IL284102B2 - Marker for acid sphingomyelinase disorders and uses thereof - Google Patents

Marker for acid sphingomyelinase disorders and uses thereof

Info

Publication number
IL284102B2
IL284102B2 IL284102A IL28410221A IL284102B2 IL 284102 B2 IL284102 B2 IL 284102B2 IL 284102 A IL284102 A IL 284102A IL 28410221 A IL28410221 A IL 28410221A IL 284102 B2 IL284102 B2 IL 284102B2
Authority
IL
Israel
Prior art keywords
spm
lyso
therapeutic agent
subject
dose
Prior art date
Application number
IL284102A
Other languages
English (en)
Hebrew (he)
Other versions
IL284102B (en
IL284102A (en
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of IL284102A publication Critical patent/IL284102A/en
Publication of IL284102B publication Critical patent/IL284102B/en
Publication of IL284102B2 publication Critical patent/IL284102B2/en

Links

Classifications

    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/92Chemical 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
    • 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)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012Sphingomyelin phosphodiesterase (3.1.4.12)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
    • G01N2333/916Hydrolases (3) acting on ester bonds (3.1), e.g. phosphatases (3.1.3), phospholipases C or phospholipases D (3.1.4)
    • G01N2333/922Ribonucleases (RNAses); Deoxyribonucleases (DNAses)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2405/00Assays, e.g. immunoassays or enzyme assays, involving lipids
    • G01N2405/08Sphingolipids
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/04Endocrine or metabolic disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/52Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (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)
  • Biophysics (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Endocrinology (AREA)
  • Food Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Pulmonology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
IL284102A 2013-06-07 2014-06-06 Marker for acid sphingomyelinase disorders and uses thereof IL284102B2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361832302P 2013-06-07 2013-06-07
PCT/US2014/041405 WO2014197859A1 (en) 2013-06-07 2014-06-06 Marker for acid sphingomyelinase disorders and uses thereof

Publications (3)

Publication Number Publication Date
IL284102A IL284102A (en) 2021-07-29
IL284102B IL284102B (en) 2022-12-01
IL284102B2 true IL284102B2 (en) 2023-04-01

Family

ID=51062988

Family Applications (3)

Application Number Title Priority Date Filing Date
IL284102A IL284102B2 (en) 2013-06-07 2014-06-06 Marker for acid sphingomyelinase disorders and uses thereof
IL242964A IL242964B (en) 2013-06-07 2015-12-06 A marker for disorders of acid sphingomyelinase and its uses
IL298675A IL298675B2 (en) 2013-06-07 2022-11-29 Marker for acid sphingomyelinase disorders and its uses

Family Applications After (2)

Application Number Title Priority Date Filing Date
IL242964A IL242964B (en) 2013-06-07 2015-12-06 A marker for disorders of acid sphingomyelinase and its uses
IL298675A IL298675B2 (en) 2013-06-07 2022-11-29 Marker for acid sphingomyelinase disorders and its uses

Country Status (29)

Country Link
US (4) US10022428B2 (enExample)
EP (2) EP3584580A1 (enExample)
JP (4) JP6457501B2 (enExample)
KR (3) KR102292298B1 (enExample)
CN (2) CN115128287A (enExample)
AU (5) AU2014274670B2 (enExample)
CA (1) CA2914751C (enExample)
CL (1) CL2015003563A1 (enExample)
CR (1) CR20150638A (enExample)
DK (1) DK3004896T3 (enExample)
EA (1) EA035342B1 (enExample)
ES (1) ES2762608T3 (enExample)
HR (1) HRP20192246T1 (enExample)
HU (1) HUE047863T2 (enExample)
IL (3) IL284102B2 (enExample)
LT (1) LT3004896T (enExample)
MA (1) MA38638B1 (enExample)
MX (1) MX370570B (enExample)
MY (1) MY185990A (enExample)
NZ (1) NZ754328A (enExample)
PH (1) PH12015502687A1 (enExample)
PL (1) PL3004896T3 (enExample)
PT (1) PT3004896T (enExample)
RS (1) RS59677B1 (enExample)
SG (2) SG10201709925SA (enExample)
SI (1) SI3004896T1 (enExample)
UA (1) UA120591C2 (enExample)
WO (1) WO2014197859A1 (enExample)
ZA (1) ZA201508630B (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102094253B1 (ko) * 2009-08-28 2020-03-31 이칸 스쿨 오브 메디슨 엣 마운트 시나이 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
PL3004896T3 (pl) * 2013-06-07 2020-04-30 Genzyme Corporation Marker zaburzeń kwaśnej spfingomielinazy i jego zastosowania
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
SG11202001544VA (en) * 2017-08-24 2020-03-30 Sanofi Sa Treatment of abnormal bone conditions in acid sphingomyelinase deficiency patients
BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
JP7568648B2 (ja) * 2019-05-31 2024-10-16 ジェンザイム・コーポレーション 二次元lc-ms/msシステム
EP4562432A2 (en) * 2022-07-28 2025-06-04 Wylder Nation Foundation Compositions and methods for diagnosing, treating, and preventing lysosomal storage diseases

