BRPI0015533B8 - enzima alfa-l-iduronidase humana recombinante, composição farmacêutica compreendendo a mesma e método para sua purificação - Google Patents

enzima alfa-l-iduronidase humana recombinante, composição farmacêutica compreendendo a mesma e método para sua purificação

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Publication number
BRPI0015533B8
BRPI0015533B8 BRPI0015533A BR0015533A BRPI0015533B8 BR PI0015533 B8 BRPI0015533 B8 BR PI0015533B8 BR PI0015533 A BRPI0015533 A BR PI0015533A BR 0015533 A BR0015533 A BR 0015533A BR PI0015533 B8 BRPI0015533 B8 BR PI0015533B8
Authority
BR
Brazil
Prior art keywords
recombinant human
iduronidase
purification
pharmaceutical composition
same
Prior art date
Application number
BRPI0015533A
Other languages
English (en)
Other versions
BRPI0015533B1 (pt
BR0015533A (pt
Inventor
Tanamachi Becky
M Starr Christopher
J Wendt Dan
D Kakkis Emil
N Zecherle Gary
M Henstrand John
Chen Lin
Qin Minmin
A Fitzpatrick Paul
Chan Wia-Pan
Original Assignee
Biomarin Pharm Inc
Harbor Ucla Res And Education Institute
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, Harbor Ucla Res And Education Institute filed Critical Biomarin Pharm Inc
Publication of BR0015533A publication Critical patent/BR0015533A/pt
Publication of BRPI0015533B1 publication Critical patent/BRPI0015533B1/pt
Publication of BRPI0015533B8 publication Critical patent/BRPI0015533B8/pt

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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88Lyases (4.)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01014Chitinase (3.2.1.14)
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    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01075Glucan endo-1,6-beta-glucosidase (3.2.1.75)
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    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076L-Iduronidase (3.2.1.76)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y402/00Carbon-oxygen lyases (4.2)
    • C12Y402/02Carbon-oxygen lyases (4.2) acting on polysaccharides (4.2.2)
    • C12Y402/02004Chondroitin ABC lyase (4.2.2.4), i.e. chondroitinase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal 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)
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  • Genetics & Genomics (AREA)
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  • Biomedical Technology (AREA)
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  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

iduronidase-alfa-l recombinante, métodos para sua produção e purificação e métodos para tratamento de doenças causadas por deficiências da mesma. a presente invenção provê uma iduronidase-alfa-l humana recombinante e fragmentos biologicamente ativos e mutantes da mesma, métodos para produzir e purificar, em grande escala, a enzima de iduronidase-alfa-l humana, recombinante e do tipo comercial bem como métodos para tratar certos distúrbios genéticos incluindo deficiência de iduronidase-alfa-l e mucopolissacaridose i (mps i).
BRPI0015533A 1999-11-12 2000-11-09 enzima alfa-l-iduronidase humana recombinante, composição farmacêutica compreendendo a mesma e método para sua purificação BRPI0015533B8 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US09/439,923 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

Publications (3)

Publication Number Publication Date
BR0015533A BR0015533A (pt) 2002-06-25
BRPI0015533B1 BRPI0015533B1 (pt) 2020-12-08
BRPI0015533B8 true BRPI0015533B8 (pt) 2021-05-25

Family

ID=23746702

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0015533A BRPI0015533B8 (pt) 1999-11-12 2000-11-09 enzima alfa-l-iduronidase humana recombinante, composição farmacêutica compreendendo a mesma e método para sua purificação

Country Status (15)

Country Link
US (3) US6426208B1 (pt)
EP (2) EP1332206B1 (pt)
JP (3) JP2004502456A (pt)
CN (2) CN1420924B (pt)
AT (2) ATE342964T1 (pt)
AU (2) AU773663B2 (pt)
BR (1) BRPI0015533B8 (pt)
CA (1) CA2391098C (pt)
DE (2) DE60031456T2 (pt)
ES (2) ES2273748T3 (pt)
HK (2) HK1057059A1 (pt)
IL (3) IL149568A0 (pt)
MX (1) MXPA02004742A (pt)
WO (1) WO2002004616A1 (pt)
ZA (1) ZA200203619B (pt)

