KR102292298B1 - 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도 - Google Patents

산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도 Download PDF

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KR102292298B1
KR102292298B1 KR1020217007269A KR20217007269A KR102292298B1 KR 102292298 B1 KR102292298 B1 KR 102292298B1 KR 1020217007269 A KR1020217007269 A KR 1020217007269A KR 20217007269 A KR20217007269 A KR 20217007269A KR 102292298 B1 KR102292298 B1 KR 102292298B1
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spm
lyso
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KR20210031541A (ko
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웨이-리엔 추앙
제럴드 에프. 콕스
엑스. 케이트 장
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젠자임 코포레이션
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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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/916—Hydrolases (3) acting on ester bonds (3.1), e.g. phosphatases (3.1.3), phospholipases C or phospholipases D (3.1.4)
    • G01N2333/922—Ribonucleases (RNAses); Deoxyribonucleases (DNAses)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2405/00—Assays, e.g. immunoassays or enzyme assays, involving lipids
    • G01N2405/08—Sphingolipids
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis
    • 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
    • G01N33/92—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving lipids, e.g. cholesterol, lipoproteins, or their receptors

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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)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • General Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Food Science & Technology (AREA)
  • Cell Biology (AREA)
  • Biotechnology (AREA)
  • Diabetes (AREA)
  • Pulmonology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
KR1020217007269A 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도 Active KR102292298B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020217025969A KR102434244B1 (ko) 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361832302P 2013-06-07 2013-06-07
US61/832,302 2013-06-07
PCT/US2014/041405 WO2014197859A1 (en) 2013-06-07 2014-06-06 Marker for acid sphingomyelinase disorders and uses thereof
KR1020167000011A KR102228367B1 (ko) 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도

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KR1020167000011A Division KR102228367B1 (ko) 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도

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KR1020217025969A Division KR102434244B1 (ko) 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도

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KR20210031541A KR20210031541A (ko) 2021-03-19
KR102292298B1 true KR102292298B1 (ko) 2021-08-24

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KR1020217007269A Active KR102292298B1 (ko) 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도
KR1020167000011A Active KR102228367B1 (ko) 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도
KR1020217025969A Active KR102434244B1 (ko) 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도

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KR1020167000011A Active KR102228367B1 (ko) 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도
KR1020217025969A Active KR102434244B1 (ko) 2013-06-07 2014-06-06 산 스핑고미엘리나제 장애에 대한 마커 및 이의 용도

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US (4) US10022428B2 (https=)
EP (2) EP3004896B1 (https=)
JP (5) JP6457501B2 (https=)
KR (3) KR102292298B1 (https=)
CN (2) CN105474017A (https=)
AU (5) AU2014274670B2 (https=)
CA (1) CA2914751C (https=)
CL (1) CL2015003563A1 (https=)
CR (1) CR20150638A (https=)
DK (1) DK3004896T3 (https=)
EA (1) EA035342B1 (https=)
ES (1) ES2762608T3 (https=)
HR (1) HRP20192246T1 (https=)
HU (1) HUE047863T2 (https=)
IL (3) IL298675B2 (https=)
LT (1) LT3004896T (https=)
MA (1) MA38638B1 (https=)
MX (1) MX370570B (https=)
MY (1) MY185990A (https=)
NZ (1) NZ754328A (https=)
PH (1) PH12015502687A1 (https=)
PL (1) PL3004896T3 (https=)
PT (1) PT3004896T (https=)
RS (1) RS59677B1 (https=)
SG (2) SG11201509622WA (https=)
SI (1) SI3004896T1 (https=)
UA (1) UA120591C2 (https=)
WO (1) WO2014197859A1 (https=)
ZA (1) ZA201508630B (https=)

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SI3998078T1 (sl) * 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
LT3004896T (lt) * 2013-06-07 2020-01-10 Genzyme Corporation Rūgštinės sfingomielinazės sutrikimų žymuo ir jo panaudojimas
JP6984854B2 (ja) * 2015-10-08 2021-12-22 国立大学法人千葉大学 ニーマン・ピック病ｃ型を予防または治療するための医薬組成物
KR20250025051A (ko) * 2017-08-24 2025-02-20 사노피 산성 스핑고미엘린분해효소 결핍증 환자에서의 비정상적 골병태의 치료
AU2018345303B2 (en) 2017-10-02 2025-09-18 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
EP3976218B1 (en) * 2019-05-31 2025-02-19 Genzyme Corporation Two-dimensional lc-ms/ms systems
EP4562432A2 (en) * 2022-07-28 2025-06-04 Wylder Nation Foundation Compositions and methods for diagnosing, treating, and preventing lysosomal storage diseases

Citations (1)

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WO2013072060A2 (en) 2011-11-15 2013-05-23 Centogene Gmbh Method for the diagnosis of niemann-pick disease

