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EA201491047A1 - TREATMENT OF COGNITIVE DISTURBANCES IN HANTER'S SYNDROME THROUGH INTRATECAL DELIVERY OF IURONAT-2-SULFATASE - Google Patents

TREATMENT OF COGNITIVE DISTURBANCES IN HANTER'S SYNDROME THROUGH INTRATECAL DELIVERY OF IURONAT-2-SULFATASE

Info

Publication number
EA201491047A1
EA201491047A1 EA201491047A EA201491047A EA201491047A1 EA 201491047 A1 EA201491047 A1 EA 201491047A1 EA 201491047 A EA201491047 A EA 201491047A EA 201491047 A EA201491047 A EA 201491047A EA 201491047 A1 EA201491047 A1 EA 201491047A1
Authority
EA
Eurasian Patent Office
Prior art keywords
treatment
sulfatase
syndrome
iuronat
hanter
Prior art date
Application number
EA201491047A
Other languages
Russian (ru)
Other versions
EA039246B1 (en
Inventor
Энн Барбиер
Томас Макколи
Чарльз В. Ричард III
Original Assignee
Шир Хьюман Дженетик Терапис, Инк.
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
Application filed by Шир Хьюман Дженетик Терапис, Инк. filed Critical Шир Хьюман Дженетик Терапис, Инк.
Priority claimed from PCT/US2012/071495 external-priority patent/WO2013096912A2/en
Publication of EA201491047A1 publication Critical patent/EA201491047A1/en
Publication of EA039246B1 publication Critical patent/EA039246B1/en

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)
    • 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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)

Landscapes

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

Abstract

Настоящее изобретение обеспечивает способ лечения когнитивных нарушений при синдроме Хантера. В числе прочего, настоящее изобретение обеспечивает способ, включающий этап интратекального введения субъекту, нуждающемуся в лечении, рекомбинантного фермента идуронат-2-сульфатазы (I2S) в терапевтически эффективной дозе и с интервалом введения в течение периода лечения, достаточным для улучшения, стабилизации или ослабления снижения одной или более из когнитивных, адаптивных, моторных и/или управляющих функций по сравнению с контролем.The present invention provides a method of treating cognitive impairment in Hunter syndrome. Among other things, the present invention provides a method comprising the step of intrathecal administration to a subject in need of treatment of a recombinant enzyme iduronate-2-sulfatase (I2S) in a therapeutically effective dose and with an administration interval during the treatment period sufficient to improve, stabilize or attenuate the decrease one or more of cognitive, adaptive, motor and / or control functions compared to control.

EA201491047A 2012-12-06 2012-12-21 Method of treating cognitive impairments associated with hunter syndrome EA039246B1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261734365P 2012-12-06 2012-12-06
PCT/US2012/071495 WO2013096912A2 (en) 2011-12-23 2012-12-21 Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase

Publications (2)

Publication Number Publication Date
EA201491047A1 true EA201491047A1 (en) 2015-02-27
EA039246B1 EA039246B1 (en) 2021-12-22

Family

ID=80632884

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201491047A EA039246B1 (en) 2012-12-06 2012-12-21 Method of treating cognitive impairments associated with hunter syndrome

Country Status (1)

Country Link
EA (1) EA039246B1 (en)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004055157A2 (en) * 2002-08-13 2004-07-01 Whitley Chester B Methods of using vectors to treat metabolic disorders
AR059089A1 (en) * 2006-01-20 2008-03-12 Genzyme Corp INTRAVENTRICULAR ADMINISTRATION OF AN ENZYME FOR LISOSOMAL STORAGE DISEASES
US7722865B2 (en) * 2008-01-18 2010-05-25 Biomarin Pharmaceutical Inc. Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
ES2754234T3 (en) * 2010-06-25 2020-04-16 Shire Human Genetic Therapies Methods and compositions for CNS administration of iduronate-2-sulfatase

Also Published As

Publication number Publication date
EA039246B1 (en) 2021-12-22

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