DE60031456D1 - 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
DE60031456D1
DE60031456D1 DE60031456T DE60031456T DE60031456D1 DE 60031456 D1 DE60031456 D1 DE 60031456D1 DE 60031456 T DE60031456 T DE 60031456T DE 60031456 T DE60031456 T DE 60031456T DE 60031456 D1 DE60031456 D1 DE 60031456D1
Authority
DE
Germany
Prior art keywords
iduronisase
suffered
treatment
production
purification method
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
DE60031456T
Other languages
English (en)
Other versions
DE60031456T2 (de
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
Harbor Ucla Medical Center
LA BioMed (The Los Angeles Biomedical Res Inst)
Original Assignee
Biomarin Pharmaceutical Inc
Harbor Ucla Medical Center
LA BioMed (The Los Angeles Biomedical 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 Pharmaceutical Inc, Harbor Ucla Medical Center, LA BioMed (The Los Angeles Biomedical Res Inst) filed Critical Biomarin Pharmaceutical Inc
Application granted granted Critical
Publication of DE60031456D1 publication Critical patent/DE60031456D1/de
Publication of DE60031456T2 publication Critical patent/DE60031456T2/de
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Classifications

    • 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
DE60031456T 1999-11-12 2000-11-09 Rekombinante alpha-l-iduronisase, deren herstellungs- und aufreinigungsverfahren, und behandlung von durch diese hervorgerufene mangelerkrankungen Expired - Lifetime DE60031456T2 (de)

Applications Claiming Priority (3)

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
PCT/US2000/031293 WO2002004616A1 (en) 1999-11-12 2000-11-09 RECOMBINANT α-L-IDURONIDASE, METHODS FOR PRODUCING AND PURIFYING THE SAME AND METHODS FOR TREATING DISEASES CAUSED BY DEFICIENCIES THEREOF

Publications (2)

Publication Number Publication Date
DE60031456D1 true DE60031456D1 (de) 2006-11-30
DE60031456T2 DE60031456T2 (de) 2007-08-23

Family

ID=23746702

Family Applications (2)

Application Number Title Priority Date Filing Date
DE60031456T Expired - Lifetime DE60031456T2 (de) 1999-11-12 2000-11-09 Rekombinante alpha-l-iduronisase, deren herstellungs- und aufreinigungsverfahren, und behandlung von durch diese hervorgerufene mangelerkrankungen
DE60042137T Expired - Lifetime DE60042137D1 (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
DE60042137T Expired - Lifetime DE60042137D1 (de) 1999-11-12 2000-11-09 Rekombinante alpha-l-iduronidase und behandlung von durch diese hervorgerufene mangelerkrankungen

Country Status (15)

Country Link
US (3) US6426208B1 (de)
EP (2) EP1792983B1 (de)
JP (3) JP2004502456A (de)
CN (2) CN1420924B (de)
AT (2) ATE430193T1 (de)
AU (2) AU773663B2 (de)
BR (1) BRPI0015533B8 (de)
CA (1) CA2391098C (de)
DE (2) DE60031456T2 (de)
ES (2) ES2273748T3 (de)
HK (2) HK1057059A1 (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
EP1368039A4 (de) * 2001-02-20 2006-03-29 Uab Research Foundation Aminoglycosid-behandlung für lysosomale speicherkrankheiten
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
JP2007514403A (ja) * 2003-08-21 2007-06-07 キュー セラピューティクス インコーポレイティッド リソソーム蓄積症において欠損しているリソソームプロ酵素の持続的分泌のための遺伝子改変体細胞
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.
WO2007035716A2 (en) 2005-09-16 2007-03-29 Raptor Pharmaceutical 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
WO2008116171A1 (en) * 2007-03-21 2008-09-25 Raptor Pharmaceutical Inc. Cyclic receptor-associated protein (rap) peptides
AU2008275229B2 (en) 2007-07-09 2014-11-06 Genentech, Inc. Prevention of disulfide bond reduction during recombinant production of polypeptides
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
ES2629853T3 (es) 2008-05-07 2017-08-16 Biomarin Pharmaceutical Inc. Péptidos de dirección lisosómica y usos de los mismos
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
DE102011102170A1 (de) 2011-05-20 2012-11-22 Emitec Gesellschaft Für Emissionstechnologie Mbh Einspritzvorrichtung zur Einspritzung eines Fluids
KR20150020250A (ko) 2012-06-19 2015-02-25 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 당뇨병을 치료하기 위한 조성물 및 방법
CN104823100B (zh) 2012-08-10 2020-04-24 奥西奥公司以约利亚健康公司名义经营 隐形眼镜和为个体的眼睛测定隐形眼镜的适配的方法
EP2984166B1 (de) 2013-03-15 2020-04-22 The Trustees Of The University Of Pennsylvania Zusammensetzungen zur behandlung von mpsi
SG10201912758TA (en) 2016-02-03 2020-02-27 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
CA3049915A1 (en) * 2017-01-31 2018-08-09 Stephen YOO Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
US11890329B2 (en) 2017-07-06 2024-02-06 The Trustees Of The University Of Pennsylvania AAV9-mediated gene therapy for treating mucopolysaccharidosis type 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
SG11202106209RA (en) 2018-12-19 2021-07-29 Univ Leland Stanford Junior 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
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
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
IL192078A (en) 2011-06-30
ES2273748T3 (es) 2007-05-16
ES2324727T3 (es) 2009-08-13
JP2006312648A (ja) 2006-11-16
US6426208B1 (en) 2002-07-30
CN1420924A (zh) 2003-05-28
CA2391098C (en) 2007-10-23
DE60042137D1 (de) 2009-06-10
EP1332206B1 (de) 2006-10-18
BRPI0015533B8 (pt) 2021-05-25
US20030013179A1 (en) 2003-01-16
JP2005046154A (ja) 2005-02-24
AU2004208651A1 (en) 2004-09-30
ATE430193T1 (de) 2009-05-15
DE60031456T2 (de) 2007-08-23
EP1332206A1 (de) 2003-08-06
AU8463501A (en) 2002-01-21
IL149568A0 (en) 2002-11-10
BR0015533A (pt) 2002-06-25
IL192078A0 (en) 2008-12-29
MXPA02004742A (es) 2005-09-08
CA2391098A1 (en) 2002-01-17
HK1104836A1 (en) 2008-01-25
CN101407796A (zh) 2009-04-15
WO2002004616A1 (en) 2002-01-17
EP1792983A1 (de) 2007-06-06
BRPI0015533B1 (pt) 2020-12-08
US20060286087A1 (en) 2006-12-21
CN1420924B (zh) 2010-05-05
AU773663B2 (en) 2004-06-03
IL149568A (en) 2010-04-29
JP2004502456A (ja) 2004-01-29
EP1792983B1 (de) 2009-04-29
ATE342964T1 (de) 2006-11-15
JP3857705B2 (ja) 2006-12-13
HK1057059A1 (en) 2004-03-12
ZA200203619B (en) 2003-07-30
US7041487B2 (en) 2006-05-09

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