IL298675B2 - Marker for acid sphingomyelinase disorders and uses thereof - Google Patents
Marker for acid sphingomyelinase disorders and uses thereofInfo
- Publication number
- IL298675B2 IL298675B2 IL298675A IL29867522A IL298675B2 IL 298675 B2 IL298675 B2 IL 298675B2 IL 298675 A IL298675 A IL 298675A IL 29867522 A IL29867522 A IL 29867522A IL 298675 B2 IL298675 B2 IL 298675B2
- Authority
- IL
- Israel
- Prior art keywords
- rhasm
- spm
- niemann
- asm
- dose
- Prior art date
Links
Classifications
-
- 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
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)
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 |
|---|---|
| IL298675A IL298675A (en) | 2023-01-01 |
| IL298675B1 IL298675B1 (en) | 2025-08-01 |
| IL298675B2 true IL298675B2 (en) | 2025-12-01 |
Family
ID=51062988
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL298675A IL298675B2 (en) | 2013-06-07 | 2014-06-06 | Marker for acid sphingomyelinase disorders and uses thereof |
| 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 |
Family Applications After (2)
| 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 |
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)
| 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)
| 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 |
-
2014
- 2014-06-06 LT LTEP14735056.5T patent/LT3004896T/lt unknown
- 2014-06-06 WO PCT/US2014/041405 patent/WO2014197859A1/en not_active Ceased
- 2014-06-06 PL PL14735056T patent/PL3004896T3/pl unknown
- 2014-06-06 IL IL298675A patent/IL298675B2/en unknown
- 2014-06-06 NZ NZ754328A patent/NZ754328A/en unknown
- 2014-06-06 HR HRP20192246TT patent/HRP20192246T1/hr unknown
- 2014-06-06 JP JP2016518053A patent/JP6457501B2/ja active Active
- 2014-06-06 CA CA2914751A patent/CA2914751C/en active Active
- 2014-06-06 CN CN201480043918.2A patent/CN105474017A/zh active Pending
- 2014-06-06 US US14/895,472 patent/US10022428B2/en active Active
- 2014-06-06 ES ES14735056T patent/ES2762608T3/es active Active
- 2014-06-06 EP EP14735056.5A patent/EP3004896B1/en active Active
- 2014-06-06 SG SG11201509622WA patent/SG11201509622WA/en unknown
- 2014-06-06 KR KR1020217007269A patent/KR102292298B1/ko active Active
- 2014-06-06 MA MA38638A patent/MA38638B1/fr unknown
- 2014-06-06 HU HUE14735056A patent/HUE047863T2/hu unknown
- 2014-06-06 EA EA201592265A patent/EA035342B1/ru not_active IP Right Cessation
- 2014-06-06 IL IL284102A patent/IL284102B2/en unknown
- 2014-06-06 RS RS20191627A patent/RS59677B1/sr unknown
- 2014-06-06 KR KR1020167000011A patent/KR102228367B1/ko active Active
- 2014-06-06 KR KR1020217025969A patent/KR102434244B1/ko active Active
- 2014-06-06 MY MYPI2015704368A patent/MY185990A/en unknown
- 2014-06-06 DK DK14735056.5T patent/DK3004896T3/da active
- 2014-06-06 SG SG10201709925SA patent/SG10201709925SA/en unknown
- 2014-06-06 EP EP19191053.8A patent/EP3584580A1/en active Pending
- 2014-06-06 AU AU2014274670A patent/AU2014274670B2/en active Active
- 2014-06-06 PT PT147350565T patent/PT3004896T/pt unknown
- 2014-06-06 UA UAA201600158A patent/UA120591C2/uk unknown
- 2014-06-06 MX MX2015016844A patent/MX370570B/es active IP Right Grant
- 2014-06-06 SI SI201431434T patent/SI3004896T1/sl unknown
- 2014-06-06 CN CN202210555370.6A patent/CN115128287A/zh active Pending
-
2015
- 2015-11-23 ZA ZA2015/08630A patent/ZA201508630B/en unknown
- 2015-12-01 PH PH12015502687A patent/PH12015502687A1/en unknown
- 2015-12-04 CR CR20150638A patent/CR20150638A/es unknown
- 2015-12-04 CL CL2015003563A patent/CL2015003563A1/es unknown
- 2015-12-06 IL IL242964A patent/IL242964B/en unknown
-
2018
- 2018-02-09 AU AU2018200977A patent/AU2018200977B2/en active Active
- 2018-06-08 US US16/003,598 patent/US10888607B2/en active Active
- 2018-12-19 JP JP2018236856A patent/JP6835806B2/ja active Active
-
2020
- 2020-10-22 AU AU2020257114A patent/AU2020257114B2/en active Active
- 2020-12-11 US US17/119,035 patent/US11998592B2/en active Active
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2021
- 2021-02-03 JP JP2021015390A patent/JP7414750B2/ja active Active
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2023
- 2023-02-23 AU AU2023201062A patent/AU2023201062B2/en active Active
- 2023-12-27 JP JP2023220385A patent/JP7830414B2/ja active Active
-
2024
- 2024-04-29 US US18/649,453 patent/US12589138B2/en active Active
-
2025
- 2025-08-25 AU AU2025220876A patent/AU2025220876A1/en active Pending
- 2025-12-10 JP JP2025244095A patent/JP2026041958A/ja active Pending
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