JP2004502456A - 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失により引き起こされる疾病の処理方法 - Google Patents

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失により引き起こされる疾病の処理方法 Download PDF

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JP2004502456A
JP2004502456A JP2002509470A JP2002509470A JP2004502456A JP 2004502456 A JP2004502456 A JP 2004502456A JP 2002509470 A JP2002509470 A JP 2002509470A JP 2002509470 A JP2002509470 A JP 2002509470A JP 2004502456 A JP2004502456 A JP 2004502456A
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iduronidase
human
enzyme
recombinant
cells
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JP2004502456A5 (enExample
Inventor
ヘンドストランド，ジョン　エム．
キン，ミンミン
チャン，ウィア−パン
チェン，リン
フィッツパトリック，ポール　エー．
ウェント，ダン　ジェイ．
ゼチャール，ゲイリー　エヌ．
スター，クリストファー　エム．
カッキス，エミール　ディー．
タナマチ，ベッキー
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Biomarin Pharmaceutical Inc
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Biomarin Pharmaceutical Inc
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Publication of JP2004502456A publication Critical patent/JP2004502456A/ja
Publication of JP2004502456A5 publication Critical patent/JP2004502456A5/ja
Withdrawn legal-status Critical Current

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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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  • Obesity (AREA)
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  • Diabetes (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2002509470A 1999-11-12 2000-11-09 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失により引き起こされる疾病の処理方法 Withdrawn JP2004502456A (ja)

Applications Claiming Priority (2)

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
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

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JP2004242343A Division JP3857705B2 (ja) 1999-11-12 2004-08-23 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失により引き起こされる疾病の処理方法

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JP2004502456A true JP2004502456A (ja) 2004-01-29
JP2004502456A5 JP2004502456A5 (enExample) 2005-11-17

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JP2002509470A Withdrawn JP2004502456A (ja) 1999-11-12 2000-11-09 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失により引き起こされる疾病の処理方法
JP2004242343A Expired - Lifetime JP3857705B2 (ja) 1999-11-12 2004-08-23 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失により引き起こされる疾病の処理方法
JP2006215076A Pending JP2006312648A (ja) 1999-11-12 2006-08-07 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失により引き起こされる疾病の処理方法

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JP2004242343A Expired - Lifetime JP3857705B2 (ja) 1999-11-12 2004-08-23 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失により引き起こされる疾病の処理方法
JP2006215076A Pending JP2006312648A (ja) 1999-11-12 2006-08-07 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失により引き起こされる疾病の処理方法

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US (3) US6426208B1 (enExample)
EP (2) EP1792983B1 (enExample)
JP (3) JP2004502456A (enExample)
CN (2) CN1420924B (enExample)
AT (2) ATE342964T1 (enExample)
AU (2) AU773663B2 (enExample)
BR (1) BRPI0015533B8 (enExample)
CA (1) CA2391098C (enExample)
DE (2) DE60042137D1 (enExample)
ES (2) ES2273748T3 (enExample)
IL (3) IL149568A0 (enExample)
MX (1) MXPA02004742A (enExample)
WO (1) WO2002004616A1 (enExample)
ZA (1) ZA200203619B (enExample)

Cited By (2)

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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
JP2022105471A (ja) * 2021-01-04 2022-07-14 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

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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
US7749971B2 (en) * 2001-02-20 2010-07-06 Bedwell David M 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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US8105788B2 (en) * 2005-12-08 2012-01-31 The University Of British Columbia Mucopolysaccharidosis (MPS) diagnostic methods, systems, kits and assays associated therewith
KR20100015786A (ko) * 2007-03-21 2010-02-12 랩터 파마슈티컬 인코포레이티드 환형의 수용체-연관된 단백질(ｒａｐ) 펩티드
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HUE051632T2 (hu) 2012-06-19 2021-03-29 Univ Florida Betegségek kezelésére szolgáló készítmények és eljárások
CN104823100B (zh) 2012-08-10 2020-04-24 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
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WO2017136500A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
KR20250022877A (ko) * 2017-01-31 2025-02-17 리젠엑스바이오 인크. 완전히-인간 글리코실화된 인간 알파-l-이두로니다아제(idua)를 이용한 뮤코다당증 1형의 치료
AU2018298133A1 (en) 2017-07-06 2020-01-23 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
US20220023434A1 (en) 2018-12-19 2022-01-27 The Board Of Trustees Of The Leland Stanford Junior University Bifunctional Molecules for Lysosomal Targeting and Related Compositions and Methods
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
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
JP2024524614A (ja) 2021-07-14 2024-07-05 ライシア セラピューティクス， インコーポレイテッド Ａｓｇｐｒ細胞表面受容体結合化合物及びコンジュゲート
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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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP2022105471A (ja) * 2021-01-04 2022-07-14 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
JP7791517B2 (ja) 2021-01-04 2025-12-24 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

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EP1792983A1 (en) 2007-06-06
EP1792983B1 (en) 2009-04-29
AU773663B2 (en) 2004-06-03
ES2324727T3 (es) 2009-08-13
EP1332206A1 (en) 2003-08-06
AU2004208651A1 (en) 2004-09-30
IL149568A0 (en) 2002-11-10
BRPI0015533B8 (pt) 2021-05-25
DE60042137D1 (de) 2009-06-10
DE60031456D1 (de) 2006-11-30
HK1104836A1 (en) 2008-01-25
WO2002004616A1 (en) 2002-01-17
MXPA02004742A (es) 2005-09-08
US20030013179A1 (en) 2003-01-16
EP1332206B1 (en) 2006-10-18
CA2391098A1 (en) 2002-01-17
IL149568A (en) 2010-04-29
DE60031456T2 (de) 2007-08-23
CA2391098C (en) 2007-10-23
JP2005046154A (ja) 2005-02-24
IL192078A (en) 2011-06-30
IL192078A0 (en) 2008-12-29
ZA200203619B (en) 2003-07-30
BRPI0015533B1 (pt) 2020-12-08
AU8463501A (en) 2002-01-21
BR0015533A (pt) 2002-06-25
JP2006312648A (ja) 2006-11-16
US6426208B1 (en) 2002-07-30
ATE342964T1 (de) 2006-11-15
US20060286087A1 (en) 2006-12-21
ATE430193T1 (de) 2009-05-15
ES2273748T3 (es) 2007-05-16
JP3857705B2 (ja) 2006-12-13
CN1420924A (zh) 2003-05-28
CN101407796A (zh) 2009-04-15
US7041487B2 (en) 2006-05-09
HK1057059A1 (en) 2004-03-12
CN1420924B (zh) 2010-05-05

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