CN120661640A - 用于治疗精氨酸酶1缺乏症的方法和组合物 - Google Patents

用于治疗精氨酸酶1缺乏症的方法和组合物

Info

Publication number
CN120661640A
CN120661640A CN202510809152.4A CN202510809152A CN120661640A CN 120661640 A CN120661640 A CN 120661640A CN 202510809152 A CN202510809152 A CN 202510809152A CN 120661640 A CN120661640 A CN 120661640A
Authority
CN
China
Prior art keywords
arginase
subject
arginine
gaa
plasma
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
CN202510809152.4A
Other languages
English (en)
Chinese (zh)
Inventor
斯科特·W·罗林森
安东尼·G·奎恩
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.)
Imeldica Pharmaceutical Co
Original Assignee
Imeldica Pharmaceutical Co
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 Imeldica Pharmaceutical Co filed Critical Imeldica Pharmaceutical Co
Publication of CN120661640A publication Critical patent/CN120661640A/zh
Pending legal-status Critical Current

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Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/50Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51Lyases (4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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
    • A61K47/59Medicinal 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
    • A61K47/60Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02Muscle relaxants, e.g. for tetanus or cramps
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/03Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amidines (3.5.3)
    • C12Y305/03001Arginase (3.5.3.1)

Landscapes

  • 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)
CN202510809152.4A 2017-12-05 2018-12-05 用于治疗精氨酸酶1缺乏症的方法和组合物 Pending CN120661640A (zh)

Applications Claiming Priority (8)

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
CN201880079119.9A CN111787941A (zh) 2017-12-05 2018-12-05 用于治疗精氨酸酶1缺乏症的方法和组合物

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN201880079119.9A Division CN111787941A (zh) 2017-12-05 2018-12-05 用于治疗精氨酸酶1缺乏症的方法和组合物

Publications (1)

Publication Number Publication Date
CN120661640A true CN120661640A (zh) 2025-09-19

Family

ID=64734275

Family Applications (3)

Application Number Title Priority Date Filing Date
CN202510809152.4A Pending CN120661640A (zh) 2017-12-05 2018-12-05 用于治疗精氨酸酶1缺乏症的方法和组合物
CN202510809272.4A Pending CN120643682A (zh) 2017-12-05 2018-12-05 用于治疗精氨酸酶1缺乏症的方法和组合物
CN201880079119.9A Pending CN111787941A (zh) 2017-12-05 2018-12-05 用于治疗精氨酸酶1缺乏症的方法和组合物

Family Applications After (2)

Application Number Title Priority Date Filing Date
CN202510809272.4A Pending CN120643682A (zh) 2017-12-05 2018-12-05 用于治疗精氨酸酶1缺乏症的方法和组合物
CN201880079119.9A Pending CN111787941A (zh) 2017-12-05 2018-12-05 用于治疗精氨酸酶1缺乏症的方法和组合物

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=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
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
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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
AU2018378485B2 (en) 2024-06-20
TW201924715A (zh) 2019-07-01
KR20260047622A (ko) 2026-04-08
JP2024028760A (ja) 2024-03-05

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