DK2325302T3 - Celler der co-udtrykker et sulfatase og et C-formylglycin-genererende enzym og fremgangsmåder og anvendelser deraf - Google Patents

Celler der co-udtrykker et sulfatase og et C-formylglycin-genererende enzym og fremgangsmåder og anvendelser deraf Download PDF

Info

Publication number
DK2325302T3
DK2325302T3 DK10182644.4T DK10182644T DK2325302T3 DK 2325302 T3 DK2325302 T3 DK 2325302T3 DK 10182644 T DK10182644 T DK 10182644T DK 2325302 T3 DK2325302 T3 DK 2325302T3
Authority
DK
Denmark
Prior art keywords
leu
gly
sulfatase
pro
ala
Prior art date
Application number
DK10182644.4T
Other languages
English (en)
Inventor
Figura Kurt Von
Bernhard Schmidt
Thomas Dierks
Michael W Heartlein
Andrea Dr Ballabio
Maria Pia Cosma
Original Assignee
Shire Human Genetic Therapies
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 Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Application granted granted Critical
Publication of DK2325302T3 publication Critical patent/DK2325302T3/da

Links

Classifications

    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • 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
    • A61K38/44—Oxidoreductases (1)
    • 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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A61P15/08—Drugs for genital or sexual disorders; Contraceptives for gonadal disorders or for enhancing fertility, e.g. inducers of ovulation or of spermatogenesis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • 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
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0051—Oxidoreductases (1.) acting on a sulfur group of donors (1.8)
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y108/00—Oxidoreductases acting on sulfur groups as donors (1.8)
    • C12Y108/99—Oxidoreductases acting on sulfur groups as donors (1.8) with other acceptors (1.8.99)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Reproductive Health (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurosurgery (AREA)
  • Endocrinology (AREA)
  • Neurology (AREA)
  • Dermatology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Gynecology & Obstetrics (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)

Claims (27)

