AU2002228991A1 - Methods for treating diseases caused by deficiencies of alpha-l-iduronidase - Google Patents

Methods for treating diseases caused by deficiencies of alpha-l-iduronidase

Info

Publication number
AU2002228991A1
AU2002228991A1 AU2002228991A AU2899102A AU2002228991A1 AU 2002228991 A1 AU2002228991 A1 AU 2002228991A1 AU 2002228991 A AU2002228991 A AU 2002228991A AU 2899102 A AU2899102 A AU 2899102A AU 2002228991 A1 AU2002228991 A1 AU 2002228991A1
Authority
AU
Australia
Prior art keywords
iduronidase
deficiencies
alpha
methods
diseases caused
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2002228991A
Inventor
Emil D. Kakkis
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Biomarin Pharmaceutical Inc
Original Assignee
Biomarin Pharmaceutical Inc
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 Pharmaceutical Inc filed Critical Biomarin Pharmaceutical Inc
Publication of AU2002228991A1 publication Critical patent/AU2002228991A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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)
    • 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
    • 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
    • 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
    • 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.)
    • 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
    • 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
AU2002228991A 2000-11-09 2001-11-09 Methods for treating diseases caused by deficiencies of alpha-l-iduronidase Abandoned AU2002228991A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US09/711,205 2000-11-09
US09/711,205 US6585971B1 (en) 1999-11-12 2000-11-09 Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
PCT/US2001/047835 WO2002038171A2 (en) 2000-11-09 2001-11-09 METHODS FOR TREATING DISEASES CAUSED BY DEFICIENCIES OF α-L-IDURONIDASE

Publications (1)

Publication Number Publication Date
AU2002228991A1 true AU2002228991A1 (en) 2002-05-21

Family

ID=24857171

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2002228991A Abandoned AU2002228991A1 (en) 2000-11-09 2001-11-09 Methods for treating diseases caused by deficiencies of alpha-l-iduronidase

Country Status (3)

Country Link
US (4) US6585971B1 (en)
AU (1) AU2002228991A1 (en)
WO (1) WO2002038171A2 (en)

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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
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
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
WO2006002283A1 (en) * 2004-06-21 2006-01-05 Medtronic, Inc. Medical systems and methods for delivering compositions to cells
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
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
MXPA03011987A (en) 2003-12-19 2005-06-23 Osio Sancho Alberto Method for treating presbyopia by inducing changes in the corneal power and physiology.
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
US8500720B2 (en) 2005-05-09 2013-08-06 Medtronic, Inc Method and apparatus for treatment of cardiac disorders
JP2009508494A (en) 2005-09-16 2009-03-05 ラプトール ファーマシューティカル インコーポレイテッド Compositions comprising receptor-binding protein (RAP) variants specific for proteins comprising CR and uses thereof
US8741260B2 (en) * 2005-10-07 2014-06-03 Armagen Technologies, Inc. Fusion proteins for delivery of GDNF to the CNS
JP5959795B2 (en) 2006-08-18 2016-08-02 アーメイゲン・テクノロジーズ・インコーポレイテッドＡｒｍａｇｅｎ Ｔｅｃｈｎｏｌｏｇｉｅｓ， Ｉｎｃ． Substances for blood-brain barrier delivery
BRPI0809234A2 (en) * 2007-03-21 2014-09-02 Raptor Pharmaceutical Inc CYCLIC RECEIVER-ASSOCIATED PROTEIN (RAP) PEPTIDES
WO2009018122A2 (en) * 2007-07-27 2009-02-05 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the cns
DK2279210T3 (en) 2008-05-07 2017-07-24 Biomarin Pharm Inc Lysosomal targeting peptides and uses thereof
WO2010108048A2 (en) 2009-03-18 2010-09-23 Armagen Technologies, Inc. Compositions and methods for blood-brain barrier delivery of igg-decoy receptor fusion proteins
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
PT2485761T (en) * 2009-10-09 2019-05-30 Armagen Inc Methods and compositions for increasing iduronate 2-sulfatase activity in the cns
US8609088B2 (en) * 2011-05-10 2013-12-17 Regents Of The University Of Minnesota Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
JP6266529B2 (en) 2011-12-02 2018-01-24 アーマジェン・インコーポレイテッドＡｒｍａｇｅｎ， Ｉｎｃ． Methods and compositions for increasing arylsulfatase A activity in the CNS
WO2014026163A2 (en) 2012-08-10 2014-02-13 Osio Corporation d/b/a Yolia Health Contact lens use in the treatment of an ophthalmologic condition
DK2958989T3 (en) 2013-02-22 2019-03-25 Genzyme Corp MICRO-CARRIER PERFUSION CULTURE PROCEDURES AND APPLICATIONS THEREOF
SG11201506343QA (en) 2013-02-22 2015-09-29 Genzyme Corp Microcarrier perfusion culturing methods and uses thereof
JP2016523835A (en) 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ Adeno-associated virus-mediated gene transfer into the central nervous system
TW202246486A (en) 2014-06-09 2022-12-01 美商健臻公司 Seed train processes and uses thereof
US10538589B2 (en) 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
MA44874A (en) 2016-04-15 2019-03-13 Univ Pennsylvania GENE THERAPY TO TREAT MUCOPOLYSACCHARIDOSIS TYPE II
MA47436A (en) * 2017-01-31 2019-12-11 Regenxbio Inc TREATMENT OF MUCOPOLYSACCHARIDOSIS I WITH FULLY HUMAN ALPHA-L-IDURONIDASE GLYCOSYLATE (IDUA)
SG11202002457RA (en) 2017-09-22 2020-04-29 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
CN107699590A (en) * 2017-10-13 2018-02-16 成都中医药大学 A kind of method of Prepare restructuring people α L iduronases

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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
WO1993010244A1 (en) 1991-11-14 1993-05-27 Women's And Children's Hospital 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
WO1997010353A1 (en) 1995-09-14 1997-03-20 Virginia Tech Intellectual Property, Inc. Production of lysosomal enzymes in plant-based expression systems
EP1029548B1 (en) * 1997-10-15 2007-04-04 Asahi Kasei Pharma Corporation Method for keeping the quality of aqueous parenteral solution of thrombomodulin in storage and distribution
GB9807464D0 (en) 1998-04-07 1998-06-10 Pharming Bv Purification of human acid µ-glucosidase
EP1078075A2 (en) 1998-05-13 2001-02-28 Harbor-Ucla Research and Education Institute Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6426208B1 (en) * 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute 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
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues

Also Published As

Publication number Publication date
US7354576B2 (en) 2008-04-08
WO2002038171A3 (en) 2003-04-24
US20050260185A1 (en) 2005-11-24
US20050281804A1 (en) 2005-12-22
WO2002038171A9 (en) 2004-05-13
US6585971B1 (en) 2003-07-01
US6858206B2 (en) 2005-02-22
WO2002038171A2 (en) 2002-05-16
US20020164758A1 (en) 2002-11-07

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