JP2021516757A - ファーバー病マーカーおよびその使用 - Google Patents

ファーバー病マーカーおよびその使用 Download PDF

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Publication number
JP2021516757A
JP2021516757A JP2020543884A JP2020543884A JP2021516757A JP 2021516757 A JP2021516757 A JP 2021516757A JP 2020543884 A JP2020543884 A JP 2020543884A JP 2020543884 A JP2020543884 A JP 2020543884A JP 2021516757 A JP2021516757 A JP 2021516757A
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JP
Japan
Prior art keywords
cd11b
mhcii
subject
farber
disease
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JP2020543884A
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English (en)
Japanese (ja)
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JP2021516757A5 (enExample
Inventor
コクリ，クリスティン
サンピー，ブランテ
Original Assignee
エンジーヴァント セラピューティックス ゲーエムベーハー
エンジーヴァント セラピューティックス ゲーエムベーハー
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Application filed by エンジーヴァント セラピューティックス ゲーエムベーハー, エンジーヴァント セラピューティックス ゲーエムベーハー filed Critical エンジーヴァント セラピューティックス ゲーエムベーハー
Publication of JP2021516757A publication Critical patent/JP2021516757A/ja
Publication of JP2021516757A5 publication Critical patent/JP2021516757A5/ja
Priority to JP2023127159A priority Critical patent/JP2023159164A/ja
Pending legal-status Critical Current

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    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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)
    • C12Y305/01—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
    • C12Y305/01023—Ceramidase (3.5.1.23)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/04—Endocrine or metabolic disorders

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Food Science & Technology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020543884A 2018-03-27 2019-03-22 ファーバー病マーカーおよびその使用 Pending JP2021516757A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023127159A JP2023159164A (ja) 2018-03-27 2023-08-03 ファーバー病マーカーおよびその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862648775P 2018-03-27 2018-03-27
US62/648,775 2018-03-27
PCT/IB2019/000290 WO2019186272A1 (en) 2018-03-27 2019-03-22 Farber disease markers and uses thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023127159A Division JP2023159164A (ja) 2018-03-27 2023-08-03 ファーバー病マーカーおよびその使用

Publications (2)

Publication Number Publication Date
JP2021516757A true JP2021516757A (ja) 2021-07-08
JP2021516757A5 JP2021516757A5 (enExample) 2022-03-28

Family

ID=66530353

Family Applications (2)

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JP2020543884A Pending JP2021516757A (ja) 2018-03-27 2019-03-22 ファーバー病マーカーおよびその使用
JP2023127159A Pending JP2023159164A (ja) 2018-03-27 2023-08-03 ファーバー病マーカーおよびその使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023127159A Pending JP2023159164A (ja) 2018-03-27 2023-08-03 ファーバー病マーカーおよびその使用

Country Status (16)

Country Link
US (2) US20190302126A1 (enExample)
EP (1) EP3775924A1 (enExample)
JP (2) JP2021516757A (enExample)
KR (2) KR20240111011A (enExample)
CN (2) CN111971562A (enExample)
AU (1) AU2019244477A1 (enExample)
BR (1) BR112020016435A2 (enExample)
CA (1) CA3090354A1 (enExample)
CL (1) CL2020002105A1 (enExample)
CO (1) CO2020010043A2 (enExample)
IL (2) IL311212A (enExample)
MX (1) MX2020008377A (enExample)
PH (1) PH12020551206A1 (enExample)
RU (1) RU2020119065A (enExample)
SG (1) SG11202007508TA (enExample)
WO (1) WO2019186272A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220088158A1 (en) * 2019-01-23 2022-03-24 Aceragen, Inc. Method of ameliorating a pro-inflammatory immunophenotype in farber disease subjects by repeated administration of a recombinant human acid ceramidase
CA3209730A1 (en) * 2021-03-29 2022-10-06 Alan Parker Modified adenovirus
CN117647649A (zh) * 2023-08-15 2024-03-05 迈健医药科技无锡有限公司 一种免疫细胞全维分析的方法

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016520519A (ja) * 2013-03-14 2016-07-14 アイカーン スクール オブ メディシン アット マウント サイナイ 治療用酸性セラミダーゼ組成物ならびにそれを作製および使用する方法

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5059421A (en) 1985-07-26 1991-10-22 The Liposome Company, Inc. Preparation of targeted liposome systems of a defined size distribution
US5631237A (en) 1992-12-22 1997-05-20 Dzau; Victor J. Method for producing in vivo delivery of therapeutic agents via liposomes
CA2163860A1 (en) 1993-06-30 1995-01-12 Chung C. Hsu Method for preparing liposomes
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5643599A (en) 1995-06-07 1997-07-01 President And Fellows Of Harvard College Intracellular delivery of macromolecules
EP3115786A1 (en) * 2015-07-08 2017-01-11 Centogene AG Method for the diagnosis of farber's disease

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016520519A (ja) * 2013-03-14 2016-07-14 アイカーン スクール オブ メディシン アット マウント サイナイ 治療用酸性セラミダーゼ組成物ならびにそれを作製および使用する方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
"Deletion of MCP-1 Impedes Pathogenesis of Acid Ceramidase Deficiency", SCIENTIFIC REPORTS, vol. 8, JPN6023004188, 29 January 2018 (2018-01-29), pages 1 - 16, ISSN: 0004983213 *

Also Published As

Publication number Publication date
BR112020016435A2 (pt) 2020-12-15
KR20200136367A (ko) 2020-12-07
MX2020008377A (es) 2020-09-25
CO2020010043A2 (es) 2020-11-10
RU2020119065A (ru) 2022-04-27
PH12020551206A1 (en) 2021-04-19
EP3775924A1 (en) 2021-02-17
CN111971562A (zh) 2020-11-20
SG11202007508TA (en) 2020-09-29
CL2020002105A1 (es) 2020-12-04
CN118112249A (zh) 2024-05-31
US20190302126A1 (en) 2019-10-03
IL311212A (en) 2024-05-01
WO2019186272A1 (en) 2019-10-03
US20210199670A1 (en) 2021-07-01
AU2019244477A1 (en) 2020-06-25
CA3090354A1 (en) 2019-10-03
JP2023159164A (ja) 2023-10-31
KR20240111011A (ko) 2024-07-16
IL276420A (en) 2020-09-30

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