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)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pulmonology (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
AT06011595T ATE430193T1 (de) 1999-11-12 2000-11-09 Rekombinante alpha-l-iduronidase und behandlung von durch diese hervorgerufene mangelerkrankungen
AT00993857T ATE342964T1 (de) 1999-11-12 2000-11-09 Rekombinante alpha-l-iduronisase, deren herstellungs- und aufreinigungsverfahren, und behandlung von durch diese hervorgerufene mangelerkrankungen

Family Applications Before (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) EP1332206B1 (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) DE60042137D1 (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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US7485314B2 (en) * 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
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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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US8105788B2 (en) * 2005-12-08 2012-01-31 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
KR102055873B1 (ko) 2007-07-09 2019-12-13 제넨테크, 인크. 폴리펩티드의 재조합 생산 동안의 디술피드 결합 환원의 방지
EP2093085B1 (de) * 2008-02-21 2012-05-02 Fiat Group Automobiles S.p.A. Glasstützbaueinheit für eine Kraftfahrzeugtür mit einer ungerahmten oberen Kante
JP5627571B2 (ja) 2008-05-07 2014-11-19 バイオマリン ファーマシューティカル インコーポレイテッド リソソーム標的化ペプチドおよびその使用
EP3679942A1 (de) 2009-06-17 2020-07-15 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
KR20150020250A (ko) 2012-06-19 2015-02-25 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 당뇨병을 치료하기 위한 조성물 및 방법
DK2883104T3 (da) 2012-08-10 2024-07-29 Osio Corp D/B/A Yolia Health Anvendelse af kontaktlinse ved behandling af en ofthalmologisk lidelse
AU2014235096B2 (en) 2013-03-15 2020-03-19 The Trustees Of The University Of Pennsylvania Compositions and methods 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
KR20250022877A (ko) * 2017-01-31 2025-02-17 리젠엑스바이오 인크. 완전히-인간 글리코실화된 인간 알파-l-이두로니다아제(idua)를 이용한 뮤코다당증 1형의 치료
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
KR102883778B1 (ko) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
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
AU2019404061A1 (en) 2018-12-19 2021-07-22 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
JP7791517B2 (ja) * 2021-01-04 2025-12-24 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
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Also Published As

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

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