CA3084801A1 - Method and composition for treating arginase 1 deficiency - Google Patents

Method and composition for treating arginase 1 deficiency Download PDF

Info

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
Authority
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
Other languages
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

Links

Classifications

    • 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)

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)
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 (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

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
FR3005420B1 (fr) 2013-05-07 2015-09-18 Erytech Pharma Procede de stabilisation de suspensions d'erythrocytes encapsulant un principe actif, suspensions obtenues.
EP4063503A1 (en) 2014-02-11 2022-09-28 The Regents of the University of Colorado, a body corporate Crispr enabled multiplexed genome engineering
CA2944903A1 (en) 2014-04-24 2015-10-29 Dana-Farber Cancer Institute, Inc. Tumor suppressor and oncogene biomarkers predictive of anti-immune checkpoint inhibitor response
ES2897635T3 (es) * 2014-04-29 2022-03-02 Bio Cancer Treat International Ltd Métodos y composiciones para modular el sistema inmunológico con la arginasa I
EP2982758A1 (en) 2014-08-04 2016-02-10 Centre Hospitalier Universitaire Vaudois (CHUV) Genome editing for the treatment of huntington's disease
HRP20190881T1 (hr) 2014-08-28 2019-07-12 Halozyme, Inc. Kombinacijska terapija s hijaluronan-razgrađujućim enzimom i inhibitorom imunološke kontrolne točke
US11136597B2 (en) 2016-02-16 2021-10-05 Yale University Compositions for enhancing targeted gene editing and methods of use thereof
ES2909074T3 (es) * 2016-05-06 2022-05-05 Phasebio Pharmaceuticals Inc Proteínas de fusión ELP para liberación controlada y sostenida

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
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
CN120661640A (zh) 2025-09-19
KR20260047622A (ko) 2026-04-08
JP2024028760A (ja) 2024-03-05

Similar Documents

Publication Publication Date Title
US20240009284A1 (en) Method and composition for treating arginase 1 deficiency
US12414985B2 (en) Augmented acid alpha-glucosidase for the treatment of Pompe disease
US11065306B2 (en) Methods for treating hypophosphatasia in children
KR20220162816A (ko) 알칼리성 포스파타아제로 근육 약화의 치료
JP2025003988A (ja) Ｇａｍｔ欠損症の治療のためのアルギニン枯渇療法
Sarkissian et al. Evaluation of orally administered PEGylated phenylalanine ammonia lyase in mice for the treatment of Phenylketonuria
CN110719786A (zh) 用于治疗成人和青少年的低磷酸酯酶症(hpp)的方法
CN118064414A (zh) 治疗同型半胱氨酸尿症的酶替代疗法优化
US20250228921A1 (en) Treatment of arginase 1 deficiency
HK40039953A (en) Method and composition for treating arginase 1 deficiency
Jakubowski The Origin and Role of N-Homocysteinylated Proteins in Cardiovascular Disease
EP3397273A1 (en) Augmented acid alpha-glucosidase for the treatment of pompe disease

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20231205

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 6TH ANNIV.) - STANDARD

Year of fee payment: 6

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241009

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241009

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20250501

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20250902

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20251112

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20251112

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20251112

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 7TH ANNIV.) - STANDARD

Year of fee payment: 7

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20251121

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20251121