AU2018378485B2 - Method and composition for treating arginase 1 deficiency - Google Patents

Method and composition for treating arginase 1 deficiency Download PDF

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Publication number
AU2018378485B2
AU2018378485B2 AU2018378485A AU2018378485A AU2018378485B2 AU 2018378485 B2 AU2018378485 B2 AU 2018378485B2 AU 2018378485 A AU2018378485 A AU 2018378485A AU 2018378485 A AU2018378485 A AU 2018378485A AU 2018378485 B2 AU2018378485 B2 AU 2018378485B2
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arginase
human
arginine
patient
patients
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AU2018378485A1 (en
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Anthony G. Quinn
Scott W. Rowlinson
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Immedica Pharma AB
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Immedica Pharma AB
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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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Biomedical Technology (AREA)
  • Dermatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pain & Pain Management (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2018378485A 2017-12-05 2018-12-05 Method and composition for treating arginase 1 deficiency Active AU2018378485B2 (en)

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

Publications (2)

Publication Number Publication Date
AU2018378485A1 AU2018378485A1 (en) 2020-07-23
AU2018378485B2 true AU2018378485B2 (en) 2024-06-20

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ID=64734275

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AU2018378485A Active AU2018378485B2 (en) 2017-12-05 2018-12-05 Method and composition for treating arginase 1 deficiency

Country Status (10)

Country Link
US (2) US11717562B2 (https=)
EP (1) EP3720476A1 (https=)
JP (2) JP7827407B2 (https=)
KR (2) KR20200122298A (https=)
CN (3) CN120661640A (https=)
AU (1) AU2018378485B2 (https=)
CA (1) CA3084801A1 (https=)
MX (2) MX2020005933A (https=)
TW (1) TWI878212B (https=)
WO (1) WO2019113157A1 (https=)

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Publication number Priority date Publication date Assignee Title
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
CA3116736A1 (en) * 2018-10-19 2020-04-23 Aerase, Inc. Arginine depletion therapy for treatment of gamt deficiency
CN114729344A (zh) * 2019-08-30 2022-07-08 安格利亚生物制药公司 人重组精氨酸酶1的生产方法及其用途
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
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
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
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

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JPH02117383A (ja) 1988-10-26 1990-05-01 Tosoh Corp ヒトアルギナーゼの製造方法
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
HK1053577A2 (en) 2002-06-20 2003-10-10 Bio-Cancer Treatment International Limited Pharmaceutical composition and method of treatment of human malignanices with arginine deprivation
GB0315248D0 (en) * 2003-06-30 2003-08-06 Hoffmann La Roche HCV regulated protein expression
FR2873925B1 (fr) 2004-08-05 2006-10-13 Erytech Pharma Soc Par Actions Procede et dispositif de lyse-rescellement pour l'incorporation de principe actif notamment asparaginase ou inositol hexaphosphate, dans des erythrocytes
CN1745847A (zh) 2004-09-08 2006-03-15 康达医药科技有限公司 用精氨酸酶治疗肝炎的药物组合物和方法
DE602006019916D1 (de) 2005-12-12 2011-03-10 Canji Inc Adenovirale expressionsvektoren mit einer expressionskassette im e1-bereich und einer inaktivierten e2b-polymerase
PT3778885T (pt) 2008-10-31 2023-05-31 Aerase Inc Composições de arginases humanas modificadas e métodos para o tratamento do cancro
US9382525B2 (en) 2009-03-26 2016-07-05 The Hong Kong Polytechnic University Site-directed pegylation of arginases and the use thereof as anti-cancer and anti-viral agents
CA2766039A1 (en) 2009-06-29 2011-01-20 The Board Of Regents Of The University Of Texas System Arginase formulations and methods
WO2012061015A2 (en) 2010-11-03 2012-05-10 Scott & White Healthcare L-citrulline supplementation during arginine depletion therapy with arginase
CN103184209B (zh) * 2011-12-27 2015-09-16 拜奥生物科技（上海）有限公司 人精氨酸酶和聚乙二醇化人精氨酸酶及其应用
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
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EP4063503A1 (en) 2014-02-11 2022-09-28 The Regents of the University of Colorado, a body corporate Crispr enabled multiplexed genome engineering
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Also Published As

Publication number Publication date
JP7827407B2 (ja) 2026-03-10
JP2021505620A (ja) 2021-02-18
TWI878212B (zh) 2025-04-01
KR20200122298A (ko) 2020-10-27
CA3084801A1 (en) 2019-06-13
AU2018378485A1 (en) 2020-07-23
CN120643682A (zh) 2025-09-16
US11717562B2 (en) 2023-08-08
EP3720476A1 (en) 2020-10-14
MX2020005933A (es) 2021-01-15
CN111787941A (zh) 2020-10-16
MX2025006207A (es) 2025-07-01
US20240009284A1 (en) 2024-01-11
WO2019113157A1 (en) 2019-06-13
US20190167770A1 (en) 2019-06-06
TW201924715A (zh) 2019-07-01
CN120661640A (zh) 2025-09-19
KR20260047622A (ko) 2026-04-08
JP2024028760A (ja) 2024-03-05

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