NO991225L - Terapi for <alfa>-galaktosidase A mangel - Google Patents

Terapi for <alfa>-galaktosidase A mangel

Info

Publication number
NO991225L
NO991225L NO991225A NO991225A NO991225L NO 991225 L NO991225 L NO 991225L NO 991225 A NO991225 A NO 991225A NO 991225 A NO991225 A NO 991225A NO 991225 L NO991225 L NO 991225L
Authority
NO
Norway
Prior art keywords
galactosidase
alfa
therapy
deficiency
sample
Prior art date
Application number
NO991225A
Other languages
English (en)
Other versions
NO328443B1 (no
NO991225D0 (no
Inventor
Richard F Selden
Marianne Borowski
Francis P Gillespie
Carol M Kinoshita
Douglas A Treco
Melanie D Williams
Original Assignee
Transkaryotic Therapies 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 Transkaryotic Therapies Inc filed Critical Transkaryotic Therapies Inc
Publication of NO991225D0 publication Critical patent/NO991225D0/no
Publication of NO991225L publication Critical patent/NO991225L/no
Publication of NO328443B1 publication Critical patent/NO328443B1/no

Links

Classifications

    • 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
    • A61P1/14Prodigestives, e.g. acids, enzymes, appetite stimulants, antidyspeptics, tonics, antiflatulents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/04Centrally acting analgesics, e.g. opioids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575Hormones
    • C07K14/61Growth hormones [GH] (Somatotropin)
    • 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)
    • C12N9/2465Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
NO19991225A 1996-09-13 1999-03-12 DNA-molekyl, ekspresjonskonstruksjon, dyrket human celle, kloncellestamme, kloncellelinje, protein, fremgangsmate for fremstilling av glykosylert human alfa-galaktosidase A og human alfa-galaktosidase A samt anvendelse av en human celle som er genetisk modifisert. NO328443B1 (no)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US71261496A 1996-09-13 1996-09-13
PCT/US1997/016603 WO1998011206A2 (en) 1996-09-13 1997-09-12 THERAPY FOR α-GALACTOSIDASE A DEFICIENCY

Publications (3)

Publication Number Publication Date
NO991225D0 NO991225D0 (no) 1999-03-12
NO991225L true NO991225L (no) 1999-05-10
NO328443B1 NO328443B1 (no) 2010-02-22

Family

ID=24862863

Family Applications (4)

Application Number Title Priority Date Filing Date
NO19991225A NO328443B1 (no) 1996-09-13 1999-03-12 DNA-molekyl, ekspresjonskonstruksjon, dyrket human celle, kloncellestamme, kloncellelinje, protein, fremgangsmate for fremstilling av glykosylert human alfa-galaktosidase A og human alfa-galaktosidase A samt anvendelse av en human celle som er genetisk modifisert.
NO20090583A NO330687B1 (no) 1996-09-13 2009-02-06 Fremgangsmate for rensing av human alfa-galaktosidase A
NO20110496A NO340408B1 (no) 1996-09-13 2011-03-31 Fremgangsmåte for å produsere humant alfa-galaktosidase A, og farmasøytisk preparat
NO20141168A NO20141168L (no) 1996-09-13 2014-09-29 Fremgangsmåte for rensing av human alfa-galaktosidase A fra en prøve

Family Applications After (3)

Application Number Title Priority Date Filing Date
NO20090583A NO330687B1 (no) 1996-09-13 2009-02-06 Fremgangsmate for rensing av human alfa-galaktosidase A
NO20110496A NO340408B1 (no) 1996-09-13 2011-03-31 Fremgangsmåte for å produsere humant alfa-galaktosidase A, og farmasøytisk preparat
NO20141168A NO20141168L (no) 1996-09-13 2014-09-29 Fremgangsmåte for rensing av human alfa-galaktosidase A fra en prøve

Country Status (21)

Country Link
EP (4) EP1538202B1 (no)
JP (7) JP4001925B2 (no)
KR (2) KR100547402B1 (no)
CN (1) CN1268741C (no)
AT (1) ATE286120T1 (no)
AU (1) AU4424497A (no)
CA (1) CA2265464C (no)
DE (1) DE69732129T2 (no)
DK (2) DK1538202T3 (no)
ES (3) ES2458292T3 (no)
HK (3) HK1022173A1 (no)
HU (2) HU230275B1 (no)
IL (2) IL128960A (no)
MX (1) MX340738B (no)
NO (4) NO328443B1 (no)
NZ (2) NZ334721A (no)
PL (1) PL190041B1 (no)
PT (1) PT1538202E (no)
RU (1) RU2179034C2 (no)
TW (1) TW585919B (no)
WO (1) WO1998011206A2 (no)