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4039388A (en) 1976-06-04 1977-08-02 The United States Of America As Represented By The Government Diagnostic test for Niemann-Pick disease
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
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
CA2634042A1 (en) 2005-12-15 2007-07-12 The Research Foundation Of State University Of New York Enzymatic methods for measuring plasma and tissue sphingomylelin and phosphatidylcholine
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
US8497122B2 (en) 2008-04-11 2013-07-30 Washington University Biomarkers for Niemann-pick C disease and related disorders
KR102094253B1 (ko) * 2009-08-28 2020-03-31 이칸 스쿨 오브 메디슨 엣 마운트 시나이 산성 스핑고미엘리나제 결핍을 치료하기 위한 투여량 단계적 증가 효소 대체 요법
JP2013002933A (ja) 2011-06-16 2013-01-07 Hitachi Plant Technologies Ltd 3次元測位システム
JP6243846B2 (ja) * 2011-11-15 2017-12-06 セントジーン アーゲー ニーマン・ピック病の診断のための方法
WO2014183873A1 (en) * 2013-05-14 2014-11-20 Centogene Ag Method for the diagnosis of niemann-pick disease
PL3004896T3 (pl) * 2013-06-07 2020-04-30 Genzyme Corporation Marker zaburzeń kwaśnej spfingomielinazy i jego zastosowania

Also Published As

Publication number Publication date
BR112015030099A2 (pt) 2017-07-25
AU2014274670B2 (en) 2017-11-09
JP6457501B2 (ja) 2019-01-23
US20240398907A1 (en) 2024-12-05
AU2023201062A1 (en) 2023-03-23
EP3584580A1 (en) 2019-12-25
WO2014197859A1 (en) 2014-12-11
CR20150638A (es) 2016-02-08
KR20210031541A (ko) 2021-03-19
MX370570B (es) 2019-12-17
PH12015502687A1 (en) 2016-03-07
HRP20192246T1 (hr) 2020-03-06
IL284102B (en) 2022-12-01
JP7414750B2 (ja) 2024-01-16
EA201592265A1 (ru) 2016-04-29
KR102292298B1 (ko) 2021-08-24
SG10201709925SA (en) 2017-12-28
AU2014274670A1 (en) 2016-01-07
US10888607B2 (en) 2021-01-12
CL2015003563A1 (es) 2016-08-05
MA38638A1 (fr) 2017-02-28
AU2018200977A1 (en) 2018-03-01
DK3004896T3 (da) 2020-01-06
KR20210104177A (ko) 2021-08-24
KR102434244B1 (ko) 2022-08-19
IL284102A (en) 2021-07-29
US11998592B2 (en) 2024-06-04
JP2016526050A (ja) 2016-09-01
US20180289778A1 (en) 2018-10-11
MA38638B1 (fr) 2018-03-30
IL298675B2 (en) 2025-12-01
PT3004896T (pt) 2019-12-23
NZ715001A (en) 2021-10-29
KR102228367B1 (ko) 2021-03-16
RS59677B1 (sr) 2020-01-31
EP3004896A1 (en) 2016-04-13
CN105474017A (zh) 2016-04-06
IL298675B1 (en) 2025-08-01
AU2023201062B2 (en) 2025-05-29
CA2914751C (en) 2023-10-03
AU2020257114B2 (en) 2023-03-16
AU2025220876A1 (en) 2025-09-11
PL3004896T3 (pl) 2020-04-30
AU2020257114A1 (en) 2020-11-19
US20160120958A1 (en) 2016-05-05
ZA201508630B (en) 2020-01-29
KR20160018651A (ko) 2016-02-17
HK1217534A1 (en) 2017-01-13
EP3004896B1 (en) 2019-09-18
MY185990A (en) 2021-06-14
JP2024038145A (ja) 2024-03-19
SG11201509622WA (en) 2015-12-30
ES2762608T3 (es) 2020-05-25
US20210162021A1 (en) 2021-06-03
JP2021075544A (ja) 2021-05-20
AU2018200977B2 (en) 2020-07-23
EA035342B1 (ru) 2020-05-29
MX2015016844A (es) 2016-04-04
US10022428B2 (en) 2018-07-17
JP6835806B2 (ja) 2021-02-24
UA120591C2 (uk) 2020-01-10
NZ754328A (en) 2021-12-24
HUE047863T2 (hu) 2020-05-28
IL242964B (en) 2021-07-29
IL298675A (en) 2023-01-01
CN115128287A (zh) 2022-09-30
LT3004896T (lt) 2020-01-10
JP2019089767A (ja) 2019-06-13
SI3004896T1 (sl) 2020-01-31
CA2914751A1 (en) 2014-12-11

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