AR059089A1 - Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal - Google Patents
Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomalInfo
- Publication number
- AR059089A1 AR059089A1 ARP070100239A ARP070100239A AR059089A1 AR 059089 A1 AR059089 A1 AR 059089A1 AR P070100239 A ARP070100239 A AR P070100239A AR P070100239 A ARP070100239 A AR P070100239A AR 059089 A1 AR059089 A1 AR 059089A1
- Authority
- AR
- Argentina
- Prior art keywords
- storage diseases
- enzyme
- lisosomal
- intraventricular administration
- lysosomal storage
- 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
- 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)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- 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)
-
- 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)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
Abstract
Enfermedades de almacenamiento lisosomal pueden tratarse exitosamente mediante la aplicacion intraventricular de la enzima etiologicamente deficiente en la enfermedad. Para lograr el efecto máximo, la administracion puede realizarse lentamente. Sorprendentemente, se observan efectos en ambos lados de la barrera sangre-cerebro, por lo cual éste es un medio de aplicacion ideal para enfermedades de almacenamiento lisosomal que afectan ambos, el cerebro y organos viscerales.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US76037806P | 2006-01-20 | 2006-01-20 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR059089A1 true AR059089A1 (es) | 2008-03-12 |
Family
ID=38288294
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP070100239A AR059089A1 (es) | 2006-01-20 | 2007-01-19 | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
ARP170102114A AR109170A2 (es) | 2006-01-20 | 2017-07-27 | Esfingomielinasa ácida para uso en la prevención o el tratamiento de la enfermedad de niemann-pick a o b en un paciente |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP170102114A AR109170A2 (es) | 2006-01-20 | 2017-07-27 | Esfingomielinasa ácida para uso en la prevención o el tratamiento de la enfermedad de niemann-pick a o b en un paciente |
Country Status (19)
Country | Link |
---|---|
US (4) | US8926967B2 (es) |
EP (3) | EP1984018B1 (es) |
JP (1) | JP5681345B2 (es) |
CN (1) | CN101431960A (es) |
AR (2) | AR059089A1 (es) |
BR (1) | BRPI0706699A2 (es) |
CA (2) | CA2966155A1 (es) |
DK (2) | DK2666476T3 (es) |
ES (2) | ES2716206T3 (es) |
HU (1) | HUE041840T2 (es) |
IL (2) | IL192740A (es) |
LT (1) | LT2666476T (es) |
MX (1) | MX349749B (es) |
PL (2) | PL2666476T3 (es) |
PT (2) | PT2666476T (es) |
RU (3) | RU2529830C2 (es) |
SI (2) | SI2666476T1 (es) |
TR (1) | TR201903855T4 (es) |
WO (1) | WO2007084737A2 (es) |
Families Citing this family (34)
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AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
ES2697502T3 (es) | 2006-02-09 | 2019-01-24 | Genzyme Corp | Administración intraventricular lenta |
AU2008254774B2 (en) | 2007-05-16 | 2013-11-14 | The Brigham And Women's Hospital, Inc. | Treatment of synucleinopathies |
CN105879047A (zh) * | 2008-12-16 | 2016-08-24 | 建新公司 | 寡糖-蛋白缀合物 |
LT3421603T (lt) | 2009-05-02 | 2022-01-10 | Genzyme Corporation | Genų terapija, skirta neurodegeneraciniams sutrikimams |
EP3998078A1 (en) | 2009-08-28 | 2022-05-18 | Icahn School of Medicine at Mount Sinai | Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency |
CA2796607C (en) | 2010-04-23 | 2019-09-10 | Synageva Biopharma Corp | Lysosomal storage disease enzyme |
JP6045493B2 (ja) * | 2010-06-25 | 2016-12-14 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | アリールスルファターゼaのcns送達の方法および組成物 |
ES2754234T3 (es) | 2010-06-25 | 2020-04-16 | Shire Human Genetic Therapies | Métodos y composiciones para la administración SNC de iduronato-2-sulfatasa |
EP3875107A1 (en) | 2010-06-25 | 2021-09-08 | Shire Human Genetic Therapies, Inc. | Pharmaceutical formulation comprising a replacement enzyme for a lysosomal enzyme for use in treating lysosomal storage disease intrathecally |
WO2011163647A2 (en) | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of heparan n-sulfatase |
PT3626257T (pt) * | 2010-06-25 | 2021-11-12 | Shire Human Genetic Therapies | Métodos e composições de arilsulfatase a para a administração no snc |
EP2588132A4 (en) * | 2010-06-25 | 2014-10-15 | Shire Human Genetic Therapies | METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS |
JP2012062312A (ja) * | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
AU2011314293B2 (en) | 2010-09-09 | 2015-06-04 | Alexion Pharmaceuticals, Inc. | Use of Lysosomal Acid Lipase for treating Lysosomal Acid Lipase Deficiency in patients |
