HK1057059A1 - Recombinant alpha-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof - Google Patents

Recombinant alpha-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof

Info

Publication number
HK1057059A1
HK1057059A1 HK03109319A HK03109319A HK1057059A1 HK 1057059 A1 HK1057059 A1 HK 1057059A1 HK 03109319 A HK03109319 A HK 03109319A HK 03109319 A HK03109319 A HK 03109319A HK 1057059 A1 HK1057059 A1 HK 1057059A1
Authority
HK
Hong Kong
Prior art keywords
methods
iduronidase
purifying
deficiencies
producing
Prior art date
Application number
HK03109319A
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 HK1057059A1 publication Critical patent/HK1057059A1/xx

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61P37/08Antiallergic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
    • 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)
    • C12Y302/01075Glucan endo-1,6-beta-glucosidase (3.2.1.75)
    • 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)
    • C12Y302/01076L-Iduronidase (3.2.1.76)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pulmonology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
HK03109319A 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 HK1057059A1 (en)

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

Publications (1)

Publication Number Publication Date
HK1057059A1 true HK1057059A1 (en) 2004-03-12

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 After (1)

Application Number Title Priority Date Filing Date
HK07113088.9A HK1104836A1 (en) 1999-11-12 2007-11-30 Recombinant alpha-l-iduronidase and methods for treating diseases caused by deficiencies thereof

Country Status (15)

Country Link
US (3) US6426208B1 (xx)
EP (2) EP1792983B1 (xx)
JP (3) JP2004502456A (xx)
CN (2) CN101407796A (xx)
AT (2) ATE342964T1 (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)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP1368039A4 (en) * 2001-02-20 2006-03-29 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
WO2005021716A2 (en) * 2003-08-21 2005-03-10 Q Therapeutics, Inc. Genetically modified somatic cells for sustained secretion of lysosomal proenzymes deficient in lysosomal storage disorders
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.
EP1945240B1 (en) 2005-09-16 2016-12-28 Raptor Pharmaceutical 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
EP2132224B1 (en) * 2007-03-21 2011-12-28 Raptor Pharmaceutical Inc Cyclic receptor-associated protein (rap) peptides
LT3597659T (lt) 2007-07-09 2023-05-10 Genentech, Inc. Disulfidinės jungties redukcijos prevencijos būdas gaminant polipeptidą rekombinantiniu būdu
EP2093085B1 (en) * 2008-02-21 2012-05-02 Fiat Group Automobiles S.p.A. Glass supporting assembly for a motor vehicle door with an unframed upper edge
AU2009244148B2 (en) 2008-05-07 2014-10-09 Biomarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
EP3075386B1 (en) 2009-06-17 2019-10-16 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
EP3818991A3 (en) 2012-06-19 2021-07-14 University of Florida Research Foundation, Inc. Compositions and methods for treating diseases
SG11201500994SA (en) 2012-08-10 2015-03-30 Osio Corp D B A Yolia Health Contact lens use in the treatment of an ophthalmologic condition
PT2984166T (pt) 2013-03-15 2020-07-16 Univ Pennsylvania Composições para tratar mps i
BR112018015751A2 (pt) 2016-02-03 2019-02-05 Univ Pennsylvania terapia gênica para tratamento de mucopolissacaridose tipo i
KR20190109506A (ko) * 2017-01-31 2019-09-25 리젠엑스바이오 인크. 완전히-인간 글리코실화된 인간 알파-l-이두로니다아제(idua)를 이용한 뮤코다당증 1형의 치료
EP3676385A1 (en) 2017-07-06 2020-07-08 The Trustees of The University of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
EP3684938A1 (en) 2017-09-22 2020-07-29 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
WO2020132100A1 (en) 2018-12-19 2020-06-25 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
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
CN115161284A (zh) * 2022-07-04 2022-10-11 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

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL281225A (xx) 1961-07-20
GB1029548A (en) 1963-07-17 1966-05-11 Amagasaki Iron & Steel Mfg Co Corrugated roofing sheet
US3472931A (en) 1969-01-17 1969-10-14 Foster Milburn Co Percutaneous absorption with lower alkyl amides
US3891757A (en) 1971-11-11 1975-06-24 Alza Corp Anaesthetic topical and percutaneous administration with selected promoters
US5270051A (en) 1991-10-15 1993-12-14 Harris Donald H Enzyme-orthokeratology
CA2099503C (en) 1991-11-14 2007-10-09 Hamish Steele Scott Synthetic alpha-l-iduronidase and genetic sequences encoding same
US6149909A (en) * 1995-06-23 2000-11-21 Women's And Children's Hospital Synthetic α-L-iduronidase and genetic sequences encoding same
US5278051A (en) * 1991-12-12 1994-01-11 New York University Construction of geometrical objects from polynucleotides
EP0865499B1 (en) 1995-09-14 2009-03-18 Virginia Tech Intellectual Properties, Inc. Production of lysosomal enzymes in plant-based expression systems
GB9807464D0 (en) 1998-04-07 1998-06-10 Pharming Bv Purification of human acid µ-glucosidase
JP2002514429A (ja) 1998-05-13 2002-05-21 ハーバー−ユーシーエルエー 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損により引き起こされる疾病の治療方法

