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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- Prior art keywords
- enzyme
- lysosomal storage
- hours
- storage disease
- patient
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- 102000004190 Enzymes Human genes 0.000 title claims 18
- 108090000790 Enzymes Proteins 0.000 title claims 18
- 201000010099 disease Diseases 0.000 title claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title claims 3
- 238000007914 intraventricular administration Methods 0.000 title claims 2
- 238000009825 accumulation Methods 0.000 title 1
- 230000002132 lysosomal effect Effects 0.000 title 1
- 238000000034 method Methods 0.000 claims 13
- 208000015439 Lysosomal storage disease Diseases 0.000 claims 7
- 238000011282 treatment Methods 0.000 claims 3
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 2
- 210000004556 brain Anatomy 0.000 claims 2
- 238000011321 prophylaxis Methods 0.000 claims 2
- 208000011580 syndromic disease Diseases 0.000 claims 2
- 102000003779 Dipeptidyl-peptidases and tripeptidyl-peptidases Human genes 0.000 claims 1
- 108090000194 Dipeptidyl-peptidases and tripeptidyl-peptidases Proteins 0.000 claims 1
- 208000015872 Gaucher disease Diseases 0.000 claims 1
- 102000004547 Glucosylceramidase Human genes 0.000 claims 1
- 108010017544 Glucosylceramidase Proteins 0.000 claims 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 102100029199 Iduronate 2-sulfatase Human genes 0.000 claims 1
- 101710096421 Iduronate 2-sulfatase Proteins 0.000 claims 1
- 102000004627 Iduronidase Human genes 0.000 claims 1
- 108010003381 Iduronidase Proteins 0.000 claims 1
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 1
- 108010028144 alpha-Glucosidases Proteins 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- 210000004055 fourth ventricle Anatomy 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 210000003140 lateral ventricle Anatomy 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 210000004072 lung Anatomy 0.000 claims 1
- 238000012544 monitoring process Methods 0.000 claims 1
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 210000000952 spleen Anatomy 0.000 claims 1
Classifications
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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
-
- 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)
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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)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- 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)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Dermatology (AREA)
- Urology & Nephrology (AREA)
- Pulmonology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Hospice & Palliative Care (AREA)
- Diabetes (AREA)
- Psychiatry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Infusion, Injection, And Reservoir Apparatuses (AREA)
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) | EP3517124A1 (ru) |
| JP (1) | JP5681345B2 (ru) |
| CN (1) | CN101431960A (ru) |
| AR (2) | AR059089A1 (ru) |
| BR (1) | BRPI0706699A2 (ru) |
| CA (2) | CA2636991C (ru) |
| DK (2) | DK1984018T3 (ru) |
| ES (2) | ES2716206T3 (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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| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| CA2641359C (en) * | 2006-02-09 | 2022-10-04 | Genzyme Corporation | Slow intraventricular delivery |
| PT2154969E (pt) | 2007-05-16 | 2016-02-17 | Brigham & Womens Hospital | Tratamento das sinucleinopatias |
| ES2735023T3 (es) | 2008-12-16 | 2019-12-13 | Genzyme Corp | Intermedios sintéticos para preparar conjugados de oligosacáridos-proteínas |
| CN102459611B (zh) | 2009-05-02 | 2016-11-09 | 建新公司 | 神经退行性疾病的基因治疗 |
| FI3998078T3 (fi) * | 2009-08-28 | 2025-12-04 | Icahn School Med Mount Sinai | Annoksensuurentamisentsyymikorvaushoito happaman sfingomyelinaasin puutoksen hoitamista varten |
| MX338426B (es) | 2010-04-23 | 2016-04-15 | Synageva Biopharma Corp | Enzima de enfermedad del almacenamiento lisosomico. |
| RU2012154576A (ru) * | 2010-06-25 | 2014-07-27 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
| DK2588130T3 (en) | 2010-06-25 | 2016-10-24 | Shire Human Genetic Therapies | Cns delivery of therapeutic agents |
| KR102318997B1 (ko) * | 2010-06-25 | 2021-10-29 | 샤이어 휴먼 지네틱 테라피즈 인크. | 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들 |
| EP2588132A4 (en) * | 2010-06-25 | 2014-10-15 | Shire Human Genetic Therapies | METHOD AND COMPOSITIONS FOR DELIVERING BETA GALACTOCEREBROSIDASE INTO THE CNS |
| NZ605863A (en) | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of iduronate-2-sulfatase |
| HRP20211520T1 (hr) | 2010-06-25 | 2021-12-24 | Shire Human Genetic Therapies, Inc. | Postupci i pripravci za dostavu iduronat-2-sulfataze u središnji živčani sustav |
| JP2012062312A (ja) * | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
| PL2613798T5 (pl) | 2010-09-09 | 2018-06-29 | Synageva Biopharma Corp. | Zastosowanie lizosomalnej kwaśnej lipazy do leczenia niedoboru lizosomalnej kwaśnej lipazy u pacjentów |
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| EP2793922B1 (en) | 2011-12-23 | 2019-10-30 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| AU2012358223B2 (en) * | 2011-12-23 | 2018-01-18 | Takeda Pharmaceutical Company Limited | 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 |
| CA2901978A1 (en) | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Methods and compositions for treatment of pompe disease |
| IL284102B2 (en) * | 2013-06-07 | 2023-04-01 | Genzyme Corp | Marker for acid sphingomyelinase disorders and uses thereof |
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| 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 |
| CN110100000B (zh) | 2016-12-26 | 2023-06-20 | Jcr制药股份有限公司 | 含有bdnf的融合蛋白 |
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