AU773663B2 - Recombinant alpha-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof - Google Patents

Recombinant alpha-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof Download PDF

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Publication number
AU773663B2
AU773663B2 AU84635/01A AU8463501A AU773663B2 AU 773663 B2 AU773663 B2 AU 773663B2 AU 84635/01 A AU84635/01 A AU 84635/01A AU 8463501 A AU8463501 A AU 8463501A AU 773663 B2 AU773663 B2 AU 773663B2
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AU
Australia
Prior art keywords
iduronidase
mutant
fragment
recombinant
enzyme
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
Application number
AU84635/01A
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English (en)
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AU8463501A (en
Inventor
Wia-Pan Chan
Lin Chen
Paul A. Fitzpatrick
John Henstrand
Emil D. Kakkis
Minmin Qin
Christopher M. Starr
Becky Tanamachi
Dan J. Wendt
Gary N. Zecherle
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
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Biomarin Pharmaceutical Inc
Harbor UCLA Research and Education Institute
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Publication of AU8463501A publication Critical patent/AU8463501A/en
Assigned to BIOMARIN PHARMACEUTICAL INC., HARBOR-UCLA RESEARCH AND EDUCATION INSTITUTE reassignment BIOMARIN PHARMACEUTICAL INC. Amend patent request/document other than specification (104) Assignors: BIOMARIN PHARMACEUTICAL INC., HARBOR-UCLA RESEARCH AND EDUCATION INSTITUTE
Application granted granted Critical
Publication of AU773663B2 publication Critical patent/AU773663B2/en
Assigned to BIOMARIN PHARMACEUTICAL INC., LOS ANGELES BIOMEDICAL RESEARCH INSTITUTE AT HARBOUR-UCLA MEDICAL CENTER reassignment BIOMARIN PHARMACEUTICAL INC. Request to Amend Deed and Register Assignors: BIOMARIN PHARMACEUTICAL INC., HARBOR-UCLA RESEARCH AND EDUCATION INSTITUTE
Anticipated expiration legal-status Critical
Expired 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

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  • 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)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
AU84635/01A 1999-11-12 2000-11-09 Recombinant alpha-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof Expired AU773663B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US09/439923 1999-11-12
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2004208651A Division AU2004208651A1 (en) 1999-11-12 2004-09-03 Recombinant Alpha -L- Iduronidase, Methods for Producing and Purifying the Same and Methods for Treating Diseases Caused by Deficiencies Thereof

Publications (2)

Publication Number Publication Date
AU8463501A AU8463501A (en) 2002-01-21
AU773663B2 true AU773663B2 (en) 2004-06-03

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Family Applications (2)

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AU84635/01A Expired AU773663B2 (en) 1999-11-12 2000-11-09 Recombinant alpha-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
AU2004208651A Abandoned AU2004208651A1 (en) 1999-11-12 2004-09-03 Recombinant Alpha -L- Iduronidase, Methods for Producing and Purifying the Same and Methods for Treating Diseases Caused by Deficiencies Thereof

Family Applications After (1)

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AU2004208651A Abandoned AU2004208651A1 (en) 1999-11-12 2004-09-03 Recombinant Alpha -L- Iduronidase, Methods for Producing and Purifying the Same and Methods for Treating Diseases Caused by Deficiencies Thereof

Country Status (14)

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

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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
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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
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KR20060123702A (ko) * 2003-08-21 2006-12-04 큐 세라퓨틱스, 아이엔씨. 리소좀성 축적병에서 결핍된 리소좀 효소전구체의 지속된분비를 위한 유전자 변형 체세포
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.
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
KR20100015786A (ko) * 2007-03-21 2010-02-12 랩터 파마슈티컬 인코포레이티드 환형의 수용체-연관된 단백질(ｒａｐ) 펩티드
KR20190140090A (ko) 2007-07-09 2019-12-18 제넨테크, 인크. 폴리펩티드의 재조합 생산 동안의 디술피드 결합 환원의 방지
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
HRP20170698T1 (hr) 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lizosomski usmjereni peptidi i njihove uporabe
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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
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RS61055B1 (sr) 2012-06-19 2020-12-31 Univ Florida Kompozicije i postupci za lečenje bolesti
MX364260B (es) 2012-08-10 2019-04-17 Osio Corp D/B/A Yolia Health Uso de lentes de contacto en el tratamiento de una afección oftalmológica.
SI2984166T1 (sl) * 2013-03-15 2020-09-30 The Trustees Of The University Of Pennsylvania Sestavki za zdravljenje MPSI
EP3411488A1 (en) 2016-02-03 2018-12-12 The Trustees Of The University Of 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)
MA50016A (fr) 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
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
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
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
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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
BR0015533A (pt) 2002-06-25
US7041487B2 (en) 2006-05-09
CN1420924B (zh) 2010-05-05
CA2391098C (en) 2007-10-23
US20030013179A1 (en) 2003-01-16
US6426208B1 (en) 2002-07-30
ES2273748T3 (es) 2007-05-16
CA2391098A1 (en) 2002-01-17
JP2006312648A (ja) 2006-11-16
JP2005046154A (ja) 2005-02-24
BRPI0015533B1 (pt) 2020-12-08
WO2002004616A1 (en) 2002-01-17
EP1332206B1 (en) 2006-10-18
CN1420924A (zh) 2003-05-28
EP1332206A1 (en) 2003-08-06
ATE342964T1 (de) 2006-11-15
HK1057059A1 (en) 2004-03-12
IL149568A0 (en) 2002-11-10
EP1792983A1 (en) 2007-06-06
EP1792983B1 (en) 2009-04-29
AU8463501A (en) 2002-01-21
ATE430193T1 (de) 2009-05-15
DE60031456D1 (de) 2006-11-30
HK1104836A1 (en) 2008-01-25
IL149568A (en) 2010-04-29
AU2004208651A1 (en) 2004-09-30
IL192078A0 (en) 2008-12-29
CN101407796A (zh) 2009-04-15
BRPI0015533B8 (pt) 2021-05-25
MXPA02004742A (es) 2005-09-08
DE60031456T2 (de) 2007-08-23
JP2004502456A (ja) 2004-01-29
JP3857705B2 (ja) 2006-12-13
ES2324727T3 (es) 2009-08-13
US20060286087A1 (en) 2006-12-21
DE60042137D1 (de) 2009-06-10
ZA200203619B (en) 2003-07-30
IL192078A (en) 2011-06-30

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