1. En celle der co-udtrykker et sulfatase- og et Ca-formylyglycin-genererende enzym (FGE) så at aktiveret sulfatase produceres; hvor cellen omfatter heterologt DNA eller RNA der resulterer i stigende udtryk af den aktiverede sulfatase i forhold til det der ville optræde i fravær af heterologt DNA eller RNA; og hvor FGE'et er et polypeptid med Ca-formylyglycin-genererende aktivitet der: a) har en sekvens valgt fra gruppen af SEQ ID NO. 2, 5, 46, 48, 50, 52, 54, 56, 58, 60, 62, 64, 66, 68, 70, 72, 74, 76, 78, eller aminosyrerne 34-374 af SEQ ID NO. 2; eller b) har mindst 50% sekvensidentitet med SEQ ID No 2; eller c) har én eller flere konservative aminosyremutationer i forhold til et polypeptid som beskrevet i a) eller b) ovenfor; eller d) er et fragment af et polypeptid som beskrevet i en hvilken som helst af a) til c) ovenfor; eller e) er et fusionsprotein af en hvilken som helst af a) til d) ovenfor.
2. Celle ifølge krav 1; hvor FGE'et udtrykkes ved et højere niveau i forhold til det der ville optræde i fraværet af det heterologe DNA eller RNA.
3. Celle ifølge krav 1 eller krav 2; hvor graden af sekvensidentitet i del b) ifølge krav 1 er mindst 75%.
4. Celle ifølge krav 3; hvor graden af sekvensidentitet i del b) ifølge krav 1 er mindst 95%.
5. Celle ifølge krav 1; hvor FGE'et er et polypeptid som beskrevet i del a) deraf.
6. Celle ifølge et hvilken som helst af kravene 1 til 5; hvor FGE'et er et polypeptid der omfatter SEQ ID No 2, eller et fragment deraf med Ca-formylyglycin- genererende aktivitet.
7. Celle ifølge krav 6; hvor FGE'et er et polypeptid der omfatter SEQ ID No 2 eller aminosyrerne 34-374 of SEQ ID NO. 2.
8. Celle ifølge et hvilken som helst af kravene 1 til 5; hvor FGE'et er et polypeptid der har et underdomæne 3 som omfatter mindst en af følgende: (i) et GFR-motiv (ii) et RVXXGG(A)S-motiv (iii) en heptamer indeholdende tre argininer (iv) tre cysteinrester.
9. Celle ifølge et hvilket som helst af de foregående krav; hvor sulfatasen er en eukaryot sulfatase, en pattedyrsulfatase eller en human sulfatase.
10. Celle ifølge et hvilket som helst af de foregående krav; hvor sulfatasen er valgt fra gruppen bestående af Iduronat-2-sulfatase, sulfamidase, N-acetylgalactosamin-6-sulfatase, N-acetylglucosamin-6-Sulfatase, arylsulfatase A, arylsulfatase B, arylsulfatase C, arylsulfatase D, arylsulfatase E, arylsulfatase F, arylsulfatase G, HSulf-1, HSulf-2, HSulf-3, HSulf-4, HSulf-5, eller HSulf-6, eller et fragment deraf med sulfataseaktivitet.
11. Celle ifølge krav 10; hvor sulfatasen er Iduronat 2-sulfatase.
12. Celle ifølge et hvilket som helst af de foregående krav; hvor sulfatasen er exogen i forhold til cellen uden det heterologe DNA eller RNA.
13. Celle ifølge et hvilket som helst af de foregående krav der er en eukaryot celle.
14. Celle ifølge et hvilket som helst af de foregående krav, hvor cellen er blevet transformeret under anvendelse af heterologt DNA eller RNA ifølge krav 1 eller i form af en cellekultur afledt derfra.
15. Celle ifølge et hvilket som helst af de foregående krav der er en primærcelle, eller en sekundærcelle, eller er en celle fra en immortaliseret cellelinje.
16. In Wfro-fremgangsmåde omfattende anvendelse af én eller flere celler ifølge et hvilket som helst af kravene 1 til 15 til at fremstille aktiveret sulfatase.