Also Published As

Publication number Publication date
IL192078A0 (en) 2008-12-29
DE60042137D1 (de) 2009-06-10
US20060286087A1 (en) 2006-12-21
US6426208B1 (en) 2002-07-30
CA2391098C (en) 2007-10-23
ES2324727T3 (es) 2009-08-13
CN1420924B (zh) 2010-05-05
US7041487B2 (en) 2006-05-09
EP1332206B1 (en) 2006-10-18
US20030013179A1 (en) 2003-01-16
WO2002004616A1 (en) 2002-01-17
BR0015533A (pt) 2002-06-25
JP2006312648A (ja) 2006-11-16
DE60031456D1 (de) 2006-11-30
HK1104836A1 (en) 2008-01-25
CN1420924A (zh) 2003-05-28
ATE430193T1 (de) 2009-05-15
MXPA02004742A (es) 2005-09-08
EP1792983A1 (en) 2007-06-06
JP3857705B2 (ja) 2006-12-13
IL149568A0 (en) 2002-11-10
IL149568A (en) 2010-04-29
BRPI0015533B1 (pt) 2020-12-08
BRPI0015533B8 (pt) 2021-05-25
AU773663B2 (en) 2004-06-03
IL192078A (en) 2011-06-30
EP1792983B1 (en) 2009-04-29
AU2004208651A1 (en) 2004-09-30
ES2273748T3 (es) 2007-05-16
DE60031456T2 (de) 2007-08-23
JP2004502456A (ja) 2004-01-29
CA2391098A1 (en) 2002-01-17
EP1332206A1 (en) 2003-08-06
ATE342964T1 (de) 2006-11-15
JP2005046154A (ja) 2005-02-24
ZA200203619B (en) 2003-07-30
CN101407796A (zh) 2009-04-15
AU8463501A (en) 2002-01-21

Similar Documents

Publication Publication Date Title
IL192078A0 (en) Recombinant ??-l-iduronidase,methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
KR100521829B1 (en) Compositions and Methods for the Treatment of Alzheimer's Disease, Central Nervous System Injury, and Inflammatory Diseases
NZ507103A (en) Cell differentiation/proliferation and maintenance factor and uses thereof
DE69938507D1 (en) Fragment des "connective tissue growth factor" (ctgf)
AU1520195A (en) Use of anti-tnf antibodies as drugs in treating diseases involving elevated interleukin-6 serum levels
WO2002038775A3 (en) METHODS FOR PRODUCING AND PURIFYING RECOMBINANT α-L-IDURONIDASE
NO20002702L (no) Tienopyrimidiner
WO1999058691A3 (en) Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
BR0316039A (pt) Precursor de n-acetilgalactosamina-4-sulfatase, métodos de tratamento usando a enzima e métodos para produção e purificação da enzima
MX9708416A (es) Derivados de piperazino como antagonistas de neuroquininas.
BG105275A (en) Tan-1057 derivatives
BG105014A (en) Ureas and carbamates of n-heterocyclic carboxylic acids and carboxylic acid isosteres
IL143382A0 (en) Human adult astrocytes, their preparation and uses thereof
WO2001083722A3 (en) Enzymes useful for treating and methods for treating mps-vi and cells lines for producing such enzymes recombinantly
IL128028A0 (en) Treatment of psychotic disorders
IL114185A0 (xx)
GB9425071D0 (en) Chondrocyte cell lines
AP2000001963A0 (en) Paroxetine ascorbate.
IL157792A0 (en) Use of thiolutin dioxide and its derivatives in the manufacture of a medicament, and a process for the preparation thereof
ES2180433A1 (es) Empleo de celulas madre hematopoyeticas en la generacion de celulas madre neurales.
TW370564B (en) The process for producing rice Cn/Zn-superoxide dismutase
AP2000001958A0 (en) Paroxetine 10-camphorsulfornate for treatment of CNS disorders.
UA34078A (uk) Спосіб корекції вегетативних порушень при бронхіальній астмі у дітей
UA33498A (uk) Спосіб лікування терапевтично резистентних форм шизофренії

Legal Events

Date Code Title Description
CHPA Change of a particular in the register (except of change of ownership)
AM Amended specification (according sect 146 of patent law)

Free format text: DELETION OF APPLICANT: JOHN M. HENDSTRAND

AM Amended specification (according sect 146 of patent law)

Free format text: ADDITION OF PRIORITY CLAIM: 12.11.1999 US 439923

Effective date: 20070301

AM Amended specification (according sect 146 of patent law)

Free format text: CORRECTION OF THE NAME OF THE INVENTOR FROM JOHN M. HENDSTRAND TO JOHN M. HENSTRAND.

Effective date: 20070322

PE Patent expired

Effective date: 20201108