MX349749B - Suministro enzimatico intraventricular para enfermedades de almacenamiento lisosomico. - Google Patents
Suministro enzimatico intraventricular para enfermedades de almacenamiento lisosomico.Info
- Publication number
- MX349749B MX349749B MX2013012372A MX2013012372A MX349749B MX 349749 B MX349749 B MX 349749B MX 2013012372 A MX2013012372 A MX 2013012372A MX 2013012372 A MX2013012372 A MX 2013012372A MX 349749 B MX349749 B MX 349749B
- Authority
- MX
- Mexico
- Prior art keywords
- lysosomal storage
- storage diseases
- enzyme delivery
- intraventricular
- delivery
- Prior art date
Links
- 208000015439 Lysosomal storage disease Diseases 0.000 title abstract 3
- 102000004190 Enzymes Human genes 0.000 title abstract 2
- 108090000790 Enzymes Proteins 0.000 title abstract 2
- 238000007914 intraventricular administration Methods 0.000 title abstract 2
- 230000000694 effects Effects 0.000 abstract 2
- 230000008499 blood brain barrier function Effects 0.000 abstract 1
- 210000001218 blood-brain barrier Anatomy 0.000 abstract 1
- 210000004556 brain Anatomy 0.000 abstract 1
- 230000002950 deficient Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 210000001835 viscera Anatomy 0.000 abstract 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)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
- Hospice & Palliative Care (AREA)
- Urology & Nephrology (AREA)
- Psychiatry (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Pulmonology (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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- General Engineering & Computer Science (AREA)
- Infusion, Injection, And Reservoir Apparatuses (AREA)
Abstract
La presente invención se refiere a las enfermedades de almacenamiento lisosomal pueden ser tratadas de manera exitosa utilizando la administración intraventricular de la enzima, misma que es deficiente etiológicamente en la enfermedad. La administración puede realizarse lentamente para lograr el máximo efecto. Sorprendentemente, los efectos se ven en ambos lados de la barrera sanguíneo-cerebral, haciendo de esto un medio de administración ideal para las enfermedades de almacenamiento lisosomal que afectan tano al cerebro como a los órganos viscerales.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US76037806P | 2006-01-20 | 2006-01-20 | |
| PCT/US2007/001566 WO2007084737A2 (en) | 2006-01-20 | 2007-01-22 | Intraventricular enzyme delivery for lysosomal storage diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX349749B true MX349749B (es) | 2017-08-10 |
Family
ID=38288294
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2013012372A MX349749B (es) | 2006-01-20 | 2007-01-22 | Suministro enzimatico intraventricular para enfermedades de almacenamiento lisosomico. |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US8926967B2 (es) |
| EP (3) | EP3517124A1 (es) |
| JP (1) | JP5681345B2 (es) |
| CN (1) | CN101431960A (es) |
| AR (2) | AR059089A1 (es) |
| BR (1) | BRPI0706699A2 (es) |
| CA (2) | CA2636991C (es) |
| DK (2) | DK1984018T3 (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) | PT1984018E (es) |
| RU (3) | RU2529830C2 (es) |
| SI (2) | SI1984018T1 (es) |
| TR (1) | TR201903855T4 (es) |
| WO (1) | WO2007084737A2 (es) |
Families Citing this family (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| MX392030B (es) | 2006-02-09 | 2025-03-21 | Genzyme Corp | Agentes y usos de los mismos para prevenir y/o tratar enfermedades neurológicas |
| ES2559859T3 (es) | 2007-05-16 | 2016-02-16 | The Brigham And Women's Hospital, Inc. | Tratamiento de sinucleinopatías |
| SI2889043T1 (sl) | 2008-12-16 | 2019-08-30 | Genzyme Corporation | Sintetične vmesne spojine za pripravo konjugatov oligosaharid-protein |
| SG10202109219SA (en) | 2009-05-02 | 2021-10-28 | Genzyme Corp | Gene therapy for neurodegenerative disorders |
| CN102596232A (zh) | 2009-08-28 | 2012-07-18 | 西奈山医学院 | 用于治疗酸性鞘磷脂酶缺乏的剂量递增酶替代疗法 |
| EP2561069B1 (en) | 2010-04-23 | 2017-03-08 | Alexion Pharmaceuticals, Inc. | Lysosomal storage disease enzyme |
| RU2012154576A (ru) * | 2010-06-25 | 2014-07-27 | Шир Хьюман Дженетик Терапис, Инк. | Способы и композиции для доставки к цнс гепаран-n-сульфатазы |
| UA115648C2 (uk) * | 2010-06-25 | 2017-12-11 | Шае Хюмен Дженетік Терапіс, Інк. | Доставка терапевтичних агентів до цнс |
| PL2593131T3 (pl) | 2010-06-25 | 2020-01-31 | Shire Human Genetic Therapies, Inc. | Sposoby i kompozycje do dostarczania sulfatazy-2-iduronianiu do oun |
| BR112012033216B1 (pt) | 2010-06-25 | 2022-10-11 | Shire Human Genetic Therapies, Inc | Uso de uma formulação estável compreendendo uma proteína arilsulfatase a (asa) e formulação estável para administração intratecal |
| CN104857504A (zh) | 2010-06-25 | 2015-08-26 | 夏尔人类遗传性治疗公司 | 芳基硫酸酯酶a cns递送的方法和组合物 |