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US4082781A (en) 1976-06-04 1978-04-04 The United States Of America As Represented By The Department Of Health, Education And Welfare Synthesis of 2-alkanoylamino-4-nitrophenyl phosphorylcholine-hydroxide
US4039388A (en) 1976-06-04 1977-08-02 The United States Of America As Represented By The Government Diagnostic test for Niemann-Pick disease
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
WO2002018539A2 (en) 2000-08-25 2002-03-07 Lysometrix Corporation Method for assaying the activity of lysosomal enzymes
US7001994B2 (en) 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
US7615224B2 (en) 2004-12-02 2009-11-10 Women's And Children's Hospital Multiplex-bead complex for determination of lysosomal storage disorders
JP2006214731A (ja) 2005-02-01 2006-08-17 Institute Of Physical & Chemical Research スフィンゴミエリンの検出方法
JP2009519713A (ja) 2005-12-15 2009-05-21 ザ リサーチ ファウンデーション オブ ステート ユニバーシティ オブ ニューヨーク 血漿中及び組織中のスフィンゴエミリン及びホスファチジルコリンの酵素的測定方法
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
US8026099B2 (en) 2007-07-26 2011-09-27 Washington University Lipid profile as a biomarker for early detection of neurological disorders
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SI3998078T1 (sl) * 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
JP2013002933A (ja) 2011-06-16 2013-01-07 Hitachi Plant Technologies Ltd ３次元測位システム
ES2731920T3 (es) * 2013-05-14 2019-11-19 Centogene Ag Método para el diagnóstico de la enfermedad de Niemann-Pick
LT3004896T (lt) * 2013-06-07 2020-01-10 Genzyme Corporation Rūgštinės sfingomielinazės sutrikimų žymuo ir jo panaudojimas

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2013072060A2 (en) 2011-11-15 2013-05-23 Centogene Gmbh Method for the diagnosis of niemann-pick disease

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Neurochemical Reserach, 1999, Vol. 24, pp 199-205.

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AU2018200977A1 (en) 2018-03-01
US10022428B2 (en) 2018-07-17
IL284102B2 (en) 2023-04-01
IL284102A (en) 2021-07-29
EP3004896B1 (en) 2019-09-18
CN115128287A (zh) 2022-09-30
JP2019089767A (ja) 2019-06-13
IL298675A (en) 2023-01-01
US20240398907A1 (en) 2024-12-05
AU2023201062B2 (en) 2025-05-29
IL284102B (en) 2022-12-01
HUE047863T2 (hu) 2020-05-28
NZ754328A (en) 2021-12-24
EA201592265A1 (ru) 2016-04-29
BR112015030099A2 (pt) 2017-07-25
US20210162021A1 (en) 2021-06-03
MA38638B1 (fr) 2018-03-30
US11998592B2 (en) 2024-06-04
PH12015502687A1 (en) 2016-03-07
JP2021075544A (ja) 2021-05-20
MX2015016844A (es) 2016-04-04
JP7830414B2 (ja) 2026-03-16
US10888607B2 (en) 2021-01-12
JP2016526050A (ja) 2016-09-01
EP3584580A1 (en) 2019-12-25
EA035342B1 (ru) 2020-05-29
NZ715001A (en) 2021-10-29
CR20150638A (es) 2016-02-08
JP6457501B2 (ja) 2019-01-23
ES2762608T3 (es) 2020-05-25
MY185990A (en) 2021-06-14
PT3004896T (pt) 2019-12-23
MA38638A1 (fr) 2017-02-28
CN105474017A (zh) 2016-04-06
IL298675B2 (en) 2025-12-01
HK1217534A1 (en) 2017-01-13
IL242964B (en) 2021-07-29
SG10201709925SA (en) 2017-12-28
DK3004896T3 (da) 2020-01-06
SI3004896T1 (sl) 2020-01-31
LT3004896T (lt) 2020-01-10
KR20210031541A (ko) 2021-03-19
WO2014197859A1 (en) 2014-12-11
AU2020257114A1 (en) 2020-11-19
AU2025220876A1 (en) 2025-09-11
KR20210104177A (ko) 2021-08-24
CA2914751A1 (en) 2014-12-11
US12589138B2 (en) 2026-03-31
CA2914751C (en) 2023-10-03
JP2026041958A (ja) 2026-03-10
AU2014274670B2 (en) 2017-11-09
UA120591C2 (uk) 2020-01-10
MX370570B (es) 2019-12-17
KR20160018651A (ko) 2016-02-17
SG11201509622WA (en) 2015-12-30
IL298675B1 (en) 2025-08-01
EP3004896A1 (en) 2016-04-13
ZA201508630B (en) 2020-01-29
JP7414750B2 (ja) 2024-01-16
AU2018200977B2 (en) 2020-07-23
HRP20192246T1 (hr) 2020-03-06
JP2024038145A (ja) 2024-03-19
KR102228367B1 (ko) 2021-03-16
JP6835806B2 (ja) 2021-02-24
US20180289778A1 (en) 2018-10-11
RS59677B1 (sr) 2020-01-31
AU2023201062A1 (en) 2023-03-23
AU2014274670A1 (en) 2016-01-07
AU2020257114B2 (en) 2023-03-16
KR102434244B1 (ko) 2022-08-19
US20160120958A1 (en) 2016-05-05
PL3004896T3 (pl) 2020-04-30
CL2015003563A1 (es) 2016-08-05

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