17. Fremgangsmåde ifølge krav 16 når anvendt til at fremstille sulfatase med en højere andel af aktiveret sulfatase til total sulfatase end det vil være tilfældet for sulfatase produceret fra den ene eller flere celler i fraværet af det heterologe DNA eller RNA.
18. Fremgangsmåde ifølge krav 16 eller 17, hvor sulfatasen er tilvejebragt i en form der kan anvendes i en farmaceutisk sammensætning til behandling af en sulfatasemangel.
19. Fremgangsmåde ifølge et hvilket som helst af kravene 16 til 18 omfattende tilvejebringelse af en farmaceutisk sammensætning der omfatter aktiveret sulfatase og en farmaceutisk acceptabel bærer.
20. Fremgangsmåde ifølge krav 18 eller 19, hvor den farmaceutiske sammensætning er tilvejebragt i en form til administration ad oral, rektal, topisk, nasal, intradermal transdermal eller parenteral vej.
21. Fremgangsmåde ifølge et hvilket som helst af kravene 18 til 20, hvor den farmaceutisk sammensætning er tilvejebragt i en form der er egnet til anvendelse i behandling af sulfatase mangel.
22. Fremgangsmåde ifølge et hvilket som helst af kravene 18 til 21, hvor den farmaceutiske sammensætning er tilvejebragt i en enhedsdosisform der er egnet til anvendelse i behandling af sulfatase mangel.
23. Fremgangsmåde ifølge et hvilket som helst af kravene 16 til 22 omfattende måling af sulfataseaktivitet
24. Celle ifølge et hvilken som helst af kravene 1 til 15, til anvendelse i en fremgangsmåde til behandling af sulfatasemangel.
25. Celle ifølge et hvilken som helst af kravene 1 til 15, til anvendelse i en fremgangsmåde til behandling af sulfatasemangel; hvor sulfatasemanglen er valgt fra gruppen bestående af: multiple sulfatasemangel, mucopolysaccharidose II, mucopolysaccharidose IIIA, mucopolysaccharidose IVA, mucopolysaccharidose VI, mucopolysaccharidose VIII, metachromatisk leukodystrofi, X-bundet recessiv chondrodysplasi punctata 1 og X-bundet ichthyose.
26. Sulfatase til anvendelse i en fremgangsmåde til behandling af sulfatasemangel hos et individ, hvor sulfatasen er er produceret af en celle med øget udtryk af formylglycin-genererende enzym (FGE) til forøgelse af forholdet af aktiv sulfatase til total sulfatase i en mængde der er effektiv til forøgelse af sulfatasens specifikke aktivitet, hvor FGE'et er et polypeptid med Ca-formylglycin-genererende aktivitet der: a) har en sekvens valgt fra gruppen af SEQ ID NO. 2, 5, 46, 48, 50, 52, 54, 56, 58, 60, 62, 64, 66, 68, 70, 72, 74, 76, 78, eller aminosyrerne 34-374 of SEQ ID NO. 2; eller b) har mindst 50% sekvensidentitet med SEQ ID No 2; eller c) har én eller flere konservative aminosyremutationer i forhold til et polypeptid som beskrevet i a) eller b) ovenfor; eller d) er et fragment af et polypeptid som beskrevet i en hvilken som helst af a) til c) ovenfor; eller e) er et fusionsprotein of en hvilken som helst af a) til d) ovenfor.
27. Sulfatasen til anvendelse ifølge krav 26, hvor sulfatasen er Iduronat 2-sulfatase.
DK10182644.4T 2003-02-11 2004-02-10 Celler der co-udtrykker et sulfatase og et C-formylglycin-genererende enzym og fremgangsmåder og anvendelser deraf DK2325302T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US44774703P 2003-02-11 2003-02-11
EP04709824A EP1592786B8 (en) 2003-02-11 2004-02-10 Diagnosis and treatment of multiple sulfatase deficiency and other sulfatase deficiencies using a formylglycine generating enzyme (fge)

