HK1057059A1 - 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

Info

Publication number
HK1057059A1
HK1057059A1 HK03109319A HK03109319A HK1057059A1 HK 1057059 A1 HK1057059 A1 HK 1057059A1 HK 03109319 A HK03109319 A HK 03109319A HK 03109319 A HK03109319 A HK 03109319A HK 1057059 A1 HK1057059 A1 HK 1057059A1
Authority
HK
Hong Kong
Prior art keywords
methods
iduronidase
purifying
deficiencies
producing
Prior art date
Application number
HK03109319A
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
Publication of HK1057059A1 publication Critical patent/HK1057059A1/xx

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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

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)
  • Wood Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Pulmonology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
HK03109319A 1999-11-12 2003-12-22 Recombinant alpha-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof HK1057059A1 (en)

Applications Claiming Priority (2)

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
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 (1)

Publication Number Publication Date
HK1057059A1 true HK1057059A1 (en) 2004-03-12

Family

ID=23746702

Family Applications (2)

Application Number Title Priority Date Filing Date
HK03109319A HK1057059A1 (en) 1999-11-12 2003-12-22 Recombinant alpha-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
HK07113088.9A HK1104836A1 (en) 1999-11-12 2007-11-30 Recombinant alpha-l-iduronidase and methods for treating diseases caused by deficiencies thereof

Family Applications After (1)

Application Number Title Priority Date Filing Date
HK07113088.9A HK1104836A1 (en) 1999-11-12 2007-11-30 Recombinant alpha-l-iduronidase and methods for treating diseases caused by deficiencies thereof

Country Status (15)

Country Link
US (3) US6426208B1 (es)
EP (2) EP1792983B1 (es)
JP (3) JP2004502456A (es)
CN (2) CN1420924B (es)
AT (2) ATE430193T1 (es)
AU (2) AU773663B2 (es)
BR (1) BRPI0015533B8 (es)
CA (1) CA2391098C (es)
DE (2) DE60042137D1 (es)
ES (2) ES2273748T3 (es)
HK (2) HK1057059A1 (es)
IL (3) IL149568A0 (es)
MX (1) MXPA02004742A (es)
WO (1) WO2002004616A1 (es)
ZA (1) ZA200203619B (es)

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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
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
WO2005021716A2 (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
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
US8105788B2 (en) * 2005-12-08 2012-01-31 The University Of British Columbia Mucopolysaccharidosis (MPS) diagnostic methods, systems, kits and assays associated therewith
ATE539089T1 (de) * 2007-03-21 2012-01-15 Raptor Pharmaceutical Inc Cyclische rezeptor-assoziierte-proteine (rap)- peptide
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
AU2009244148B2 (en) 2008-05-07 2014-10-09 Biomarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
US8785168B2 (en) 2009-06-17 2014-07-22 Biomarin Pharmaceutical 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
SI3292875T1 (sl) 2012-06-19 2020-08-31 University Of Florida Research Foundation, Inc. Sestavki in postopki za zdravljenje bolezni
SG11201500994SA (en) 2012-08-10 2015-03-30 Osio Corp D B A Yolia Health Contact lens use in the treatment of an ophthalmologic condition
JP6591956B2 (ja) 2013-03-15 2019-10-16 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 Ｍｐｓ１を治療するための組成物および方法
KR20180121899A (ko) 2016-02-03 2018-11-09 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 I형 점액다당류증을 치료하기 위한 유전자 요법
KR20190109506A (ko) * 2017-01-31 2019-09-25 리젠엑스바이오 인크. 완전히-인간 글리코실화된 인간 알파-l-이두로니다아제(idua)를 이용한 뮤코다당증 1형의 치료
JP2020526514A (ja) 2017-07-06 2020-08-31 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ｉ型を治療するための遺伝子治療
KR20200104852A (ko) 2017-09-22 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 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
EP3897747A4 (en) 2018-12-19 2022-12-07 The Board of Trustees of the Leland Stanford Junior University BI-FUNCTIONAL MOLECULES FOR THE TARGETING OF LYSOSOMES, COMPOSITIONS AND ASSOCIATED 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
CN115161284B (zh) * 2022-07-04 2024-07-09 无锡多宁生物科技有限公司 一种lmh悬浮细胞复苏培养方法及其应用

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Also Published As

Publication number Publication date
US6426208B1 (en) 2002-07-30
US7041487B2 (en) 2006-05-09
CA2391098C (en) 2007-10-23
IL192078A0 (en) 2008-12-29
ZA200203619B (en) 2003-07-30
IL149568A0 (en) 2002-11-10
DE60031456T2 (de) 2007-08-23
ATE430193T1 (de) 2009-05-15
CA2391098A1 (en) 2002-01-17
JP2004502456A (ja) 2004-01-29
MXPA02004742A (es) 2005-09-08
BRPI0015533B8 (pt) 2021-05-25
EP1792983B1 (en) 2009-04-29
AU2004208651A1 (en) 2004-09-30
WO2002004616A1 (en) 2002-01-17
ES2273748T3 (es) 2007-05-16
JP2005046154A (ja) 2005-02-24
AU773663B2 (en) 2004-06-03
BR0015533A (pt) 2002-06-25
HK1104836A1 (en) 2008-01-25
BRPI0015533B1 (pt) 2020-12-08
EP1792983A1 (en) 2007-06-06
ATE342964T1 (de) 2006-11-15
CN1420924A (zh) 2003-05-28
ES2324727T3 (es) 2009-08-13
DE60042137D1 (de) 2009-06-10
US20060286087A1 (en) 2006-12-21
IL149568A (en) 2010-04-29
JP3857705B2 (ja) 2006-12-13
CN1420924B (zh) 2010-05-05
JP2006312648A (ja) 2006-11-16
IL192078A (en) 2011-06-30
US20030013179A1 (en) 2003-01-16
DE60031456D1 (de) 2006-11-30
EP1332206A1 (en) 2003-08-06
CN101407796A (zh) 2009-04-15
AU8463501A (en) 2002-01-21
EP1332206B1 (en) 2006-10-18

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AM Amended specification (according sect 146 of patent law)

Free format text: DELETION OF APPLICANT: JOHN M. HENDSTRAND

AM Amended specification (according sect 146 of patent law)

Free format text: ADDITION OF PRIORITY CLAIM: 12.11.1999 US 439923

Effective date: 20070301

AM Amended specification (according sect 146 of patent law)

Free format text: CORRECTION OF THE NAME OF THE INVENTOR FROM JOHN M. HENDSTRAND TO JOHN M. HENSTRAND.

Effective date: 20070322

PE Patent expired

Effective date: 20201108