EA201792099A1 - Лечение когнитивных нарушений при синдроме хантера путем интратекальной доставки идуронат-2-сульфатазы - Google Patents

Лечение когнитивных нарушений при синдроме хантера путем интратекальной доставки идуронат-2-сульфатазы

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Publication number
EA201792099A1
EA201792099A1 EA201792099A EA201792099A EA201792099A1 EA 201792099 A1 EA201792099 A1 EA 201792099A1 EA 201792099 A EA201792099 A EA 201792099A EA 201792099 A EA201792099 A EA 201792099A EA 201792099 A1 EA201792099 A1 EA 201792099A1
Authority
EA
Eurasian Patent Office
Prior art keywords
treatment
sulfatase
syndrome
iuronat
hanter
Prior art date
Application number
EA201792099A
Other languages
English (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.
Publication of EA201792099A1 publication Critical patent/EA201792099A1/ru

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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)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Dermatology (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (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.
EA201792099A 2011-12-23 2012-12-21 Лечение когнитивных нарушений при синдроме хантера путем интратекальной доставки идуронат-2-сульфатазы EA201792099A1 (ru)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161580027P 2011-12-23 2011-12-23
US201261590804P 2012-01-25 2012-01-25
US201261590797P 2012-01-25 2012-01-25
US201261609173P 2012-03-09 2012-03-09
US201261734365P 2012-12-06 2012-12-06

Publications (1)

Publication Number Publication Date
EA201792099A1 true EA201792099A1 (ru) 2018-05-31

Family

ID=48669720

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201792099A EA201792099A1 (ru) 2011-12-23 2012-12-21 Лечение когнитивных нарушений при синдроме хантера путем интратекальной доставки идуронат-2-сульфатазы

Country Status (9)

Country Link
US (3) US9682129B2 (ru)
EP (1) EP2793933B1 (ru)
AU (1) AU2012358223B2 (ru)
BR (1) BR112014015352A8 (ru)
CA (1) CA2859988A1 (ru)
EA (1) EA201792099A1 (ru)
ES (1) ES2728447T3 (ru)
MX (2) MX367395B (ru)
WO (1) WO2013096912A2 (ru)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2728447T3 (es) * 2011-12-23 2019-10-24 Shire Human Genetic Therapies Tratamiento del déficit cognitivo del síndrome de Hunter para la administración intratecal de iduronato-2-sulfatasa
KR20170004814A (ko) * 2015-07-02 2017-01-11 주식회사 녹십자 헌터증후군 치료제
PE20181329A1 (es) 2015-12-30 2018-08-20 Green Cross Corp Metodos y composiciones para el tratamiento del sindrome de hunter
EP3692070A1 (en) 2017-10-02 2020-08-12 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
WO2004055157A2 (en) 2002-08-13 2004-07-01 Whitley Chester B Methods of using vectors to treat metabolic disorders
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
US7722865B2 (en) 2008-01-18 2010-05-25 Biomarin Pharmaceutical Inc. Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
IL291554B2 (en) * 2010-06-25 2024-02-01 Shire Human Genetic Therapies Administering therapeutic agents to the central nervous system
JP6045492B2 (ja) 2010-06-25 2016-12-14 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物
DK3103469T3 (en) * 2010-06-25 2021-02-22 Shire Human Genetic Therapies Indgivelse af terapeutiske midler til centralnervesystemet
JP6073783B2 (ja) 2010-06-25 2017-02-01 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物
ES2728447T3 (es) 2011-12-23 2019-10-24 Shire Human Genetic Therapies Tratamiento del déficit cognitivo del síndrome de Hunter para la administración intratecal de iduronato-2-sulfatasa

Also Published As

Publication number Publication date
AU2012358223B2 (en) 2018-01-18
WO2013096912A2 (en) 2013-06-27
EP2793933B1 (en) 2019-04-17
US9682129B2 (en) 2017-06-20
US20180071371A1 (en) 2018-03-15
EP2793933A2 (en) 2014-10-29
MX2014007642A (es) 2015-03-19
MX367395B (es) 2019-08-20
MX2019009846A (es) 2019-10-14
BR112014015352A2 (pt) 2017-06-13
US10568941B2 (en) 2020-02-25
BR112014015352A8 (pt) 2017-06-13
EP2793933A4 (en) 2016-06-08
ES2728447T3 (es) 2019-10-24
US20150328289A1 (en) 2015-11-19
AU2012358223A1 (en) 2014-07-03
WO2013096912A3 (en) 2015-06-18
US11357832B2 (en) 2022-06-14
CA2859988A1 (en) 2013-06-27
US20200376093A1 (en) 2020-12-03

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