ZA200203619B - Recombinant alpha-I-induronidase, methods for producing and purifying the same ad methods for treating diseases caused by deficiencies thereof. - Google Patents

Recombinant alpha-I-induronidase, methods for producing and purifying the same ad methods for treating diseases caused by deficiencies thereof. Download PDF

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Publication number
ZA200203619B
ZA200203619B ZA200203619A ZA200203619A ZA200203619B ZA 200203619 B ZA200203619 B ZA 200203619B ZA 200203619 A ZA200203619 A ZA 200203619A ZA 200203619 A ZA200203619 A ZA 200203619A ZA 200203619 B ZA200203619 B ZA 200203619B
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ZA
South Africa
Prior art keywords
iduronidase
enzyme
human
recombinant
cells
Prior art date
Application number
ZA200203619A
Other languages
English (en)
Inventor
Minmin Qin
Wai-Pan Chan
Lin Chen
Paula A Fitzpatrick
John M Henstrand
Dan J Wendt
Gary N Zecherle
Christopher M Starr
Emil D Kakkis
Becky Tanamachi
Original Assignee
Biomarin Pharm Inc
Harbor Ucla Res & Education
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, Harbor Ucla Res & Education filed Critical Biomarin Pharm Inc
Publication of ZA200203619B publication Critical patent/ZA200203619B/en

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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88Lyases (4.)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/08Antiallergic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01014Chitinase (3.2.1.14)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01075Glucan endo-1,6-beta-glucosidase (3.2.1.75)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076L-Iduronidase (3.2.1.76)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y402/00Carbon-oxygen lyases (4.2)
    • C12Y402/02Carbon-oxygen lyases (4.2) acting on polysaccharides (4.2.2)
    • C12Y402/02004Chondroitin ABC lyase (4.2.2.4), i.e. chondroitinase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Pulmonology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
ZA200203619A 1999-11-12 2002-05-07 Recombinant alpha-I-induronidase, methods for producing and purifying the same ad methods for treating diseases caused by deficiencies thereof. ZA200203619B (en)

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
ZA200203619B true ZA200203619B (en) 2003-07-30

Family

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Family Applications (1)

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ZA200203619A ZA200203619B (en) 1999-11-12 2002-05-07 Recombinant alpha-I-induronidase, methods for producing and purifying the same ad methods for treating diseases caused by deficiencies thereof.

Country Status (14)

Country Link
US (3) US6426208B1 (enExample)
EP (2) EP1792983B1 (enExample)
JP (3) JP2004502456A (enExample)
CN (2) CN101407796A (enExample)
AT (2) ATE430193T1 (enExample)
AU (2) AU773663B2 (enExample)
BR (1) BRPI0015533B8 (enExample)
CA (1) CA2391098C (enExample)
DE (2) DE60042137D1 (enExample)
ES (2) ES2273748T3 (enExample)
IL (3) IL149568A0 (enExample)
MX (1) MXPA02004742A (enExample)
WO (1) WO2002004616A1 (enExample)
ZA (1) ZA200203619B (enExample)

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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
JP2004522763A (ja) * 2001-02-20 2004-07-29 ユーエイビー リサーチ ファンデーション リソソーム蓄積症のアミノグリコシド治療
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 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
CA2535816A1 (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
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US9062126B2 (en) 2005-09-16 2015-06-23 Raptor Pharmaceuticals Inc. Compositions comprising receptor-associated protein (RAP) variants specific for CR-containing proteins and uses thereof
WO2007065273A1 (en) * 2005-12-08 2007-06-14 The University Of British Columbia Mucopolysaccharidosis (mps) diagnostic methods, systems, kits and assays associated therewith
EP2132224B1 (en) * 2007-03-21 2011-12-28 Raptor Pharmaceutical Inc Cyclic receptor-associated protein (rap) peptides
PL3327026T3 (pl) 2007-07-09 2020-02-28 Genentech, Inc. Zapobieganie redukcji wiązań disiarczkowych podczas rekombinacyjnego wytwarzania polipeptydów
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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
HK1209633A1 (en) 2012-06-19 2016-04-08 University Of Florida Research Foundation, Inc. Compositions and methods for treating diabetes
IN2015KN00596A (enExample) 2012-08-10 2015-07-17 Osio Corp D B A Yolia Health
MX386059B (es) * 2013-03-15 2025-03-18 Univ Pennsylvania COMPOSICIONES Y SUS usos EN EL TRATAMIENTO DE LA MUCOPOLISACARIDOSIS TIPO I.
IL260959B2 (en) 2016-02-03 2024-09-01 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
WO2018144441A1 (en) * 2017-01-31 2018-08-09 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
AU2018335752B2 (en) 2017-09-22 2025-04-10 Christian HINDERER 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
WO2020132100A1 (en) 2018-12-19 2020-06-25 The Board Of Trustees Of The Leland Stanford Junior University Bifunctional molecules for lysosomal targeting and related compositions and methods
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
JP2022105471A (ja) * 2021-01-04 2022-07-14 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
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Also Published As

Publication number Publication date
AU773663B2 (en) 2004-06-03
BR0015533A (pt) 2002-06-25
AU2004208651A1 (en) 2004-09-30
ATE430193T1 (de) 2009-05-15
BRPI0015533B8 (pt) 2021-05-25
US6426208B1 (en) 2002-07-30
US20060286087A1 (en) 2006-12-21
IL149568A0 (en) 2002-11-10
ES2273748T3 (es) 2007-05-16
IL149568A (en) 2010-04-29
DE60042137D1 (de) 2009-06-10
WO2002004616A1 (en) 2002-01-17
AU8463501A (en) 2002-01-21
EP1332206A1 (en) 2003-08-06
JP3857705B2 (ja) 2006-12-13
CA2391098A1 (en) 2002-01-17
DE60031456T2 (de) 2007-08-23
US7041487B2 (en) 2006-05-09
CA2391098C (en) 2007-10-23
IL192078A0 (en) 2008-12-29
ATE342964T1 (de) 2006-11-15
EP1332206B1 (en) 2006-10-18
EP1792983A1 (en) 2007-06-06
DE60031456D1 (de) 2006-11-30
HK1104836A1 (en) 2008-01-25
MXPA02004742A (es) 2005-09-08
JP2004502456A (ja) 2004-01-29
IL192078A (en) 2011-06-30
JP2006312648A (ja) 2006-11-16
HK1057059A1 (en) 2004-03-12
CN101407796A (zh) 2009-04-15
CN1420924A (zh) 2003-05-28
JP2005046154A (ja) 2005-02-24
US20030013179A1 (en) 2003-01-16
EP1792983B1 (en) 2009-04-29
ES2324727T3 (es) 2009-08-13
BRPI0015533B1 (pt) 2020-12-08
CN1420924B (zh) 2010-05-05

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