ES2273748T3 - Alfa-l-iduronidasa recombinante, procedimientos para producir y purificar la misma y procedimientos para tratar enfermedades causadas por su deficiencia. - Google Patents

Alfa-l-iduronidasa recombinante, procedimientos para producir y purificar la misma y procedimientos para tratar enfermedades causadas por su deficiencia. Download PDF

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Publication number
ES2273748T3
ES2273748T3 ES00993857T ES00993857T ES2273748T3 ES 2273748 T3 ES2273748 T3 ES 2273748T3 ES 00993857 T ES00993857 T ES 00993857T ES 00993857 T ES00993857 T ES 00993857T ES 2273748 T3 ES2273748 T3 ES 2273748T3
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ES
Spain
Prior art keywords
iduronidase
recombinant
enzyme
mutants
fragments
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Lifetime
Application number
ES00993857T
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English (en)
Spanish (es)
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
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biomarin Pharmaceutical Inc
Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center
Original Assignee
Biomarin Pharmaceutical Inc
Los Angeles Biomedical Research Institute at Harbor-Ucla Medical Center
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 Pharmaceutical Inc, Los Angeles Biomedical Research Institute at Harbor-Ucla Medical Center filed Critical Biomarin Pharmaceutical Inc
Application granted granted Critical
Publication of ES2273748T3 publication Critical patent/ES2273748T3/es
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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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)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (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)
ES00993857T 1999-11-12 2000-11-09 Alfa-l-iduronidasa recombinante, procedimientos para producir y purificar la misma y procedimientos para tratar enfermedades causadas por su deficiencia. Expired - Lifetime ES2273748T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US439923 1995-05-12
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
ES2273748T3 true ES2273748T3 (es) 2007-05-16

Family

ID=23746702

Family Applications (2)

Application Number Title Priority Date Filing Date
ES00993857T Expired - Lifetime ES2273748T3 (es) 1999-11-12 2000-11-09 Alfa-l-iduronidasa recombinante, procedimientos para producir y purificar la misma y procedimientos para tratar enfermedades causadas por su deficiencia.
ES06011595T Expired - Lifetime ES2324727T3 (es) 1999-11-12 2000-11-09 Alfa-l-iduronidasa recombinante y procedimientos para el tratamiento de las enfermedades causadas por las deficiencias de la misma.

Family Applications After (1)

Application Number Title Priority Date Filing Date
ES06011595T Expired - Lifetime ES2324727T3 (es) 1999-11-12 2000-11-09 Alfa-l-iduronidasa recombinante y procedimientos para el tratamiento de las enfermedades causadas por las deficiencias de la misma.

Country Status (14)

Country Link
US (3) US6426208B1 (cg-RX-API-DMAC7.html)
EP (2) EP1332206B1 (cg-RX-API-DMAC7.html)
JP (3) JP2004502456A (cg-RX-API-DMAC7.html)
CN (2) CN101407796A (cg-RX-API-DMAC7.html)
AT (2) ATE430193T1 (cg-RX-API-DMAC7.html)
AU (2) AU773663B2 (cg-RX-API-DMAC7.html)
BR (1) BRPI0015533B8 (cg-RX-API-DMAC7.html)
CA (1) CA2391098C (cg-RX-API-DMAC7.html)
DE (2) DE60042137D1 (cg-RX-API-DMAC7.html)
ES (2) ES2273748T3 (cg-RX-API-DMAC7.html)
IL (3) IL149568A0 (cg-RX-API-DMAC7.html)
MX (1) MXPA02004742A (cg-RX-API-DMAC7.html)
WO (1) WO2002004616A1 (cg-RX-API-DMAC7.html)
ZA (1) ZA200203619B (cg-RX-API-DMAC7.html)

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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
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
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
EP1668110A2 (en) * 2003-08-21 2006-06-14 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.
JP2009508494A (ja) 2005-09-16 2009-03-05 ラプトール ファーマシューティカル インコーポレイテッド Ｃｒを含むタンパク質に対して特異的な受容体−結合タンパク質（ｒａｐ）変異体を含む組成物およびその使用
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
DK3597659T5 (da) 2007-07-09 2024-09-02 Genentech Inc Forhindring af disulfidbindingsreduktion under rekombinant produktion af polypeptider
EP2093085B1 (en) * 2008-02-21 2012-05-02 Fiat Group Automobiles S.p.A. Glass supporting assembly for a motor vehicle door with an unframed upper edge
CA2723412A1 (en) 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
WO2010148253A2 (en) 2009-06-17 2010-12-23 Zystor Therapeutics, Inc. Formulations for lysosomal enzymes
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
EP3818991A3 (en) 2012-06-19 2021-07-14 University of Florida Research Foundation, Inc. Compositions and methods for treating diseases
JP2015524580A (ja) 2012-08-10 2015-08-24 オシオ コーポレイション ディー／ビー／エー ヨリア ヘルス 眼科的状態の治療におけるコンタクトレンズ使用
EP2984166B1 (en) 2013-03-15 2020-04-22 The Trustees Of The University Of Pennsylvania Compositions for treating mpsi
CA3012195A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
EP3576768A4 (en) * 2017-01-31 2020-11-04 REGENXBIO Inc. TREATMENT OF MUCOPOLYSACCHARIDOSIS I WITH FULLY HUMAN ALPHA-L-IDURONIDASE GLYCOSYLATE (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
JP7449223B2 (ja) 2017-09-22 2024-03-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉ型を治療するための遺伝子療法
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
CN113301925A (zh) 2018-12-19 2021-08-24 小利兰·斯坦福大学理事会 用于溶酶体靶向的双官能分子以及相关的组合物和方法
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
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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
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Also Published As

Publication number Publication date
BRPI0015533B1 (pt) 2020-12-08
WO2002004616A1 (en) 2002-01-17
US20030013179A1 (en) 2003-01-16
US20060286087A1 (en) 2006-12-21
US6426208B1 (en) 2002-07-30
JP3857705B2 (ja) 2006-12-13
EP1792983A1 (en) 2007-06-06
US7041487B2 (en) 2006-05-09
AU773663B2 (en) 2004-06-03
BRPI0015533B8 (pt) 2021-05-25
CN1420924A (zh) 2003-05-28
JP2006312648A (ja) 2006-11-16
ATE430193T1 (de) 2009-05-15
EP1792983B1 (en) 2009-04-29
ZA200203619B (en) 2003-07-30
AU2004208651A1 (en) 2004-09-30
EP1332206B1 (en) 2006-10-18
CA2391098C (en) 2007-10-23
ES2324727T3 (es) 2009-08-13
CN1420924B (zh) 2010-05-05
AU8463501A (en) 2002-01-21
DE60042137D1 (de) 2009-06-10
ATE342964T1 (de) 2006-11-15
DE60031456D1 (de) 2006-11-30
HK1104836A1 (en) 2008-01-25
JP2004502456A (ja) 2004-01-29
CN101407796A (zh) 2009-04-15
HK1057059A1 (en) 2004-03-12
IL192078A0 (en) 2008-12-29
CA2391098A1 (en) 2002-01-17
DE60031456T2 (de) 2007-08-23
IL149568A (en) 2010-04-29
EP1332206A1 (en) 2003-08-06
IL192078A (en) 2011-06-30
JP2005046154A (ja) 2005-02-24
IL149568A0 (en) 2002-11-10
BR0015533A (pt) 2002-06-25
MXPA02004742A (es) 2005-09-08

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