CA3084801A1 - 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
CA3084801A1
CA3084801A1 CA3084801A CA3084801A CA3084801A1 CA 3084801 A1 CA3084801 A1 CA 3084801A1 CA 3084801 A CA3084801 A CA 3084801A CA 3084801 A CA3084801 A CA 3084801A CA 3084801 A1 CA3084801 A1 CA 3084801A1
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CA
Canada
Prior art keywords
arginase
subject
arginine
plasma level
gaa
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
CA3084801A
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English (en)
French (fr)
Inventor
Scott W. Rowlinson
Anthony G. Quinn
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.)
Immedica Pharma AB
Original Assignee
AERase 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 AERase Inc filed Critical AERase Inc
Publication of CA3084801A1 publication Critical patent/CA3084801A1/en
Pending legal-status Critical Current

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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
    • A61K47/02—Inorganic compounds
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • 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
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Dermatology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Inorganic Chemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pain & Pain Management (AREA)
  • Physical Education & Sports Medicine (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)
CA3084801A 2017-12-05 2018-12-05 Method and composition for treating arginase 1 deficiency Pending CA3084801A1 (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 (1)

Publication Number Publication Date
CA3084801A1 true CA3084801A1 (en) 2019-06-13

Family

ID=64734275

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3084801A Pending CA3084801A1 (en) 2017-12-05 2018-12-05 Method and composition for treating arginase 1 deficiency

Country Status (10)

Country Link
US (2) US11717562B2 (cg-RX-API-DMAC10.html)
EP (1) EP3720476A1 (cg-RX-API-DMAC10.html)
JP (2) JP7827407B2 (cg-RX-API-DMAC10.html)
KR (2) KR20260047622A (cg-RX-API-DMAC10.html)
CN (3) CN120661640A (cg-RX-API-DMAC10.html)
AU (1) AU2018378485B2 (cg-RX-API-DMAC10.html)
CA (1) CA3084801A1 (cg-RX-API-DMAC10.html)
MX (2) MX2020005933A (cg-RX-API-DMAC10.html)
TW (1) TWI878212B (cg-RX-API-DMAC10.html)
WO (1) WO2019113157A1 (cg-RX-API-DMAC10.html)

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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
TWI878212B (zh) 2017-12-05 2025-04-01 瑞典商伊梅爾迪卡製藥公司 用於治療精胺酸酶1缺乏症之方法及組合物
WO2020081994A1 (en) * 2018-10-19 2020-04-23 Aerase, Inc. Arginine depletion therapy for treatment of gamt deficiency
BR112022003633A2 (pt) * 2019-08-30 2022-06-07 Aeglea Biotherapeutics Inc Métodos para produção de arginase 1 recombinante humana e usos da mesma
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
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
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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US5780286A (en) 1996-03-14 1998-07-14 Smithkline Beecham Corporation Arginase II
WO2003063780A2 (en) 2002-01-25 2003-08-07 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
EP1974044B1 (en) 2005-12-12 2011-01-26 CANJI, Inc. Adenoviral expression vectors having an expression cassette in the e1 region and an inactivated e2b polymerase
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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
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TWI878212B (zh) 2017-12-05 2025-04-01 瑞典商伊梅爾迪卡製藥公司 用於治療精胺酸酶1缺乏症之方法及組合物
BR112022003633A2 (pt) 2019-08-30 2022-06-07 Aeglea Biotherapeutics Inc Métodos para produção de arginase 1 recombinante humana e usos da mesma
CN119301247A (zh) 2022-04-11 2025-01-10 伊梅尔迪卡制药公司 精氨酸酶1缺乏症的治疗

Also Published As

Publication number Publication date
WO2019113157A1 (en) 2019-06-13
TWI878212B (zh) 2025-04-01
JP7827407B2 (ja) 2026-03-10
MX2025006207A (es) 2025-07-01
US20190167770A1 (en) 2019-06-06
KR20200122298A (ko) 2020-10-27
US20240009284A1 (en) 2024-01-11
TW201924715A (zh) 2019-07-01
CN120643682A (zh) 2025-09-16
KR20260047622A (ko) 2026-04-08
US11717562B2 (en) 2023-08-08
CN120661640A (zh) 2025-09-19
EP3720476A1 (en) 2020-10-14
AU2018378485B2 (en) 2024-06-20
JP2021505620A (ja) 2021-02-18
MX2020005933A (es) 2021-01-15
JP2024028760A (ja) 2024-03-05
CN111787941A (zh) 2020-10-16
AU2018378485A1 (en) 2020-07-23
US12673093B2 (en) 2026-07-07

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