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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
WO2002065989A2 (en) * 2001-02-20 2002-08-29 Uab Research Foundation Aminoglycoside treatment for lysosomal storage diseases
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
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
US8105788B2 (en) * 2005-12-08 2012-01-31 The University Of British Columbia Mucopolysaccharidosis (MPS) diagnostic methods, systems, kits and assays associated therewith
BRPI0809234A2 (pt) * 2007-03-21 2014-09-02 Raptor Pharmaceutical Inc Peptídeos de proteína associada a receptor (rap) cíclico
LT3327026T (lt) 2007-07-09 2019-11-11 Genentech Inc Disulfidinio ryšio redukcijos prevencija rekombinantinių polipeptidų gaminimo metu
ES2387251T3 (es) * 2008-02-21 2012-09-19 Fiat Group Automobiles S.P.A. Conjunto de soporte de vidrio para una puerta de vehículo a motor con un borde superior sin armazón
EP3778652A1 (en) 2008-05-07 2021-02-17 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
MX2014015985A (es) 2012-06-19 2015-07-21 Univ Florida Composiciones y metodos para tratar enfermedades.
KR102114111B1 (ko) 2012-08-10 2020-05-25 오시오 코포레이션 디/비/에이 율리아 헬스 안과적 상태 치료시의 컨택트 렌즈 사용
KR102346455B1 (ko) * 2013-03-15 2022-01-04 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Mpsi 치료를 위한 조성물 및 방법
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
AU2018216807A1 (en) * 2017-01-31 2019-08-08 Regenxbio Inc. Treatment of mucopolysaccharidosis I with fully-human glycosylated human alpha-l-iduronidase (IDUA)
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis 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
BR112021012113A2 (pt) 2018-12-19 2021-09-08 The Board Of Trustees Of The Leland Stanford Junior University Moléculas bifuncionais para direcionamento lisossomal e composições e métodos relacionados
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
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Also Published As

Publication number Publication date
BRPI0015533B1 (pt) 2020-12-08
BR0015533A (pt) 2002-06-25
AU8463501A (en) 2002-01-21
EP1332206A1 (en) 2003-08-06
AU773663B2 (en) 2004-06-03
WO2002004616A1 (en) 2002-01-17
CN101407796A (zh) 2009-04-15
ES2273748T3 (es) 2007-05-16
JP2006312648A (ja) 2006-11-16
ZA200203619B (en) 2003-07-30
JP2005046154A (ja) 2005-02-24
EP1792983B1 (en) 2009-04-29
US20060286087A1 (en) 2006-12-21
AU2004208651A1 (en) 2004-09-30
DE60031456D1 (de) 2006-11-30
JP3857705B2 (ja) 2006-12-13
DE60031456T2 (de) 2007-08-23
ATE430193T1 (de) 2009-05-15
MXPA02004742A (es) 2005-09-08
CN1420924A (zh) 2003-05-28
DE60042137D1 (de) 2009-06-10
US6426208B1 (en) 2002-07-30
CA2391098C (en) 2007-10-23
ES2324727T3 (es) 2009-08-13
ATE342964T1 (de) 2006-11-15
JP2004502456A (ja) 2004-01-29
IL149568A (en) 2010-04-29
HK1057059A1 (en) 2004-03-12
CA2391098A1 (en) 2002-01-17
IL192078A (en) 2011-06-30
CN1420924B (zh) 2010-05-05
US20030013179A1 (en) 2003-01-16
EP1332206B1 (en) 2006-10-18
US7041487B2 (en) 2006-05-09
EP1792983A1 (en) 2007-06-06
IL192078A0 (en) 2008-12-29
HK1104836A1 (en) 2008-01-25
IL149568A0 (en) 2002-11-10

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