Publications (1)

Publication Number Publication Date
DK2325302T3 true DK2325302T3 (da) 2016-03-14

Family

ID=32869644

Family Applications (2)

Application Number Title Priority Date Filing Date
DK10182644.4T DK2325302T3 (da) 2003-02-11 2004-02-10 Celler der co-udtrykker et sulfatase og et C-formylglycin-genererende enzym og fremgangsmåder og anvendelser deraf
DK04709824.9T DK1592786T3 (da) 2003-02-11 2004-02-10 Diagnosticering og behandling af multipel sulfatase-defekt og andet ved anvendelse af et formylglycin-dannende enzym (FGE).

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK04709824.9T DK1592786T3 (da) 2003-02-11 2004-02-10 Diagnosticering og behandling af multipel sulfatase-defekt og andet ved anvendelse af et formylglycin-dannende enzym (FGE).

Country Status (18)

Country Link
US (4) US8227212B2 (da)
EP (3) EP2325302B1 (da)
JP (3) JP5241101B2 (da)
CN (4) CN101444621B (da)
AT (1) ATE534730T1 (da)
AU (2) AU2004210936C1 (da)
CA (1) CA2515708A1 (da)
CY (1) CY1117346T1 (da)
DK (2) DK2325302T3 (da)
ES (3) ES2555056T3 (da)
HK (3) HK1090088A1 (da)
HU (1) HUE027210T2 (da)
MX (2) MX345056B (da)
NZ (4) NZ542267A (da)
PT (1) PT1592786E (da)
SI (1) SI2325302T1 (da)
WO (1) WO2004072275A2 (da)
ZA (1) ZA200506378B (da)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2325302T3 (da) * 2003-02-11 2016-03-14 Shire Human Genetic Therapies Celler der co-udtrykker et sulfatase og et C-formylglycin-genererende enzym og fremgangsmåder og anvendelser deraf
US8741260B2 (en) * 2005-10-07 2014-06-03 Armagen Technologies, Inc. Fusion proteins for delivery of GDNF to the CNS
US8759297B2 (en) 2006-08-18 2014-06-24 Armagen Technologies, Inc. Genetically encoded multifunctional compositions bidirectionally transported between peripheral blood and the cns
US7985783B2 (en) 2006-09-21 2011-07-26 The Regents Of The University Of California Aldehyde tags, uses thereof in site-specific protein modification
WO2009018122A2 (en) 2007-07-27 2009-02-05 Armagen Technologies, Inc. Methods and compositions for increasing alpha-iduronidase activity in the cns
JP2010540534A (ja) 2007-09-28 2010-12-24 イントレキソン コーポレーション 生体治療分子の発現のための治療遺伝子スイッチ構築物およびバイオリアクター、ならびにその使用
CA3112632A1 (en) 2008-01-18 2009-07-23 Biomarin Pharmaceutical Inc. Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
US7722865B2 (en) 2008-01-18 2010-05-25 Biomarin Pharmaceutical Inc. Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
US8729232B2 (en) * 2008-03-27 2014-05-20 The Regents Of The University Of California Aldehyde tags, uses thereof in site-specific protein modification
US9238878B2 (en) 2009-02-17 2016-01-19 Redwood Bioscience, Inc. Aldehyde-tagged protein-based drug carriers and methods of use
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
EP2483407A2 (en) 2009-09-30 2012-08-08 President and Fellows of Harvard College Methods for modulation of autophagy through the modulation of autophagy-enhancing gene products
ES2725200T3 (es) * 2009-10-09 2019-09-20 Armagen Inc Métodos y composiciones para aumentar la actividad de iduronato 2-sulfatasa en el SNC
US8351868B2 (en) * 2009-11-20 2013-01-08 Motorola Solutions, Inc. Radio status indicator
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
AR082319A1 (es) 2010-07-22 2012-11-28 Biomarin Pharm Inc Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos
