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 (https=
JPWO2019186272A5 (https=
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
Publication of JPWO2019186272A5 publication Critical patent/JPWO2019186272A5/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)
    • 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)
  • Immunology (AREA)
  • General Health & Medical Sciences (AREA)
  • Hematology (AREA)
  • Medicinal Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Food Science & Technology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Organic Chemistry (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (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 (3)

Publication Number Publication Date
JP2021516757A true JP2021516757A (ja) 2021-07-08
JP2021516757A5 JP2021516757A5 (https=) 2022-03-28
JPWO2019186272A5 JPWO2019186272A5 (https=) 2022-03-28

Family

ID=66530353

Family Applications (2)

Application Number Title Priority Date Filing Date
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 (15)

Country Link
EP (1) EP3775924A1 (https=)
JP (2) JP2021516757A (https=)
KR (2) KR20200136367A (https=)
CN (2) CN111971562A (https=)
AU (1) AU2019244477A1 (https=)
BR (1) BR112020016435A2 (https=)
CA (1) CA3090354A1 (https=)
CL (1) CL2020002105A1 (https=)
CO (1) CO2020010043A2 (https=)
IL (2) IL311212A (https=)
MX (1) MX2020008377A (https=)
PH (1) PH12020551206A1 (https=)
RU (1) RU2020119065A (https=)
SG (1) SG11202007508TA (https=)
WO (1) WO2019186272A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020152532A1 (en) * 2019-01-23 2020-07-30 Enzyvant Therapeutics Gmbh Method of ameliorating a pro-inflammatory immunophenotype in farber disease subjects by repeated administration of a recombinant human acid ceramidase
US20240180982A1 (en) * 2021-03-29 2024-06-06 University College Cardiff Consultants Limited Modified adenovirus

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
WO1995001164A1 (en) 1993-06-30 1995-01-12 Genentech, Inc. 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
CN111971562A (zh) 2020-11-20
RU2020119065A (ru) 2022-04-27
WO2019186272A1 (en) 2019-10-03
EP3775924A1 (en) 2021-02-17
AU2019244477A1 (en) 2020-06-25
CN118112249A (zh) 2024-05-31
IL276420A (en) 2020-09-30
JP2023159164A (ja) 2023-10-31
CA3090354A1 (en) 2019-10-03
MX2020008377A (es) 2020-09-25
PH12020551206A1 (en) 2021-04-19
BR112020016435A2 (pt) 2020-12-15
SG11202007508TA (en) 2020-09-29
CL2020002105A1 (es) 2020-12-04
IL311212A (en) 2024-05-01
KR20200136367A (ko) 2020-12-07
CO2020010043A2 (es) 2020-11-10
KR20240111011A (ko) 2024-07-16

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