HRP20192246T1 - Marker za poremećaje kisele sfingomijelinaze i njegova uporaba - Google Patents

Marker za poremećaje kisele sfingomijelinaze i njegova uporaba Download PDF

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Publication number
HRP20192246T1
HRP20192246T1 HRP20192246TT HRP20192246T HRP20192246T1 HR P20192246 T1 HRP20192246 T1 HR P20192246T1 HR P20192246T T HRP20192246T T HR P20192246TT HR P20192246 T HRP20192246 T HR P20192246T HR P20192246 T1 HRP20192246 T1 HR P20192246T1
Authority
HR
Croatia
Prior art keywords
therapeutic agent
dose
use according
subject
lyso
Prior art date
Application number
HRP20192246TT
Other languages
English (en)
Croatian (hr)
Inventor
Wei-Lien Chuang
Gerald F. Cox
X. Kate Zhang
Original Assignee
Genzyme Corporation
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 Corporation filed Critical Genzyme Corporation
Publication of HRP20192246T1 publication Critical patent/HRP20192246T1/hr

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Classifications

    • 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
    • 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
    • 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)
  • 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)
HRP20192246TT 2013-06-07 2014-06-06 Marker za poremećaje kisele sfingomijelinaze i njegova uporaba HRP20192246T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361832302P 2013-06-07 2013-06-07
EP14735056.5A EP3004896B1 (en) 2013-06-07 2014-06-06 Marker for acid sphingomyelinase disorders and uses thereof
PCT/US2014/041405 WO2014197859A1 (en) 2013-06-07 2014-06-06 Marker for acid sphingomyelinase disorders and uses thereof

Publications (1)

Publication Number Publication Date
HRP20192246T1 true HRP20192246T1 (hr) 2020-03-06

Family

ID=51062988

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20192246TT HRP20192246T1 (hr) 2013-06-07 2014-06-06 Marker za poremećaje kisele sfingomijelinaze i njegova uporaba

Country Status (29)

Country Link
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=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US8497122B2 (en) 2008-04-11 2013-07-30 Washington University Biomarkers for Niemann-pick C disease and related disorders
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
CA2854668A1 (en) 2011-11-15 2013-05-23 Centogene Ag Method for the diagnosis of niemann-pick disease
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

Also Published As

Publication number Publication date
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
JP2024038145A (ja) 2024-03-19
KR102292298B1 (ko) 2021-08-24
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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