法及び組成物 Pending JP2021505620A (ja)

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Application Number Priority Date Filing Date Title
JP2023199616A JP2024028760A (ja) 2017-12-05 2023-11-27 アルギナーゼ１欠損症を治療するための方法及び組成物

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762594747P 2017-12-05 2017-12-05
US62/594,747 2017-12-05
US201862725612P 2018-08-31 2018-08-31
US62/725,612 2018-08-31
US201862745000P 2018-10-12 2018-10-12
US62/745,000 2018-10-12
PCT/US2018/063982 WO2019113157A1 (en) 2017-12-05 2018-12-05 Method and composition for treating arginase 1 deficiency

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JP2021505620A5 JP2021505620A5 (enExample) 2022-01-11

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US (2) US11717562B2 (enExample)
EP (1) EP3720476A1 (enExample)
JP (2) JP2021505620A (enExample)
KR (1) KR20200122298A (enExample)
CN (3) CN120643682A (enExample)
AU (1) AU2018378485B2 (enExample)
CA (1) CA3084801A1 (enExample)
MX (2) MX2020005933A (enExample)
TW (1) TWI878212B (enExample)
WO (1) WO2019113157A1 (enExample)

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EP3449935A1 (en) * 2017-08-31 2019-03-06 Erytech Pharma Arginine deiminase encapsulated inside erythrocytes and their use in treating cancer and arginase-1 deficiency
MX2021004463A (es) * 2018-10-19 2021-08-24 Aerase Inc Terapia de disminución de arginina para el tratamiento de la deficiencia de gamt.
MX2022002437A (es) 2019-08-30 2022-06-02 Aeglea Biotherapeutics Inc Metodos para la produccion de arginasa humana recombinante 1 y sus usos.
CN114214353B (zh) * 2021-11-30 2023-07-18 新泰市佳禾生物科技有限公司 一种发酵生产人重组精氨酸酶i的方法
CN114350721B (zh) * 2021-11-30 2024-05-24 新泰市佳禾生物科技有限公司 微生物酶法生产l-鸟氨酸的方法
CN114196712B (zh) * 2021-11-30 2024-04-02 新泰市佳禾生物科技有限公司 一种固定化酶法生产l-鸟氨酸的方法
CN116179452A (zh) * 2022-07-11 2023-05-30 山东第一医科大学(山东省医学科学院) 一种用于治疗精氨酸酶缺乏症的工程益生菌及其构建方法和应用
WO2025171346A1 (en) * 2024-02-08 2025-08-14 University Of Florida Research Foundation, Incorporated Indoleamine 2,3-dioxygenase variants

Citations (1)

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US5780286A (en) 1996-03-14 1998-07-14 Smithkline Beecham Corporation Arginase II
EP1499342A2 (en) 2002-01-25 2005-01-26 Cancer Treatments International Therapeutic composition for treatment of cancer by arginine depletion
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MX2025006207A (es) 2025-07-01
TW201924715A (zh) 2019-07-01
AU2018378485A1 (en) 2020-07-23
MX2020005933A (es) 2021-01-15
CA3084801A1 (en) 2019-06-13
US20190167770A1 (en) 2019-06-06
KR20200122298A (ko) 2020-10-27
US11717562B2 (en) 2023-08-08
WO2019113157A1 (en) 2019-06-13
CN111787941A (zh) 2020-10-16
TWI878212B (zh) 2025-04-01
AU2018378485B2 (en) 2024-06-20
EP3720476A1 (en) 2020-10-14
CN120643682A (zh) 2025-09-16
JP2024028760A (ja) 2024-03-05
US20240009284A1 (en) 2024-01-11
CN120661640A (zh) 2025-09-19

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