BR9910323A - Alfa-l-iduronidase recombinante, métodos para produção e purificação da mesma e métodos para tratamento de doenças ocasionadas deficiência da mesma - Google Patents

Alfa-l-iduronidase recombinante, métodos para produção e purificação da mesma e métodos para tratamento de doenças ocasionadas deficiência da mesma

Info

Publication number
BR9910323A
BR9910323A BR9910323-0A BR9910323A BR9910323A BR 9910323 A BR9910323 A BR 9910323A BR 9910323 A BR9910323 A BR 9910323A BR 9910323 A BR9910323 A BR 9910323A
Authority
BR
Brazil
Prior art keywords
methods
iduronidase
deficiency
purification
production
Prior art date
Application number
BR9910323-0A
Other languages
English (en)
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
Publication of BR9910323A publication Critical patent/BR9910323A/pt

Links

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

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

Abstract

"ALFA-L- IDURONIDASE RECOMBINANTE, MéTODOS PARA PRODUçãO E PURIFICAçãO DA MESMA E MéTODOS PARA TRATAMENTO DE DOENçAS OCASIONADAS POR DEFICIêNCIA DA MESMA" A presente invenção propicia uma <244>-L-iduronidase recombinante e fragmentos e mutantes biologicamente ativos da mesma, métodos para produzir e purificar esta enzima, bem como métodos para tratar alguns distúrbios genéticos incluindo deficiência de <244>-L-iduronidase e mucopolissacaridose I (MPS I).
BR9910323-0A 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 BR9910323A (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US7820998A 1998-05-13 1998-05-13
US17097798A 1998-10-13 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
BR9910323A true BR9910323A (pt) 2001-01-30

Family

ID=26760239

Family Applications (1)

Application Number Title Priority Date Filing Date
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

Country Status (8)

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

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
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
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
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
JP6049623B2 (ja) * 2010-10-22 2016-12-21 カッパーアールエヌエー，インコーポレイテッド α‐Ｌ‐イズロニダーゼ（ＩＤＵＡ）への天然アンチセンス転写物の阻害によるＩＤＵＡ関連疾患の治療
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
MA47436A (fr) * 2017-01-31 2019-12-11 Regenxbio Inc Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylée entièrement humaine (idua)
EP3676385A1 (en) 2017-07-06 2020-07-08 The Trustees of The University of 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
WO1993010244A1 (en) * 1991-11-14 1993-05-27 Women's And Children's Hospital Synthetic alpha-l-iduronidase and genetic sequences encoding same
AU7071196A (en) * 1995-09-14 1997-04-01 Croptech Development Corporation Production of lysosomal enzymes in plant-based expression systems

Also Published As

Publication number Publication date
AU4072499A (en) 1999-11-29
JP2002514429A (ja) 2002-05-21
WO1999058691A3 (en) 2000-02-17
WO1999058691A2 (en) 1999-11-18
EP1078075A2 (en) 2001-02-28
CA2328518A1 (en) 1999-11-18
IL139616A0 (en) 2002-02-10
CN1300323A (zh) 2001-06-20

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Legal Events

Date Code Title Description
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

Free format text: REFERENTE A 5A, 6A, 7A E 8A ANUIDADES.

B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: REFERENTE AO DESPACHO 8.6 PUBLICADO NA RPI 1911 DE 21/08/2007.