HK1104836A1 - Recombinant alpha-l-iduronidase and methods for treating diseases caused by deficiencies thereof - Google Patents

Recombinant alpha-l-iduronidase and methods for treating diseases caused by deficiencies thereof

Info

Publication number
HK1104836A1
HK1104836A1 HK07113088.9A HK07113088A HK1104836A1 HK 1104836 A1 HK1104836 A1 HK 1104836A1 HK 07113088 A HK07113088 A HK 07113088A HK 1104836 A1 HK1104836 A1 HK 1104836A1
Authority
HK
Hong Kong
Prior art keywords
iduronidase
methods
deficiencies
diseases caused
treating diseases
Prior art date
Application number
HK07113088.9A
Other languages
English (en)
Inventor
John M Henstrand
Minmin Qin
Wia-Pan Chan
Lin Chen
Paul A Fitzpatrick
Dan J Wendt
Gary N Zecherle
Christopher M Starr
Emil D Kakkis
Becky Tanamachi
Original Assignee
Biomarin Pharm Inc
Los Angeles Biomed Res Inst
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, Los Angeles Biomed Res Inst filed Critical Biomarin Pharm Inc
Publication of HK1104836A1 publication Critical patent/HK1104836A1/xx

Links

Classifications

    • 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
    • C12N9/88—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pulmonology (AREA)
  • Diabetes (AREA)
  • Hematology (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)
HK07113088.9A 1999-11-12 2007-11-30 Recombinant alpha-l-iduronidase and methods for treating diseases caused by deficiencies thereof HK1104836A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
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

Publications (1)

Publication Number Publication Date
HK1104836A1 true HK1104836A1 (en) 2008-01-25

Family

ID=23746702

Family Applications (2)

Application Number Title Priority Date Filing Date
HK03109319A HK1057059A1 (en) 1999-11-12 2003-12-22 Recombinant alpha-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
HK07113088.9A HK1104836A1 (en) 1999-11-12 2007-11-30 Recombinant alpha-l-iduronidase and methods for treating diseases caused by deficiencies thereof

Family Applications Before (1)

Application Number Title Priority Date Filing Date
HK03109319A HK1057059A1 (en) 1999-11-12 2003-12-22 Recombinant alpha-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof

Country Status (15)

Country Link
US (3) US6426208B1 (xx)
EP (2) EP1332206B1 (xx)
JP (3) JP2004502456A (xx)
CN (2) CN101407796A (xx)
AT (2) ATE430193T1 (xx)
AU (2) AU773663B2 (xx)
BR (1) BRPI0015533B8 (xx)
CA (1) CA2391098C (xx)
DE (2) DE60031456T2 (xx)
ES (2) ES2273748T3 (xx)
HK (2) HK1057059A1 (xx)
IL (3) IL149568A0 (xx)
MX (1) MXPA02004742A (xx)
WO (1) WO2002004616A1 (xx)
ZA (1) ZA200203619B (xx)

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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
CA2438750A1 (en) * 2001-02-20 2002-08-29 Uab Research Foundation Aminoglycoside treatment for lysosomal storage diseases
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 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
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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
EP2132224B1 (en) * 2007-03-21 2011-12-28 Raptor Pharmaceutical Inc Cyclic receptor-associated protein (rap) peptides
KR20190140090A (ko) 2007-07-09 2019-12-18 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
DK2279210T3 (en) 2008-05-07 2017-07-24 Biomarin Pharm Inc Lysosomal targeting peptides and uses thereof
ES2569514T3 (es) 2009-06-17 2016-05-11 Biomarin Pharmaceutical 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
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.
CN111290143B (zh) 2012-08-10 2023-03-31 奥西奥公司以约利亚健康公司名义经营 隐形眼镜和为个体的眼睛测定隐形眼镜的适配的方法
EP3747998A1 (en) 2013-03-15 2020-12-09 The Trustees of the University of Pennsylvania Compositions for treating mpsi
SG11201806270XA (en) 2016-02-03 2018-08-30 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
US20190358303A1 (en) * 2017-01-31 2019-11-28 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
BR112020000063A2 (pt) 2017-07-06 2020-07-14 The Trustees Of The University Of Pennsylvania terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i
CA3076036A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis 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
JP2022513299A (ja) 2018-12-19 2022-02-07 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー リソソーム標的化のための二官能性分子ならびに関連する組成物および方法
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
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
CN115161284B (zh) * 2022-07-04 2024-07-09 无锡多宁生物科技有限公司 一种lmh悬浮细胞复苏培养方法及其应用

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Also Published As

Publication number Publication date
AU773663B2 (en) 2004-06-03
CA2391098C (en) 2007-10-23
IL192078A0 (en) 2008-12-29
ATE430193T1 (de) 2009-05-15
IL149568A (en) 2010-04-29
BRPI0015533B1 (pt) 2020-12-08
US7041487B2 (en) 2006-05-09
CN1420924B (zh) 2010-05-05
CA2391098A1 (en) 2002-01-17
EP1792983A1 (en) 2007-06-06
BRPI0015533B8 (pt) 2021-05-25
CN1420924A (zh) 2003-05-28
DE60031456D1 (de) 2006-11-30
JP3857705B2 (ja) 2006-12-13
EP1332206B1 (en) 2006-10-18
AU2004208651A1 (en) 2004-09-30
JP2004502456A (ja) 2004-01-29
JP2005046154A (ja) 2005-02-24
IL149568A0 (en) 2002-11-10
JP2006312648A (ja) 2006-11-16
WO2002004616A1 (en) 2002-01-17
ATE342964T1 (de) 2006-11-15
EP1792983B1 (en) 2009-04-29
AU8463501A (en) 2002-01-21
US20060286087A1 (en) 2006-12-21
ZA200203619B (en) 2003-07-30
ES2273748T3 (es) 2007-05-16
US20030013179A1 (en) 2003-01-16
MXPA02004742A (es) 2005-09-08
CN101407796A (zh) 2009-04-15
IL192078A (en) 2011-06-30
ES2324727T3 (es) 2009-08-13
EP1332206A1 (en) 2003-08-06
HK1057059A1 (en) 2004-03-12
DE60042137D1 (de) 2009-06-10
US6426208B1 (en) 2002-07-30
DE60031456T2 (de) 2007-08-23
BR0015533A (pt) 2002-06-25

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