AU2012205301B2 (en) 2011-01-14 2017-01-05 Redwood Bioscience, Inc. Aldehyde-tagged immunoglobulin polypeptides and method of use thereof
WO2012101671A1 (en) 2011-01-25 2012-08-02 Jcr Pharmaceuticals Co., Ltd. Method for production of human recombinant iduronate 2-sulfatase
US20160145589A1 (en) 2011-06-24 2016-05-26 Green Cross Corporation Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof
KR101158673B1 (ko) 2011-06-24 2012-07-03 주식회사 지씨바이오 재조합 인간 이듀로네이트-2-설파타아제를 포함하는 조성물, 제제 및 이의 제조방법
US8486399B2 (en) 2011-12-02 2013-07-16 Armagen Technologies, Inc. Methods and compositions for increasing arylsulfatase A activity in the CNS
JP6124439B2 (ja) 2011-12-20 2017-05-10 Ｊｃｒファーマ株式会社 ホルミルグリシン残基の分析法
WO2013148323A1 (en) * 2012-03-30 2013-10-03 Shire Human Genetic Therapies Methods of analyzing and preparing protein compositions
EA201491578A1 (ru) 2012-03-30 2015-04-30 Шир Хьюман Дженетик Терапис, Инк. Подкожное введение идуронат-2-сульфатазы
US9150841B2 (en) * 2012-06-29 2015-10-06 Shire Human Genetic Therapies, Inc. Cells for producing recombinant iduronate-2-sulfatase
KR101380740B1 (ko) 2012-06-29 2014-04-11 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 이듀로네이트-2-설파타제의 정제
US20140004097A1 (en) * 2012-06-29 2014-01-02 Shire Human Genetic Therapies, Inc. Method of producing recombinant iduronate-2-sulfatase
WO2014016873A1 (en) 2012-07-26 2014-01-30 Jcr Pharmaceuticals Co., Ltd. Method for production of recombinant human alpha-galactosidase a
JP6692293B2 (ja) * 2013-07-22 2020-05-13 アーマジェン・インコーポレイテッドＡｒｍａｇｅｎ， Ｉｎｃ． Ｃｎｓにおける酵素活性を増大するための方法および組成物
US20170191041A1 (en) * 2014-07-11 2017-07-06 Biostrategies LC Materials and methods for treating disorders associated with sulfatase enzymes
MA41198A (fr) 2014-12-18 2017-10-24 Shire Human Genetic Therapies Essais d'activité enzymatique pour i2s
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
WO2016116966A1 (en) 2015-01-22 2016-07-28 Jcr Pharmaceuticals Co., Ltd. Method for purification of recombinant human alpha-galactosidase a from material containing contaminant host cell proteins
JPWO2016121715A1 (ja) * 2015-01-26 2017-11-02 国立大学法人名古屋大学 肺がん患者の予後を評価するための情報を提供する方法、肺がん患者の予後予測方法、内部標準、抗体、肺がん患者の予後予測装置、予後予測装置のプログラム及び記録媒体
AU2015381699B2 (en) 2015-02-05 2020-07-16 R.P. Scherer Technologies, Llc Activated formylglycine-generating enzymes and methods of producing and using the same
CN109071634A (zh) 2016-04-26 2018-12-21 R.P.谢勒技术有限责任公司 抗体偶联物及其制备和使用方法
WO2018060097A1 (en) * 2016-09-30 2018-04-05 Laboratorios Del Dr. Esteve, S. A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
MX2019007824A (es) 2016-12-28 2019-09-09 Japan Chem Res Preparacion liofilizada.
WO2018136626A1 (en) 2017-01-18 2018-07-26 Visterra, Inc. Antibody molecule-drug conjugates and uses thereof
KR20200058510A (ko) 2017-10-02 2020-05-27 데날리 테라퓨틱스 인크. 효소 대체 요법 효소를 포함하는 융합 단백질
CN109628426B (zh) * 2019-01-02 2023-05-09 山东大学 一种δ4,5-己糖醛酸-2-o-硫酸酯酶及其编码基因与应用以及同类酶的鉴定方法
CN109781997A (zh) * 2019-01-22 2019-05-21 中国人民解放军总医院 用于诊断粘多糖病ⅱ型的生物标志物及其应用
AU2021248643A1 (en) 2020-04-03 2022-10-27 Visterra, Inc. Antibody molecule-drug conjugates and uses thereof
CA3173207A1 (en) * 2020-04-06 2021-10-14 Jacinthe GINGRAS Adeno-associated virus compositions for ids gene transfer and methods of use thereof
CN112630430B (zh) * 2020-11-16 2021-08-27 北京美联泰科生物技术有限公司 一种定量检测uchl-1的试剂盒及其应用