US9387236B2 (en) * | 2011-06-10 | 2016-07-12 | Prothera Inc. | Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases |
WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
EP2793933B1 (en) | 2011-12-23 | 2019-04-17 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
EA039246B1 (ru) * | 2012-12-06 | 2021-12-22 | Шир Хьюман Дженетик Терапис, Инк. | Способ лечения когнитивных нарушений, связанных с синдромом хантера |
US10017581B2 (en) | 2013-02-20 | 2018-07-10 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
IL284102B2 (en) * | 2013-06-07 | 2023-04-01 | Genzyme Corp | A marker for disorders of acid sphingomyelinase and its uses |
EP3450557A1 (en) | 2013-12-25 | 2019-03-06 | JCR Pharmaceuticals Co., Ltd. | Novel anti-transferrin receptor antibody that passes through blood-brain barrier |
CA2938577A1 (en) | 2014-02-04 | 2015-08-13 | New York University | Progranulin (pgrn) and its derivatives for diagnosis and treatment of lysosomal storage diseases |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
JP6914193B2 (ja) | 2015-06-24 | 2021-08-04 | Jcrファーマ株式会社 | Bdnfを含む融合蛋白質 |
EA039366B1 (ru) | 2015-06-24 | 2022-01-19 | Джей Си Ар ФАРМАСЬЮТИКАЛЗ КО., ЛТД. | Антитело к рецептору трансферрина человека, проникающее через гематоэнцефалический барьер |
WO2017024137A1 (en) * | 2015-08-04 | 2017-02-09 | New York University | Progranulin (pgrn) fragments and derivatives for treatment or alleviation of lysosomal storage diseases |
RU2634113C1 (ru) * | 2016-11-18 | 2017-10-24 | Государственное бюджетное учреждение "Академия наук Республики Саха (Якутия)" (ГБУ АН РС(Я)) | Способ фиксации головного мозга ископаемого позднеплейстоценового млекопитающего |
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AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
AR059088A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una proteina para esclerosis lateral amiotrofica |
ES2697502T3 (es) | 2006-02-09 | 2019-01-24 | Genzyme Corp | Administración intraventricular lenta |
DK2158322T3 (en) * | 2007-06-06 | 2017-08-28 | Genzyme Corp | GENTERAPY AGAINST LYSOSOMAL STORAGE DISEASES |
-
2007
- 2007-01-19 AR ARP070100239A patent/AR059089A1/es not_active Application Discontinuation
- 2007-01-22 PT PT13173895T patent/PT2666476T/pt unknown
- 2007-01-22 PL PL13173895T patent/PL2666476T3/pl unknown
- 2007-01-22 PT PT77168581T patent/PT1984018E/pt unknown
- 2007-01-22 LT LTEP13173895.7T patent/LT2666476T/lt unknown
- 2007-01-22 WO PCT/US2007/001566 patent/WO2007084737A2/en active Application Filing
- 2007-01-22 ES ES13173895T patent/ES2716206T3/es active Active
- 2007-01-22 PL PL07716858T patent/PL1984018T3/pl unknown
- 2007-01-22 EP EP07716858.1A patent/EP1984018B1/en active Active
- 2007-01-22 CA CA2966155A patent/CA2966155A1/en not_active Abandoned
- 2007-01-22 CA CA2636991A patent/CA2636991C/en not_active Expired - Fee Related
- 2007-01-22 JP JP2008551442A patent/JP5681345B2/ja not_active Expired - Fee Related
- 2007-01-22 RU RU2008134120/15A patent/RU2529830C2/ru not_active IP Right Cessation
- 2007-01-22 CN CNA2007800069255A patent/CN101431960A/zh active Pending
- 2007-01-22 MX MX2013012372A patent/MX349749B/es unknown
- 2007-01-22 TR TR2019/03855T patent/TR201903855T4/tr unknown
- 2007-01-22 SI SI200732098T patent/SI2666476T1/sl unknown
- 2007-01-22 EP EP13173895.7A patent/EP2666476B1/en active Active
- 2007-01-22 DK DK13173895.7T patent/DK2666476T3/en active
- 2007-01-22 HU HUE13173895A patent/HUE041840T2/hu unknown
- 2007-01-22 BR BRPI0706699-6A patent/BRPI0706699A2/pt not_active Application Discontinuation
- 2007-01-22 EP EP18212320.8A patent/EP3517124A1/en not_active Withdrawn
- 2007-01-22 ES ES07716858.1T patent/ES2526710T3/es active Active
- 2007-01-22 SI SI200731529T patent/SI1984018T1/sl unknown
- 2007-01-22 DK DK07716858.1T patent/DK1984018T3/da active
-
2008
- 2008-07-10 IL IL192740A patent/IL192740A/en not_active IP Right Cessation
- 2008-07-18 US US12/175,610 patent/US8926967B2/en active Active
-
2014
- 2014-07-16 RU RU2014129319A patent/RU2665381C2/ru not_active IP Right Cessation
- 2014-12-03 US US14/559,851 patent/US10080783B2/en active Active
-
2017
- 2017-03-05 IL IL250937A patent/IL250937A0/en unknown
- 2017-07-27 AR ARP170102114A patent/AR109170A2/es unknown
-
2018
- 2018-08-15 RU RU2018129747A patent/RU2018129747A/ru not_active Application Discontinuation
- 2018-08-23 US US16/111,009 patent/US20190083582A1/en not_active Abandoned
-
2020
- 2020-12-29 US US17/137,060 patent/US20210228692A1/en not_active Abandoned
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