CY1114815T1 - Χορηγηση θεραπευτικων χημικων ενωσεων στον εγκεφαλο και σε αλλους ιστους - Google Patents

Χορηγηση θεραπευτικων χημικων ενωσεων στον εγκεφαλο και σε αλλους ιστους

Info

Publication number
CY1114815T1
CY1114815T1 CY20141100019T CY141100019T CY1114815T1 CY 1114815 T1 CY1114815 T1 CY 1114815T1 CY 20141100019 T CY20141100019 T CY 20141100019T CY 141100019 T CY141100019 T CY 141100019T CY 1114815 T1 CY1114815 T1 CY 1114815T1
Authority
CY
Cyprus
Prior art keywords
administration
mps
brain
tissue
iduronidase
Prior art date
Application number
CY20141100019T
Other languages
English (en)
Inventor
Emil D Kakkis
Original Assignee
Biomarin Pharmaceutical Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=34217412&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CY1114815(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Biomarin Pharmaceutical Inc. filed Critical Biomarin Pharmaceutical Inc.
Publication of CY1114815T1 publication Critical patent/CY1114815T1/el

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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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)
    • C12Y301/06008—Cerebroside-sulfatase (3.1.6.8)
    • 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)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Η παρούσα εφεύρεση σχετίζεται με την ενδορραχιαία χορήγηση (IT) του ανασυνδυασμένου ενζύμου για την θεραπεία των διαταραχών της λυσοσωματικής αποθήκευσης. Σε μία παραδειγματική εφαρμογή, η ενδορραχιαία χορήγηση των ενέσεων της ανθρώπινης α-Λ-ιδουρονιδάσης (rhIDU) σε μολυσμένα ζώα με MPS I είχε ως αποτέλεσμα την σημαντική πρόσληψη του ενζύμου, την σημαντική δραστικότητα της rh-ιδουρονιδάσης στον εγκέφαλο και τους μήνιγγες και μία μείωση στην αποθήκευση της γλυκοσαμινογλυκάνης (GAG) στα κύτταρα των υποκειμένων με MPS I σε σχέση με αυτή των φυσιολογικών υποκειμένων. Η ενδορραχιαία χορήγηση αποδείχθηκε περισσότερο αποτελεσματική από την ενδοφλέβια θεραπεία ως προς την ανακούφιση των συμπτωμάτων της MPS I, καταδεικνύοντας την ως μία χρήσιμη μέθοδο για την θεραπεία των διαταραχών στην λυσοσωματική αποθήκευση.
CY20141100019T 2003-08-29 2014-01-09 Χορηγηση θεραπευτικων χημικων ενωσεων στον εγκεφαλο και σε αλλους ιστους CY1114815T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US10/651,493 US7442372B2 (en) 2003-08-29 2003-08-29 Delivery of therapeutic compounds to the brain and other tissues
EP04782579.9A EP1673104B2 (en) 2003-08-29 2004-08-30 Delivery of therapeutic compounds to the brain and other tissues

Publications (1)

Publication Number Publication Date
CY1114815T1 true CY1114815T1 (el) 2016-12-14

Family

ID=34217412

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20141100019T CY1114815T1 (el) 2003-08-29 2014-01-09 Χορηγηση θεραπευτικων χημικων ενωσεων στον εγκεφαλο και σε αλλους ιστους

Country Status (12)

Country Link
US (5) US7442372B2 (el)
EP (1) EP1673104B2 (el)
JP (1) JP5819574B2 (el)
AU (1) AU2004268645B2 (el)
CA (1) CA2537238C (el)
CY (1) CY1114815T1 (el)
DK (1) DK1673104T4 (el)
ES (1) ES2441612T5 (el)
PL (1) PL1673104T5 (el)
PT (1) PT1673104E (el)
SI (1) SI1673104T2 (el)
WO (1) WO2005021064A2 (el)

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