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3307A (en) 1843-10-18 Laben eddy
US307A (en) 1837-07-29 Rqss winans
US3854480A (en) 1969-04-01 1974-12-17 Alza Corp Drug-delivery system
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4675189A (en) 1980-11-18 1987-06-23 Syntex (U.S.A.) Inc. Microencapsulation of water soluble active polypeptides
US4452775A (en) 1982-12-03 1984-06-05 Syntex (U.S.A.) Inc. Cholesterol matrix delivery system for sustained release of macromolecules
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US5225539A (en) 1986-03-27 1993-07-06 Medical Research Council Recombinant altered antibodies and methods of making altered antibodies
US5075109A (en) 1986-10-24 1991-12-24 Southern Research Institute Method of potentiating an immune response
US4748024A (en) 1987-04-06 1988-05-31 Endocon, Inc. Flash flow fused medicinal implants
US5674722A (en) 1987-12-11 1997-10-07 Somatix Therapy Corporation Genetic modification of endothelial cells
CA2345497A1 (en) 1988-10-28 1990-04-28 Genentech, Inc. Growth hormone variants and method for forming growth hormone variants
US5530101A (en) 1988-12-28 1996-06-25 Protein Design Labs, Inc. Humanized immunoglobulins
US5133974A (en) 1989-05-05 1992-07-28 Kv Pharmaceutical Company Extended release pharmaceutical formulations
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5859205A (en) 1989-12-21 1999-01-12 Celltech Limited Humanised antibodies
GB9019812D0 (en) 1990-09-11 1990-10-24 Scotgen Ltd Novel antibodies for treatment and prevention of infection in animals and man
US5641670A (en) 1991-11-05 1997-06-24 Transkaryotic Therapies, Inc. Protein production and protein delivery
US6270989B1 (en) 1991-11-05 2001-08-07 Transkaryotic Therapies, Inc. Protein production and delivery
US5968502A (en) 1991-11-05 1999-10-19 Transkaryotic Therapies, Inc. Protein production and protein delivery
US5932211A (en) * 1991-11-12 1999-08-03 Women's And Children's Hospital Glycosylation variants of iduronate 2-sulfatase
US5407686A (en) 1991-11-27 1995-04-18 Sidmak Laboratories, Inc. Sustained release composition for oral administration of active ingredient
US5645829A (en) 1993-06-18 1997-07-08 Beth Israel Hospital Association Mesothelial cell gene therapy
WO1995024929A2 (en) 1994-03-15 1995-09-21 Brown University Research Foundation Polymeric gene delivery system
AU700369B2 (en) * 1994-03-16 1999-01-07 Regents Of The University Of California, The Treating inflammation via the administration of specific sulfatase enzymes and/or sulfation inhibitor
US5863782A (en) * 1995-04-19 1999-01-26 Women's And Children's Hospital Synthetic mammalian sulphamidase and genetic sequences encoding same
US5643599A (en) 1995-06-07 1997-07-01 President And Fellows Of Harvard College Intracellular delivery of macromolecules
US5736152A (en) 1995-10-27 1998-04-07 Atrix Laboratories, Inc. Non-polymeric sustained release delivery system
US7368531B2 (en) * 1997-03-07 2008-05-06 Human Genome Sciences, Inc. Human secreted proteins
US7083793B2 (en) * 1999-02-26 2006-08-01 Millennium Pharmaceuticals, Inc. Tango 243 polypeptides and uses thereof
AU3382800A (en) * 1999-02-26 2000-09-14 Millennium Pharmaceuticals, Inc. Secreted proteins and uses thereof
US20040010134A1 (en) * 2000-04-12 2004-01-15 Rosen Craig A. Albumin fusion proteins
JP2002017376A (ja) * 1999-07-08 2002-01-22 Herikkusu Kenkyusho:Kk 分泌蛋白質、または膜蛋白質
EP1212343A4 (en) * 1999-09-03 2004-11-03 Human Genome Sciences Inc 52 HUMAN SECRETED PROTEINS
US6780627B1 (en) 2000-01-31 2004-08-24 Millennium Pharmaceuticals, Inc. 22438, 23553, 25278, and 26212 novel human sulfatases
EP1255819A2 (en) * 2000-02-17 2002-11-13 Incyte Genomics, Inc. Human kinases
EP1268541A1 (en) * 2000-03-17 2003-01-02 Human Genome Sciences, Inc. 7 human ovarian and ovarian cancer associated proteins
WO2002052019A2 (en) 2000-12-21 2002-07-04 Millennium Pharmaceuticals, Inc. 22437, a human sulfatase and uses therefor
US20030148920A1 (en) * 2000-12-27 2003-08-07 Steven Rosen Sulfatases and methods of use thereof
US20030147875A1 (en) 2000-12-27 2003-08-07 Steven Rosen Sulfatases and methods of use thereof
AU2002317700A1 (en) * 2001-06-07 2002-12-16 Hemebiotech A/S Production of recombinant human arylsulfatase a
AU2002332648A1 (en) 2001-08-31 2003-03-18 Millennium Pharmaceuticals, Inc. Mid 9002, a human sulfatase family member and uses therefor
DK2325302T3 (da) * 2003-02-11 2016-03-14 Shire Human Genetic Therapies Celler der co-udtrykker et sulfatase og et C-formylglycin-genererende enzym og fremgangsmåder og anvendelser deraf
US8510225B2 (en) 2004-09-01 2013-08-13 Research In Motion Limited Split channel authenticity queries in multi-party dialog
CN105450838B (zh) 2014-09-01 2019-12-24 联想(北京)有限公司 一种信息处理方法及电子设备

