CA2328518A1 - 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
CA2328518A1
CA2328518A1 CA002328518A CA2328518A CA2328518A1 CA 2328518 A1 CA2328518 A1 CA 2328518A1 CA 002328518 A CA002328518 A CA 002328518A CA 2328518 A CA2328518 A CA 2328518A CA 2328518 A1 CA2328518 A1 CA 2328518A1
Authority
CA
Canada
Prior art keywords
iduronidase
alpha
cell line
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.)
Abandoned
Application number
CA002328518A
Other languages
English (en)
French (fr)
Inventor
Becky Tanamachi
Emil D. Kakkis
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.)
HARBOR-UCLA
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2328518A1 publication Critical patent/CA2328518A1/en
Abandoned legal-status Critical Current

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Classifications

    • 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)
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)
CA002328518A 1998-05-13 1999-05-07 Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof Abandoned CA2328518A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US7820998A 1998-05-13 1998-05-13
US09/078,209 1998-05-13
US17097798A 1998-10-13 1998-10-13
US09/170,977 1998-10-13
PCT/US1999/010102 WO1999058691A2 (en) 1998-05-13 1999-05-07 Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof

Publications (1)

Publication Number Publication Date
CA2328518A1 true CA2328518A1 (en) 1999-11-18

Family

ID=26760239

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002328518A Abandoned CA2328518A1 (en) 1998-05-13 1999-05-07 Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof

Country Status (8)

Country Link
EP (1) EP1078075A2 (zh)
JP (1) JP2002514429A (zh)
CN (1) CN1300323A (zh)
AU (1) AU4072499A (zh)
BR (1) BR9910323A (zh)
CA (1) CA2328518A1 (zh)
IL (1) IL139616A0 (zh)
WO (1) WO1999058691A2 (zh)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases 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
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
EP3449934B1 (en) 2000-07-18 2020-05-20 Duke University Treatment of glycogen storage disease type ii
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US9222088B2 (en) * 2010-10-22 2015-12-29 Curna, Inc. Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
CN103487414A (zh) * 2012-06-14 2014-01-01 北京和信非凡生物技术有限公司 一种α-L-艾杜糖醛酸酶活性检测方法、底物和试剂
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
AU2017215211C1 (en) 2016-02-03 2023-11-16 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
WO2018144441A1 (en) * 2017-01-31 2018-08-09 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
SG11201912631PA (en) 2017-07-06 2020-01-30 Univ Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
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
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

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0578790A4 (en) * 1991-11-14 1994-11-02 Womens & Childrens Hospital SYNTHETIC ALPHA-L-IDURONIDASE AND THESE ENCODING GENETIC SEQUENCES.
ATE426031T1 (de) * 1995-09-14 2009-04-15 Virginia Tech Intell Prop Produktion von lysosomalen enzymen in pflanzlichen expressionssystemen

Also Published As

Publication number Publication date
JP2002514429A (ja) 2002-05-21
BR9910323A (pt) 2001-01-30
AU4072499A (en) 1999-11-29
IL139616A0 (en) 2002-02-10
WO1999058691A3 (en) 2000-02-17
CN1300323A (zh) 2001-06-20
WO1999058691A2 (en) 1999-11-18
EP1078075A2 (en) 2001-02-28

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Legal Events

Date Code Title Description
EEER Examination request
FZDE Discontinued