RU2018129747A - Внутрижелудочковая доставка ферментов при лизосомных болезнях накопления - Google Patents
Внутрижелудочковая доставка ферментов при лизосомных болезнях накопления Download PDFInfo
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- RU2018129747A RU2018129747A RU2018129747A RU2018129747A RU2018129747A RU 2018129747 A RU2018129747 A RU 2018129747A RU 2018129747 A RU2018129747 A RU 2018129747A RU 2018129747 A RU2018129747 A RU 2018129747A RU 2018129747 A RU2018129747 A RU 2018129747A
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- lysosomal storage
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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
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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)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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)
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- 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)
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- 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)
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- 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
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- 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)
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- 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)
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
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- Medicinal Chemistry (AREA)
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- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Dermatology (AREA)
- Obesity (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Urology & Nephrology (AREA)
- Pulmonology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Infusion, Injection, And Reservoir Apparatuses (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
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Claims (13)
1. Способ профилактики или лечения лизосомной болезни накопления, которая вызвана дефицитом фермента у пациента, включающий внутрижелудочковое введение фермента в головной мозг пациента, при этом введение одной дозы фермента занимает более трех часов.
2. Способ по п.1, где введение одной дозы фермента занимает более четырех часов, более пяти часов, более шести часов, более семи часов или более восьми часов.
3. Способ по п.1, где указанная профилактика или лечение включает введение фермента в боковые желудочки и/или в четвертый желудочек головного мозга.
4. Способ по п.1, где введенного пациенту количества фермента достаточно для снижения уровня фермента в печени, легких, селезенки или почках пациента.
5. Способ по п.1, где профилактика или лечение включает мониторинг уровней сфингомиелина у пациента и введение дополнительного фермента в ответ на зарегистрированные уровни фермента.
6. Способ по п.1, где фермент вводят с помощью постоянного катетера.
7. Способ по п.1, где введение включает множественные инфузии.
8. Способ по любому из пп.1-7, где лизосомная болезнь накопления является синдромом мукополисахаридоза типа I, и фермент является альфа-L-идуронидазой.
9. Способ по любому из пп.1-7, где лизосомная болезнь накопления является синдромом мукополисахаридоза типа II, и фермент является идуронат-2-сульфатазой.
10. Способ по любому из пп.1-7, где лизосомная болезнь накопления является болезнью Гоше, и фермент является глюкоцереброзидазой.
11. Способ по любому из пп.1-7, где лизосомная болезнь накопления является болезнью Помпе, и фермент является альфа-глюкозидазой.
12. Способ по любому из пп.1-7, где лизосомная болезнь накопления является классической поздней инфантильной болезнью Баттена (CLN2), и фермент является трипептидилпептидазой.
13. Способ по любому из пп.1-7, где лизосомная болезнь накопления является одним из заболеваний, указанных в таблице 1, и фермент является соответствующим дефектным ферментом, указанным в таблице 1.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US76037806P | 2006-01-20 | 2006-01-20 | |
| US60/760,378 | 2006-01-20 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2014129319A Division RU2665381C2 (ru) | 2006-01-20 | 2014-07-16 | Внутрижелудочковая доставка ферментов при лизосомных болезнях накопления |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2018129747A true RU2018129747A (ru) | 2020-02-19 |
Family
ID=38288294
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2008134120/15A RU2529830C2 (ru) | 2006-01-20 | 2007-01-22 | Внутрижелудочковая доставка ферментов при лизосомных болезнях накопления |
| RU2014129319A RU2665381C2 (ru) | 2006-01-20 | 2014-07-16 | Внутрижелудочковая доставка ферментов при лизосомных болезнях накопления |
| RU2018129747A RU2018129747A (ru) | 2006-01-20 | 2018-08-15 | Внутрижелудочковая доставка ферментов при лизосомных болезнях накопления |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2008134120/15A RU2529830C2 (ru) | 2006-01-20 | 2007-01-22 | Внутрижелудочковая доставка ферментов при лизосомных болезнях накопления |
| RU2014129319A RU2665381C2 (ru) | 2006-01-20 | 2014-07-16 | Внутрижелудочковая доставка ферментов при лизосомных болезнях накопления |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US8926967B2 (ru) |
| EP (3) | EP2666476B1 (ru) |
| JP (1) | JP5681345B2 (ru) |
| CN (1) | CN101431960A (ru) |
| AR (2) | AR059089A1 (ru) |
| BR (1) | BRPI0706699A2 (ru) |
| CA (2) | CA2966155A1 (ru) |
| DK (2) | DK2666476T3 (ru) |
| ES (2) | ES2526710T3 (ru) |
| HU (1) | HUE041840T2 (ru) |
| IL (2) | IL192740A (ru) |
| LT (1) | LT2666476T (ru) |
| MX (1) | MX349749B (ru) |
| PL (2) | PL2666476T3 (ru) |
| PT (2) | PT1984018E (ru) |
| RU (3) | RU2529830C2 (ru) |
| SI (2) | SI2666476T1 (ru) |
| TR (1) | TR201903855T4 (ru) |
| WO (1) | WO2007084737A2 (ru) |
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