MX367395B - Iduronato-2-sulfatasa para usarse en el tratamiento del deterioro cognitivo del síndrome de hunter mediante suministro intratecal. - Google Patents

Iduronato-2-sulfatasa para usarse en el tratamiento del deterioro cognitivo del síndrome de hunter mediante suministro intratecal.

Info

Publication number
MX367395B
MX367395B MX2014007642A MX2014007642A MX367395B MX 367395 B MX367395 B MX 367395B MX 2014007642 A MX2014007642 A MX 2014007642A MX 2014007642 A MX2014007642 A MX 2014007642A MX 367395 B MX367395 B MX 367395B
Authority
MX
Mexico
Prior art keywords
treatment
sulfatase
cognitive impairment
iduronate
hunter syndrome
Prior art date
Application number
MX2014007642A
Other languages
English (en)
Other versions
MX2014007642A (es
Inventor
Thomas Mccauley
Ann Barbier
Charles W Richard Iii
Original Assignee
Shire Human Genetic Therapies
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 Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of MX2014007642A publication Critical patent/MX2014007642A/es
Publication of MX367395B publication Critical patent/MX367395B/es

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)
  • 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)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Dermatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente invención se refiere a una enzima iduronato-2-sulfatasa (I2S) recombinante para usarse en el tratamiento de Síndrome de Hunter en un paciente humano, en donde la enzima I2S recombinante está adaptada para ser administrable intratecalmente a un sujeto en necesidad de tratamiento a una dosis terapéuticamente efectiva de 10 mg o 30 mg y un intervalo de administración mensual durante un período de tratamiento suficiente para (i) mejorar, estabilizar o reducir la disminución de una o más funciones cognitivas, adaptativas, motoras, y/o ejecutivas en relación con un control, o (ii) disminuir el nivel de glicosaminoglicanos (GAG) en el líquido cefalorraquídeo (LCR) en relación con un control.
MX2014007642A 2011-12-23 2012-12-21 Iduronato-2-sulfatasa para usarse en el tratamiento del deterioro cognitivo del síndrome de hunter mediante suministro intratecal. MX367395B (es)

Applications Claiming Priority (6)

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
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
MX2014007642A MX2014007642A (es) 2015-03-19
MX367395B true MX367395B (es) 2019-08-20

Family

ID=48669720

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2014007642A MX367395B (es) 2011-12-23 2012-12-21 Iduronato-2-sulfatasa para usarse en el tratamiento del deterioro cognitivo del síndrome de hunter mediante suministro intratecal.
MX2019009846A MX2019009846A (es) 2011-12-23 2014-06-20 Iduronato-2-sulfatasa para usarse en el tratamiento del deterioro cognitivo del sindrome de hunter mediante suministro intratecal.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2019009846A MX2019009846A (es) 2011-12-23 2014-06-20 Iduronato-2-sulfatasa para usarse en el tratamiento del deterioro cognitivo del sindrome de hunter mediante suministro intratecal.

Country Status (9)

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

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112014015352A8 (pt) 2011-12-23 2017-06-13 Shire Human Genetic Therapies tratamento de comprometimento cognitivo da síndrome de hunter por distribuição intratecal de iduronato-2-sulfatase
KR20170004814A (ko) 2015-07-02 2017-01-11 주식회사 녹십자 헌터증후군 치료제
CN108430494A (zh) 2015-12-30 2018-08-21 株式会社绿十字 用于治疗亨特综合征的方法和组合物
CA3076369A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
WO2025125542A1 (en) * 2023-12-15 2025-06-19 F. Hoffmann-La Roche Ag Intrathecal dosage method

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
LT2588130T (lt) 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
BR112012033197A2 (pt) 2010-06-25 2017-06-06 Shire Human Genetic Therapies métodos e composições para liberação de cns de n-sulfatase de heparano.
PL3626258T3 (pl) 2010-06-25 2022-01-17 Shire Human Genetic Therapies, Inc. Sposoby i kompozycje do dostarczania sulfatazy-2-iduronianiu do oun
RS61683B1 (sr) * 2010-06-25 2021-05-31 Shire Human Genetic Therapies Isporuka terapijskih agenasa u centralni nervni sistem
BR112014015352A8 (pt) 2011-12-23 2017-06-13 Shire Human Genetic Therapies tratamento de comprometimento cognitivo da síndrome de hunter por distribuição intratecal de iduronato-2-sulfatase

Also Published As

Publication number Publication date
AU2012358223B2 (en) 2018-01-18
AU2012358223A1 (en) 2014-07-03
EP2793933A4 (en) 2016-06-08
EP2793933B1 (en) 2019-04-17
EP2793933A2 (en) 2014-10-29
US20200376093A1 (en) 2020-12-03
MX2019009846A (es) 2019-10-14
WO2013096912A3 (en) 2015-06-18
US11357832B2 (en) 2022-06-14
US10568941B2 (en) 2020-02-25
CA2859988A1 (en) 2013-06-27
US20180071371A1 (en) 2018-03-15
EA201792099A1 (ru) 2018-05-31
MX2014007642A (es) 2015-03-19
BR112014015352A2 (pt) 2017-06-13
WO2013096912A2 (en) 2013-06-27
BR112014015352A8 (pt) 2017-06-13
US20150328289A1 (en) 2015-11-19
US9682129B2 (en) 2017-06-20
ES2728447T3 (es) 2019-10-24

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