ATE342964T1 - Rekombinante alpha-l-iduronisase, deren herstellungs- und aufreinigungsverfahren, und behandlung von durch diese hervorgerufene mangelerkrankungen - Google Patents

Rekombinante alpha-l-iduronisase, deren herstellungs- und aufreinigungsverfahren, und behandlung von durch diese hervorgerufene mangelerkrankungen

Info

Publication number
ATE342964T1
ATE342964T1 AT00993857T AT00993857T ATE342964T1 AT E342964 T1 ATE342964 T1 AT E342964T1 AT 00993857 T AT00993857 T AT 00993857T AT 00993857 T AT00993857 T AT 00993857T AT E342964 T1 ATE342964 T1 AT E342964T1
Authority
AT
Austria
Prior art keywords
iduronisase
treatment
production
purification method
diseases caused
Prior art date
Application number
AT00993857T
Other languages
English (en)
Inventor
John M Henstrand
Minmin Qin
Wia-Pan Chan
Lin Chen
Paul A Fitzpatrick
Dan J Wendt
Gary N Zecherle
Christopher M Starr
Emil D Kakkis
Becky Tanamachi
Original Assignee
Biomarin Pharm Inc
Los Angeles Biomed Res Inst
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 Biomarin Pharm Inc, Los Angeles Biomed Res Inst filed Critical Biomarin Pharm Inc
Application granted granted Critical
Publication of ATE342964T1 publication Critical patent/ATE342964T1/de

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    • 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
    • C12N9/88—Lyases (4.)
    • 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
    • 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
    • A61P19/00—Drugs for skeletal disorders
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/08—Antiallergic agents
    • 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
    • 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)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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)
    • C12Y302/01014—Chitinase (3.2.1.14)
    • 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)
    • C12Y302/01075—Glucan endo-1,6-beta-glucosidase (3.2.1.75)
    • 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
    • C12Y402/00—Carbon-oxygen lyases (4.2)
    • C12Y402/02—Carbon-oxygen lyases (4.2) acting on polysaccharides (4.2.2)
    • C12Y402/02004—Chondroitin ABC lyase (4.2.2.4), i.e. chondroitinase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Pulmonology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
AT00993857T 1999-11-12 2000-11-09 Rekombinante alpha-l-iduronisase, deren herstellungs- und aufreinigungsverfahren, und behandlung von durch diese hervorgerufene mangelerkrankungen ATE342964T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US09/439,923 US6426208B1 (en) 1999-11-12 1999-11-12 Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof

Publications (1)

Publication Number Publication Date
ATE342964T1 true ATE342964T1 (de) 2006-11-15

Family

ID=23746702

Family Applications (2)

Application Number Title Priority Date Filing Date
AT00993857T ATE342964T1 (de) 1999-11-12 2000-11-09 Rekombinante alpha-l-iduronisase, deren herstellungs- und aufreinigungsverfahren, und behandlung von durch diese hervorgerufene mangelerkrankungen
AT06011595T ATE430193T1 (de) 1999-11-12 2000-11-09 Rekombinante alpha-l-iduronidase und behandlung von durch diese hervorgerufene mangelerkrankungen

Family Applications After (1)

Application Number Title Priority Date Filing Date
AT06011595T ATE430193T1 (de) 1999-11-12 2000-11-09 Rekombinante alpha-l-iduronidase und behandlung von durch diese hervorgerufene mangelerkrankungen

Country Status (14)

Country Link
US (3) US6426208B1 (de)
EP (2) EP1792983B1 (de)
JP (3) JP2004502456A (de)
CN (2) CN1420924B (de)
AT (2) ATE342964T1 (de)
AU (2) AU773663B2 (de)
BR (1) BRPI0015533B8 (de)
CA (1) CA2391098C (de)
DE (2) DE60031456T2 (de)
ES (2) ES2324727T3 (de)
IL (3) IL149568A0 (de)
MX (1) MXPA02004742A (de)
WO (1) WO2002004616A1 (de)
ZA (1) ZA200203619B (de)