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US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
AU2016250357B2 (en) * 1999-03-11 2018-11-01 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
AU2012241170B2 (en) * 1999-03-11 2016-07-28 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
AU2004242550B2 (en) * 1999-03-11 2008-04-03 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
WO2002064799A2 (en) 1999-09-28 2002-08-22 Transkaryotic Therapies, Inc. Optimized messenger rna
EP2275559A3 (en) * 1999-09-28 2011-03-23 Shire Human Genetic Therapies, Inc. Optimized messenger RNA
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US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7138262B1 (en) * 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
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AU2003228722B2 (en) 2002-04-25 2009-06-25 Takeda Pharmaceutical Company Limited Treatment of alpha-galactosidase A deficiency
EP1587923B1 (en) * 2003-01-22 2011-08-24 Duke University Improved constructs for expressing lysosomal polypeptides
US7935336B2 (en) 2005-11-18 2011-05-03 Tokyo Metropolitan Organization For Medical Research Highly functional enzyme having α-galactosidase activity
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
JP5364382B2 (ja) 2006-02-07 2013-12-11 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 遊離チオール部分を有するタンパク質の安定化された組成物
ES2697502T3 (es) 2006-02-09 2019-01-24 Genzyme Corp Administración intraventricular lenta
WO2008143354A1 (ja) * 2007-05-18 2008-11-27 Tokyo Metropolitan Organization For Medical Research 酵素補充療法用医薬組成物
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RU2733466C2 (ru) 2009-07-28 2020-10-01 Шайр Хьюман Дженетик Терапиз Композиции и способы для лечения болезни гоше
CA2796607C (en) 2010-04-23 2019-09-10 Synageva Biopharma Corp Lysosomal storage disease enzyme
WO2012012461A2 (en) 2010-07-19 2012-01-26 Shire Human Genetic Therapies, Inc. Mannose receptor c type 1 (mrc1) codon optimized cell line and uses thereof
AU2011314293B2 (en) 2010-09-09 2015-06-04 Alexion Pharmaceuticals, Inc. Use of Lysosomal Acid Lipase for treating Lysosomal Acid Lipase Deficiency in patients
KR20140006037A (ko) 2011-02-15 2014-01-15 시나게바 바이오파르마, 코포레이션 리소좀의 산 리파제 결핍을 치료하는 방법들
SI2773438T2 (sl) * 2011-11-02 2022-05-31 F.Hoffmann-La Roche Ag Izpiralna kromatografija s preobremenitvijo
US9623090B2 (en) 2012-03-02 2017-04-18 Shire Human Genetic Therapies, Inc. Compositions and methods for treating type III gaucher disease
BR112014032919B1 (pt) * 2012-06-29 2023-03-28 Universitat De Barcelona Conjugado e seu método de produção, lipossomas, uso e método de produção dos mesmos, bem como composição farmacêutica
WO2014016873A1 (en) * 2012-07-26 2014-01-30 Jcr Pharmaceuticals Co., Ltd. Method for production of recombinant human alpha-galactosidase a
JP6226435B2 (ja) * 2012-07-26 2017-11-08 Jcrファーマ株式会社 組換えヒトα−ガラクトシダーゼAの製造方法
WO2014066471A1 (en) 2012-10-24 2014-05-01 Genzyme Corporation Elution of biomolecules from multi-modal resins using mes and mops as mobile phase modifiers
TWI793159B (zh) 2013-10-23 2023-02-21 美商健臻公司 重組醣蛋白及其用途
EP4234699A1 (en) 2014-12-22 2023-08-30 Codexis, Inc. Human alpha-galactosidase variants
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
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Also Published As

Publication number Publication date
PL332188A1 (en) 1999-08-30
DE69732129D1 (de) 2005-02-03
AU4424497A (en) 1998-04-02
IL184637A (en) 2015-07-30
JP2007289201A (ja) 2007-11-08
NO328443B1 (no) 2010-02-22
KR20050084473A (ko) 2005-08-26
JP2012019793A (ja) 2012-02-02
JP4313381B2 (ja) 2009-08-12
JP5590788B2 (ja) 2014-09-17
ES2234032T3 (es) 2005-06-16
HU227189B1 (en) 2010-10-28
CA2265464C (en) 2007-06-26
WO1998011206A2 (en) 1998-03-19
EP2327775A2 (en) 2011-06-01
JP2009102321A (ja) 2009-05-14
DK1538202T3 (en) 2014-03-24
JP2001504324A (ja) 2001-04-03
NO991225D0 (no) 1999-03-12
ES2458292T3 (es) 2014-04-30
HK1022173A1 (en) 2000-07-28
NZ506214A (en) 2002-11-26
JP4313405B2 (ja) 2009-08-12
JP2007000145A (ja) 2007-01-11
EP1538202A2 (en) 2005-06-08
KR20000036078A (ko) 2000-06-26
JP2009060918A (ja) 2009-03-26
DK2374876T3 (en) 2016-05-30
HU1000445D0 (en) 2010-11-29
NO20110496L (no) 1999-05-10
WO1998011206A3 (en) 1998-08-13
MX340738B (es) 2016-07-22
HK1074058A1 (en) 2005-10-28
CN1268741C (zh) 2006-08-09
TW585919B (en) 2004-05-01
NO330687B1 (no) 2011-06-06
EP2327775A3 (en) 2012-10-24
EP0935651A2 (en) 1999-08-18
EP2374876A3 (en) 2012-10-24
NO20090583L (no) 1999-05-10
PT1538202E (pt) 2014-04-22
EP1538202A3 (en) 2007-05-30
NO20141168L (no) 1999-05-10
JP4001925B2 (ja) 2007-10-31
ES2581828T3 (es) 2016-09-07
RU2179034C2 (ru) 2002-02-10
CN1230220A (zh) 1999-09-29
PL190041B1 (pl) 2005-10-31
IL184637A0 (en) 2009-09-22
CA2265464A1 (en) 1998-03-19
HUP9904666A3 (en) 2001-09-28
IL128960A0 (en) 2000-02-17
EP2374876B1 (en) 2016-04-13
NO340408B1 (no) 2017-04-18
NZ334721A (en) 2001-01-26
HK1162580A1 (zh) 2012-08-31
KR100547402B1 (ko) 2006-02-01
JP2015044830A (ja) 2015-03-12
DE69732129T2 (de) 2005-12-08
HU9904666A (en) 2000-05-28
HU230275B1 (hu) 2015-11-30
IL128960A (en) 2007-10-31
EP2374876A2 (en) 2011-10-12
ATE286120T1 (de) 2005-01-15
EP1538202B1 (en) 2014-01-22
EP0935651B1 (en) 2004-12-29

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