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WO1999058691A3 - (alpha)-l-iduronidase recombinee, procedes de production et de purification de celle-ci et methodes de traitement de maladies dues a des deficits en celle-ci - Google Patents

(alpha)-l-iduronidase recombinee, procedes de production et de purification de celle-ci et methodes de traitement de maladies dues a des deficits en celle-ci Download PDF

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Publication number
WO1999058691A3
WO1999058691A3 PCT/US1999/010102 US9910102W WO9958691A3 WO 1999058691 A3 WO1999058691 A3 WO 1999058691A3 US 9910102 W US9910102 W US 9910102W WO 9958691 A3 WO9958691 A3 WO 9958691A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
iduronidase
recombinant
purifying
deficiencies
Prior art date
Application number
PCT/US1999/010102
Other languages
English (en)
Other versions
WO1999058691A2 (fr
Inventor
Emil D Kakkis
Becky Tanamachi
Original Assignee
Harbor Ucla
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 Harbor Ucla filed Critical Harbor Ucla
Priority to AU40724/99A priority Critical patent/AU4072499A/en
Priority to JP2000548482A priority patent/JP2002514429A/ja
Priority to BR9910323-0A priority patent/BR9910323A/pt
Priority to EP99924155A priority patent/EP1078075A2/fr
Priority to IL13961699A priority patent/IL139616A0/xx
Priority to CA002328518A priority patent/CA2328518A1/fr
Publication of WO1999058691A2 publication Critical patent/WO1999058691A2/fr
Publication of WO1999058691A3 publication Critical patent/WO1999058691A3/fr

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    • 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

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente invention concerne une α-L-iduronidase, des fragments et des mutants biologiquement actifs de celle-ci, des procédés de production et de purification de cette enzyme, ainsi que des méthodes de traitement de certaines affections génétiques comprenant un déficit en α-L-iduronidase et la mucopolysaccharidose I (MPS I).
PCT/US1999/010102 1998-05-13 1999-05-07 (alpha)-l-iduronidase recombinee, procedes de production et de purification de celle-ci et methodes de traitement de maladies dues a des deficits en celle-ci WO1999058691A2 (fr)

Priority Applications (6)

Application Number Priority Date Filing Date Title
AU40724/99A AU4072499A (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
JP2000548482A JP2002514429A (ja) 1998-05-13 1999-05-07 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠損により引き起こされる疾病の治療方法
BR9910323-0A BR9910323A (pt) 1998-05-13 1999-05-07 Alfa-l-iduronidase recombinante, métodos para produção e purificação da mesma e métodos para tratamento de doenças ocasionadas deficiência da mesma
EP99924155A EP1078075A2 (fr) 1998-05-13 1999-05-07 (alpha)-l-iduronidase recombinee, procedes de production et de purification de celle-ci et methodes de traitement de maladies dues a des deficits en celle-ci
IL13961699A IL139616A0 (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
CA002328518A CA2328518A1 (fr) 1998-05-13 1999-05-07 (alpha)-l-iduronidase recombinee, procedes de production et de purification de celle-ci et methodes de traitement de maladies dues a des deficits en celle-ci

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US7820998A 1998-05-13 1998-05-13
US17097798A 1998-10-13 1998-10-13
US09/078,209 1998-10-13
US09/170,977 1998-10-13

Publications (2)

Publication Number Publication Date
WO1999058691A2 WO1999058691A2 (fr) 1999-11-18
WO1999058691A3 true WO1999058691A3 (fr) 2000-02-17

Family

ID=26760239

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1999/010102 WO1999058691A2 (fr) 1998-05-13 1999-05-07 (alpha)-l-iduronidase recombinee, procedes de production et de purification de celle-ci et methodes de traitement de maladies dues a des deficits en celle-ci

Country Status (8)

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

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
DK1782825T3 (da) 2000-07-18 2014-11-03 Univ Duke Behandling af glycogenlagringssygdom type II
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
KR101865433B1 (ko) * 2010-10-22 2018-07-13 큐알엔에이, 인크. 알파-l 이두로니다아제 (idua)에 대한 자연 안티센스 전사체의 저해에 의한 idua 관련된 질환의 치료
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
SG10201707319UA (en) * 2013-03-15 2017-10-30 Univ Pennsylvania Compositions and methods for treating mpsi
WO2017136500A1 (fr) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
EP3576768A4 (fr) * 2017-01-31 2020-11-04 REGENXBIO Inc. Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylée entièrement humaine (idua)
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
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993010244A1 (fr) * 1991-11-14 1993-05-27 Women's And Children's Hospital α-L IDURONIDASE SYNTHETIQUE ET SEQUENCES GENETIQUES LA CODANT
WO1997010353A1 (fr) * 1995-09-14 1997-03-20 Virginia Tech Intellectual Property, Inc. Production d'enzymes lysosomiales par des systemes d'expression vegetaux

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993010244A1 (fr) * 1991-11-14 1993-05-27 Women's And Children's Hospital α-L IDURONIDASE SYNTHETIQUE ET SEQUENCES GENETIQUES LA CODANT
WO1997010353A1 (fr) * 1995-09-14 1997-03-20 Virginia Tech Intellectual Property, Inc. Production d'enzymes lysosomiales par des systemes d'expression vegetaux

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
CLEMENTS P ET AL: "Human alpha-L-iduronidase. 1. Purification, monoclonal antibody production, native and subunit molecular mass", EUR J BIOCHEM, vol. 152, no. 1, 1 October 1985 (1985-10-01), pages 21 - 28, XP000857400 *
KAKKIS E ET AL: "Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I", BIOCHEM MOL MED, vol. 58, no. 2, August 1996 (1996-08-01), pages 156 - 167, XP000862844 *
KAKKIS E ET AL: "Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells", PROTEIN EXPR PURIF, vol. 5, no. 3, June 1994 (1994-06-01), pages 225 - 232, XP000857380 *
SHULL R ET AL: "Enzyme replacement in a canine model of Hurler syndrome", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA., vol. 91, no. 26, 20 December 1994 (1994-12-20), pages 12937 - 12941, XP002125064 *
UNGER E ET AL: "Recombinant alpha-L-iduronidase: characterization of the purified enzyme and correction of mucopolysaccharidosis type I fibroblasts", BIOCHEM J, vol. 304, 15 November 1994 (1994-11-15), pages 43 - 49, XP000857388 *

Also Published As

Publication number Publication date
BR9910323A (pt) 2001-01-30
EP1078075A2 (fr) 2001-02-28
WO1999058691A2 (fr) 1999-11-18
CA2328518A1 (fr) 1999-11-18
IL139616A0 (en) 2002-02-10
AU4072499A (en) 1999-11-29
CN1300323A (zh) 2001-06-20
JP2002514429A (ja) 2002-05-21

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