WO1999058691A3 - 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 Download PDF

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Publication number
WO1999058691A3
WO1999058691A3 PCT/US1999/010102 US9910102W WO9958691A3 WO 1999058691 A3 WO1999058691 A3 WO 1999058691A3 US 9910102 W US9910102 W US 9910102W WO 9958691 A3 WO9958691 A3 WO 9958691A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
iduronidase
recombinant
purifying
deficiencies
Prior art date
Application number
PCT/US1999/010102
Other languages
French (fr)
Other versions
WO1999058691A2 (en
Inventor
Emil D Kakkis
Becky Tanamachi
Original Assignee
Harbor Ucla
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 Harbor Ucla filed Critical Harbor Ucla
Priority to IL13961699A priority Critical patent/IL139616A0/en
Priority to JP2000548482A priority patent/JP2002514429A/en
Priority to CA002328518A priority patent/CA2328518A1/en
Priority to BR9910323-0A priority patent/BR9910323A/en
Priority to EP99924155A priority patent/EP1078075A2/en
Priority to AU40724/99A priority patent/AU4072499A/en
Publication of WO1999058691A2 publication Critical patent/WO1999058691A2/en
Publication of WO1999058691A3 publication Critical patent/WO1999058691A3/en

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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
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Abstract

The present invention provides a recombinant α-L-iduronidase and biologically active fragments and mutants thereof, methods to produce and purify this enzyme as well as methods to treat certain genetic disorders including α-L-iduronidase deficiency and mucopolysaccharidosis I (MPS I).
PCT/US1999/010102 1998-05-13 1999-05-07 Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof WO1999058691A2 (en)

Priority Applications (6)

Application Number Priority Date Filing Date Title
IL13961699A IL139616A0 (en) 1998-05-13 1999-05-07 Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating, diseases caused by deficiencies thereof
JP2000548482A JP2002514429A (en) 1998-05-13 1999-05-07 Recombinant α-L-iduronidase, method for producing and purifying the same, and method for treating diseases caused by deficiency thereof
CA002328518A CA2328518A1 (en) 1998-05-13 1999-05-07 Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
BR9910323-0A BR9910323A (en) 1998-05-13 1999-05-07 Recombinant alpha-l-iduronidase, methods for its production and purification and methods for treating diseases caused by its deficiency
EP99924155A EP1078075A2 (en) 1998-05-13 1999-05-07 Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
AU40724/99A AU4072499A (en) 1998-05-13 1999-05-07 Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US7820998A 1998-05-13 1998-05-13
US17097798A 1998-10-13 1998-10-13
US09/170,977 1998-10-13
US09/078,209 1998-10-13

Publications (2)

Publication Number Publication Date
WO1999058691A2 WO1999058691A2 (en) 1999-11-18
WO1999058691A3 true WO1999058691A3 (en) 2000-02-17

Family

ID=26760239

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1999/010102 WO1999058691A2 (en) 1998-05-13 1999-05-07 Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof

Country Status (8)

Country Link
EP (1) EP1078075A2 (en)
JP (1) JP2002514429A (en)
CN (1) CN1300323A (en)
AU (1) AU4072499A (en)
BR (1) BR9910323A (en)
CA (1) CA2328518A1 (en)
IL (1) IL139616A0 (en)
WO (1) WO1999058691A2 (en)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
PT3108895T (en) 2000-07-18 2018-12-18 Univ Duke Treatment of glycogen storage disease type ii
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
EP2630241B1 (en) * 2010-10-22 2018-10-17 CuRNA, Inc. Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
CN103487414A (en) * 2012-06-14 2014-01-01 北京和信非凡生物技术有限公司 Method, substrate and reagents for alpha-L-iduronidase activity detection
JP6591956B2 (en) * 2013-03-15 2019-10-16 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Compositions and methods for treating MPS1
CA3012195A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
JP2020508289A (en) * 2017-01-31 2020-03-19 レジェンクスバイオ インコーポレーテッド Treatment of mucopolysaccharidosis type I with fully human glycosylated human α-L-iduronidase (IDUA)
AU2018298133A1 (en) 2017-07-06 2020-01-23 The Trustees Of The University Of Pennsylvania AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
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
CN115109790A (en) * 2021-03-23 2022-09-27 北京据德医药科技有限公司 Recombinant a-L-iduronate prase and preparation method thereof
CN114181318B (en) * 2021-11-08 2023-08-01 四川大学 Recombinant adeno-associated virus for tissue-specific expression of IDUA fusion protein penetrating blood brain barrier and application thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993010244A1 (en) * 1991-11-14 1993-05-27 Women's And Children's Hospital Synthetic alpha-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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993010244A1 (en) * 1991-11-14 1993-05-27 Women's And Children's Hospital Synthetic alpha-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

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
CLEMENTS P ET AL: "Human alpha-L-iduronidase. 1. Purification, monoclonal antibody production, native and subunit molecular mass", EUR J BIOCHEM, vol. 152, no. 1, 1 October 1985 (1985-10-01), pages 21 - 28, XP000857400 *
KAKKIS E ET AL: "Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I", BIOCHEM MOL MED, vol. 58, no. 2, August 1996 (1996-08-01), pages 156 - 167, XP000862844 *
KAKKIS E ET AL: "Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells", PROTEIN EXPR PURIF, vol. 5, no. 3, June 1994 (1994-06-01), pages 225 - 232, XP000857380 *
SHULL R ET AL: "Enzyme replacement in a canine model of Hurler syndrome", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA., vol. 91, no. 26, 20 December 1994 (1994-12-20), pages 12937 - 12941, XP002125064 *
UNGER E ET AL: "Recombinant alpha-L-iduronidase: characterization of the purified enzyme and correction of mucopolysaccharidosis type I fibroblasts", BIOCHEM J, vol. 304, 15 November 1994 (1994-11-15), pages 43 - 49, XP000857388 *

Also Published As

Publication number Publication date
CA2328518A1 (en) 1999-11-18
EP1078075A2 (en) 2001-02-28
IL139616A0 (en) 2002-02-10
WO1999058691A2 (en) 1999-11-18
CN1300323A (en) 2001-06-20
AU4072499A (en) 1999-11-29
BR9910323A (en) 2001-01-30
JP2002514429A (en) 2002-05-21

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