Also Published As

Publication number Publication date
CN101444621B (zh) 2013-11-20
MX345056B (es) 2017-01-16
ES2555056T3 (es) 2015-12-28
CN101444621A (zh) 2009-06-03
AU2004210936B2 (en) 2010-05-13
ATE534730T1 (de) 2011-12-15
JP5527854B2 (ja) 2014-06-25
JP2012090630A (ja) 2012-05-17
NZ570201A (en) 2011-03-31
AU2010212261C1 (en) 2012-12-06
JP2014131515A (ja) 2014-07-17
EP2325301A1 (en) 2011-05-25
WO2004072275A3 (en) 2005-04-14
NZ542267A (en) 2008-12-24
EP2325302B1 (en) 2016-01-06
HK1090088A1 (en) 2006-12-15
US8227212B2 (en) 2012-07-24
US20130172403A1 (en) 2013-07-04
AU2010212261A1 (en) 2010-09-02
HK1152337A1 (zh) 2012-02-24
JP2006517412A (ja) 2006-07-27
AU2004210936C1 (en) 2010-12-02
JP5241101B2 (ja) 2013-07-17
ZA200506378B (en) 2006-12-27
DK1592786T3 (da) 2012-02-13
EP1592786A2 (en) 2005-11-09
MXPA05008533A (es) 2005-10-20
AU2004210936A1 (en) 2004-08-26
CN110496230A (zh) 2019-11-26
US20130028881A1 (en) 2013-01-31
NZ603330A (en) 2015-02-27
EP2325302A1 (en) 2011-05-25
CY1117346T1 (el) 2017-04-26
EP1592786B1 (en) 2011-11-23
WO2004072275A2 (en) 2004-08-26
US20040229250A1 (en) 2004-11-18
PT1592786E (pt) 2012-01-19
CN103055306A (zh) 2013-04-24
CN1759176A (zh) 2006-04-12
CN1759176B (zh) 2012-10-03
EP2325301B1 (en) 2015-09-02
CA2515708A1 (en) 2004-08-26
CN103055306B (zh) 2020-10-27
JP6007203B2 (ja) 2016-10-12
ES2566641T3 (es) 2016-04-14
HK1152336A1 (en) 2012-02-24
AU2010212261B2 (en) 2012-04-26
EP2325302A8 (en) 2012-02-22
US20160367703A1 (en) 2016-12-22
ES2380147T3 (es) 2012-05-09
EP1592786B8 (en) 2012-03-21
NZ596420A (en) 2012-11-30
SI2325302T1 (sl) 2016-05-31
HUE027210T2 (en) 2016-10-28

Similar Documents

Publication Publication Date Title
DK2325302T3 (da) Celler der co-udtrykker et sulfatase og et C-formylglycin-genererende enzym og fremgangsmåder og anvendelser deraf
AU2012206984B2 (en) Diagnosis and treatment of multiple sulfatase deficiency and other using a formylglycine generating enzyme (FGE)
DK2968602T3 (da) Syntetisk methylmalonyl-coa-mutase-transgen til behandling af mut-klasse-methylmalonacidæmi (mma)
US6093396A (en) Modified glutamic acid decarboxylase (GAD)
AU755251B2 (en) Compositions and methods for sensitizing and inhibiting growth of human tumor cells
US7906637B2 (en) Compositions and methods for inducing or inhibiting activities of selected human cells
WO2015042281A1 (en) Histone deacetylase compositions and uses thereof