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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

Info

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

Links

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Pulmonology (AREA)
  • Obesity (AREA)
  • Physical Education & Sports Medicine (AREA)
  • 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 (14)

Country Link
US (3) US6426208B1 (pt)
EP (2) EP1332206B1 (pt)
JP (3) JP2004502456A (pt)
CN (2) CN101407796A (pt)
AT (2) ATE342964T1 (pt)
AU (2) AU773663B2 (pt)
BR (1) BRPI0015533B8 (pt)
CA (1) CA2391098C (pt)
DE (2) DE60042137D1 (pt)
ES (2) ES2324727T3 (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
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
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
WO2007065273A1 (en) * 2005-12-08 2007-06-14 The University Of British Columbia Mucopolysaccharidosis (mps) diagnostic methods, systems, kits and assays associated therewith
CN101861334A (zh) * 2007-03-21 2010-10-13 雷普特药品公司 受体相关蛋白（rap）环肽
KR102055873B1 (ko) 2007-07-09 2019-12-13 제넨테크, 인크. 폴리펩티드의 재조합 생산 동안의 디술피드 결합 환원의 방지
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
CN102066422B (zh) 2008-05-07 2015-07-22 宝玛瑞制药公司 溶酶体靶向肽及其应用
ES2758827T3 (es) 2009-06-17 2020-05-06 Biomarin Pharm Inc Formulaciones para enzimas lisosómicas
WO2012101671A1 (en) * 2011-01-25 2012-08-02 Jcr Pharmaceuticals Co., Ltd. Method for production of human recombinant iduronate 2-sulfatase
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PL3292875T3 (pl) 2012-06-19 2020-11-16 University Of Florida Research Foundation, Inc. Kompozycje i sposoby leczenia chorób
KR102184340B1 (ko) 2012-08-10 2020-12-01 오시오 코포레이션 디/비/에이 율리아 헬스 안과적 상태 치료시의 컨택트 렌즈 사용
CA2901328C (en) 2013-03-15 2023-08-01 The Trustees Of The University Of Pennsylvania Compositions and methods for treating mps1
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
BR112019015482A2 (pt) * 2017-01-31 2020-03-31 Regenxbio Inc. Método para tratar um indivíduo humano diagnosticado com mucopolissacaridose i (mps i)
EP3676385A1 (en) 2017-07-06 2020-07-08 The Trustees of The University of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
WO2019060662A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II
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
ES2324727T3 (es) 2009-08-13
US20060286087A1 (en) 2006-12-21
BRPI0015533B1 (pt) 2020-12-08
DE60031456D1 (de) 2006-11-30
WO2002004616A1 (en) 2002-01-17
DE60042137D1 (de) 2009-06-10
HK1057059A1 (en) 2004-03-12
JP2005046154A (ja) 2005-02-24
AU773663B2 (en) 2004-06-03
EP1792983A1 (en) 2007-06-06
CA2391098A1 (en) 2002-01-17
IL192078A0 (en) 2008-12-29
ZA200203619B (en) 2003-07-30
AU8463501A (en) 2002-01-21
US7041487B2 (en) 2006-05-09
ATE342964T1 (de) 2006-11-15
ATE430193T1 (de) 2009-05-15
JP2004502456A (ja) 2004-01-29
HK1104836A1 (en) 2008-01-25
US20030013179A1 (en) 2003-01-16
EP1332206B1 (en) 2006-10-18
ES2273748T3 (es) 2007-05-16
JP2006312648A (ja) 2006-11-16
JP3857705B2 (ja) 2006-12-13
CN1420924A (zh) 2003-05-28
IL192078A (en) 2011-06-30
CN1420924B (zh) 2010-05-05
DE60031456T2 (de) 2007-08-23
CN101407796A (zh) 2009-04-15
AU2004208651A1 (en) 2004-09-30
IL149568A0 (en) 2002-11-10
MXPA02004742A (es) 2005-09-08
IL149568A (en) 2010-04-29
US6426208B1 (en) 2002-07-30
EP1792983B1 (en) 2009-04-29
CA2391098C (en) 2007-10-23
EP1332206A1 (en) 2003-08-06
BR0015533A (pt) 2002-06-25

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B06G Technical and formal requirements: other requirements [chapter 6.7 patent gazette]

Free format text: EM VIRTUDE DO ACRESCIMO DE 6 (SEIS) REIVINDICACOES NO NOVO QUADRO REIVINDICATORIO SUBMETIDO ATRAVES DA PETICAO NO 020100100016 DE 25/10/2010, SEM QUE TENHA SIDO FEITA A DEVIDA RETRIBUICAO DAS REIVINDICACOES ADICIONAIS, PEDE-SE A REQUERENTE QUE SEJA SANADA TAL IRREGULARIDADE PARA QUE SE DE PROSSEGUIMENTO AO EXAME TECNICO.

B06A Patent application procedure suspended [chapter 6.1 patent gazette]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]
B06A Patent application procedure suspended [chapter 6.1 patent gazette]
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