| WO2011163651A2 (en) * | 2010-06-25 | 2011-12-29 | Shire Human Genetic Therapies, Inc. | METHODS AND COMPOSITIONS FOR CNS DELIVERY OF β-GALACTOCEREBROSIDASE |
| JP2012062312A (ja) * | 2010-08-19 | 2012-03-29 | Yoshikatsu Eto | ハンター症候群の治療剤 |
| NZ715014A (en) * | 2010-09-09 | 2018-10-26 | Synageva Biopharma Corp | 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 |
| US9682129B2 (en) | 2011-12-23 | 2017-06-20 | Shire Human Genetic Therapies, Inc. | Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase |
| WO2013096899A2 (en) | 2011-12-23 | 2013-06-27 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| 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 |
| IL298675B2 (en) * | 2013-06-07 | 2025-12-01 | Genzyme Corp | Marker for acid sphingomyelinase disorders and uses thereof |
| WO2015098989A1 (ja) | 2013-12-25 | 2015-07-02 | Jcrファーマ株式会社 | 血液脳関門を通過する新規抗トランスフェリン受容体抗体 |
| CN106573153A (zh) | 2014-02-04 | 2017-04-19 | 纽约大学 | 用于诊断和治疗溶酶体贮积病的颗粒蛋白前体(pgrn)及其衍生物 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CA2990565A1 (en) | 2015-06-24 | 2016-12-29 | Jcr Pharmaceuticals Co., Ltd. | Fusion protein containing bdnf and anti-human transferrin receptor antibody |
| TWI769982B (zh) | 2015-06-24 | 2022-07-11 | 日商Jcr製藥股份有限公司 | 通過血腦障壁之抗人類運鐵蛋白受體抗體 |
| US11390656B2 (en) * | 2015-08-04 | 2022-07-19 | 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 |
| CN109983031B (zh) | 2016-12-26 | 2022-11-25 | Jcr制药股份有限公司 | 通过血脑屏障的新的抗人转铁蛋白受体抗体 |
| KR102573622B1 (ko) | 2016-12-26 | 2023-08-31 | 제이씨알 파마 가부시키가이샤 | Bdnf를 포함하는 융합 단백질 |
| MA50746A (fr) | 2017-10-02 | 2020-08-12 | Denali Therapeutics Inc | Protéines de fusion comprenant des enzymes d'enzymothérapie substitutive |
| UY38238A (es) * | 2018-05-25 | 2019-12-31 | Genzyme Corp | Composiciones farmacéuticas para el tratamiento de la deficiencia de esfingomielinasa ácida |
| WO2020157248A1 (en) | 2019-02-01 | 2020-08-06 | Oxyrane Uk Ltd | Glucocerebrosidase polypeptides |
| RU2723187C1 (ru) * | 2019-10-04 | 2020-06-09 | федеральное государственное автономное образовательное учреждение высшего образования "Казанский (Приволжский) федеральный университет" (ФГАОУ ВО КФУ) | Генетическая кассета, содержащая кодон-оптимизированные нуклеотидные последовательности генов HEXA и HEXВ, и фармацевтическая композиция для лечения болезни Тея-Сакса |
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| PT3252161T (pt) * | 2007-06-06 | 2022-02-01 | Genzyme Corp | Terapia genética para doenças do armazenamento lisossomal |
-
2007
- 2007-01-19 AR ARP070100239A patent/AR059089A1/es not_active Application Discontinuation
- 2007-01-22 DK DK07716858.1T patent/DK1984018T3/da active
- 2007-01-22 SI SI200731529T patent/SI1984018T1/sl unknown
- 2007-01-22 JP JP2008551442A patent/JP5681345B2/ja not_active Expired - Fee Related
- 2007-01-22 BR BRPI0706699-6A patent/BRPI0706699A2/pt not_active Application Discontinuation
- 2007-01-22 HU HUE13173895A patent/HUE041840T2/hu unknown
- 2007-01-22 MX MX2013012372A patent/MX349749B/es unknown
- 2007-01-22 TR TR2019/03855T patent/TR201903855T4/tr unknown
- 2007-01-22 PL PL13173895T patent/PL2666476T3/pl unknown
- 2007-01-22 WO PCT/US2007/001566 patent/WO2007084737A2/en not_active Ceased
- 2007-01-22 LT LTEP13173895.7T patent/LT2666476T/lt unknown
- 2007-01-22 DK DK13173895.7T patent/DK2666476T3/en active
- 2007-01-22 PT PT77168581T patent/PT1984018E/pt unknown
- 2007-01-22 PL PL07716858T patent/PL1984018T3/pl unknown
- 2007-01-22 SI SI200732098T patent/SI2666476T1/sl unknown
- 2007-01-22 RU RU2008134120/15A patent/RU2529830C2/ru not_active IP Right Cessation
- 2007-01-22 PT PT13173895T patent/PT2666476T/pt unknown
- 2007-01-22 CA CA2636991A patent/CA2636991C/en not_active Expired - Fee Related
- 2007-01-22 EP EP18212320.8A patent/EP3517124A1/en not_active Withdrawn
- 2007-01-22 CN CNA2007800069255A patent/CN101431960A/zh active Pending
- 2007-01-22 ES ES13173895T patent/ES2716206T3/es active Active
- 2007-01-22 ES ES07716858.1T patent/ES2526710T3/es active Active
- 2007-01-22 EP EP07716858.1A patent/EP1984018B1/en not_active Not-in-force
- 2007-01-22 CA CA2966155A patent/CA2966155A1/en not_active Abandoned
- 2007-01-22 EP EP13173895.7A patent/EP2666476B1/en active Active
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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
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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
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2017
- 2017-03-05 IL IL250937A patent/IL250937A0/en unknown
- 2017-07-27 AR ARP170102114A patent/AR109170A2/es unknown
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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
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2020
- 2020-12-29 US US17/137,060 patent/US20210228692A1/en not_active Abandoned
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