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US6569661B1 (en) * 1999-11-12 2003-05-27 Biomarin Pharmaceutical Inc. Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6585971B1 (en) * 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
CA2438750A1 (en) * 2001-02-20 2002-08-29 Uab Research Foundation Aminoglycoside treatment for lysosomal storage diseases
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7485314B2 (en) * 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
US20050026823A1 (en) 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
AU2004268207A1 (en) * 2003-08-21 2005-03-10 Q Therapeutics, Inc. Genetically modified somatic cells for sustained secretion of lysosomal proenzymes deficient in lysosomal storage disorders
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
MXPA03011987A (es) * 2003-12-19 2005-06-23 Osio Sancho Alberto Metodo para el tratamiento de la presbicia induciendo cambios en el poder y fisiologia corneal.
EP1945240B1 (de) 2005-09-16 2016-12-28 Raptor Pharmaceutical Inc Zusammensetzungen mit rezeptor-assoziierten protein (rap) varianten, die für cr-haltige proteine spezifisch sind und ihre verwendungen
WO2007065273A1 (en) * 2005-12-08 2007-06-14 The University Of British Columbia Mucopolysaccharidosis (mps) diagnostic methods, systems, kits and assays associated therewith
US8440629B2 (en) * 2007-03-21 2013-05-14 Raptor Pharmaceuticals Inc. Cyclic receptor-associated protein (RAP) peptides
HUE046992T2 (hu) 2007-07-09 2020-04-28 Genentech Inc Diszulfidkötés redukciójának megelõzése polipeptidek rekombináns elõállítása során
ES2387251T3 (es) * 2008-02-21 2012-09-19 Fiat Group Automobiles S.P.A. Conjunto de soporte de vidrio para una puerta de vehículo a motor con un borde superior sin armazón
HRP20170698T1 (hr) 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lizosomski usmjereni peptidi i njihove uporabe
EP2475376B1 (de) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulierungen für lysosomale enzyme
WO2012101671A1 (en) * 2011-01-25 2012-08-02 Jcr Pharmaceuticals Co., Ltd. Method for production of human recombinant iduronate 2-sulfatase
DE102011102170B4 (de) 2011-05-20 2024-06-06 Vitesco Technologies GmbH Einspritzvorrichtung zur Einspritzung eines Fluids
PT3292875T (pt) 2012-06-19 2020-08-31 Univ Florida Composições e métodos para tratamento de doenças
ES2994608T3 (en) 2012-08-10 2025-01-27 Osio Corp D/B/A Yolia Health Contact lens use in the treatment of an ophthalmologic condition
HRP20201063T1 (hr) 2013-03-15 2020-11-27 The Trustees Of The University Of Pennsylvania Pripravci za liječenje mpsi
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
AU2018216807A1 (en) * 2017-01-31 2019-08-08 Regenxbio Inc. Treatment of mucopolysaccharidosis I with fully-human glycosylated human alpha-l-iduronidase (IDUA)
BR112020000063A2 (pt) 2017-07-06 2020-07-14 The Trustees Of The University Of Pennsylvania terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i
CA3076036A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
CN107699590A (zh) * 2017-10-13 2018-02-16 成都中医药大学 一种制备重组人α‑L‑艾杜糖醛酸酶的方法
EP3897747A4 (de) 2018-12-19 2022-12-07 The Board of Trustees of the Leland Stanford Junior University Bifunktionelle moleküle für lysosomales targeting und verwandte zusammensetzungen und verfahren
CN110577559B (zh) * 2019-08-19 2023-02-17 江西科技师范大学 α-L-艾杜糖醛酸酶测定用荧光糖苷酶底物的合成方法
WO2022145449A1 (ja) * 2021-01-04 2022-07-07 国立大学法人徳島大学 ライソゾームを標的とした新規ｄｄｓの開発
CN115109790A (zh) * 2021-03-23 2022-09-27 北京据德医药科技有限公司 一种重组a-L-艾杜糖醛酸普酶及其制备方法
EP4370157A1 (de) 2021-07-14 2024-05-22 Lycia Therapeutics, Inc. Verbindungen und konjugate zur bindung des aspr-zelloberflächenrezeptors
CN114181318B (zh) * 2021-11-08 2023-08-01 四川大学 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用
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Also Published As

Publication number Publication date
DE60042137D1 (de) 2009-06-10
US6426208B1 (en) 2002-07-30
EP1792983A1 (de) 2007-06-06
DE60031456D1 (de) 2006-11-30
HK1104836A1 (en) 2008-01-25
AU773663B2 (en) 2004-06-03
JP3857705B2 (ja) 2006-12-13
ES2273748T3 (es) 2007-05-16
DE60031456T2 (de) 2007-08-23
IL149568A (en) 2010-04-29
JP2006312648A (ja) 2006-11-16
CA2391098A1 (en) 2002-01-17
US7041487B2 (en) 2006-05-09
CA2391098C (en) 2007-10-23
IL149568A0 (en) 2002-11-10
BRPI0015533B8 (pt) 2021-05-25
ATE430193T1 (de) 2009-05-15
MXPA02004742A (es) 2005-09-08
HK1057059A1 (en) 2004-03-12
JP2005046154A (ja) 2005-02-24
EP1792983B1 (de) 2009-04-29
EP1332206A1 (de) 2003-08-06
AU8463501A (en) 2002-01-21
US20060286087A1 (en) 2006-12-21
IL192078A0 (en) 2008-12-29
US20030013179A1 (en) 2003-01-16
IL192078A (en) 2011-06-30
BRPI0015533B1 (pt) 2020-12-08
EP1332206B1 (de) 2006-10-18
WO2002004616A1 (en) 2002-01-17
CN1420924A (zh) 2003-05-28
CN1420924B (zh) 2010-05-05
BR0015533A (pt) 2002-06-25
ES2324727T3 (es) 2009-08-13
JP2004502456A (ja) 2004-01-29
CN101407796A (zh) 2009-04-15
AU2004208651A1 (en) 2004-09-30
ZA200203619B (en) 2003-07-30

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