DE69939705D1 - Behandlung der pompeschen krankheit - Google Patents

Behandlung der pompeschen krankheit

Info

Publication number
DE69939705D1
DE69939705D1 DE69939705T DE69939705T DE69939705D1 DE 69939705 D1 DE69939705 D1 DE 69939705D1 DE 69939705 T DE69939705 T DE 69939705T DE 69939705 T DE69939705 T DE 69939705T DE 69939705 D1 DE69939705 D1 DE 69939705D1
Authority
DE
Germany
Prior art keywords
disease
pompet
treatment
patient
body weight
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.)
Expired - Lifetime
Application number
DE69939705T
Other languages
English (en)
Inventor
Bree Johannes Brenardus Van
Edna Henriette Venneker
David P Meeker
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.)
Genzyme Corp
Original Assignee
Genzyme Corp
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Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=22337648&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DE69939705(D1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Application granted granted Critical
Publication of DE69939705D1 publication Critical patent/DE69939705D1/de
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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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
    • 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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276Knock-out vertebrates
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278Knock-in vertebrates, e.g. humanised vertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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)
    • C12N9/2405Glucanases
    • C12N9/2408Glucanases acting on alpha -1,4-glucosidic bonds
    • 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)
    • C12Y302/0102Alpha-glucosidase (3.2.1.20)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00Modified animals
    • A01K2207/15Humanized animals
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/107Rabbit
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/01Animal expressing industrially exogenous proteins
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Environmental Sciences (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Husbandry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Obesity (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Cardiology (AREA)
  • Dermatology (AREA)
  • Endocrinology (AREA)
DE69939705T 1998-12-07 1999-12-06 Behandlung der pompeschen krankheit Expired - Lifetime DE69939705D1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11129198P 1998-12-07 1998-12-07
PCT/US1999/029042 WO2000034451A1 (en) 1998-12-07 1999-12-06 Treatment of pompe's disease

Publications (1)

Publication Number Publication Date
DE69939705D1 true DE69939705D1 (de) 2008-11-20

Family

ID=22337648

Family Applications (2)

Application Number Title Priority Date Filing Date
DE122009000018C Pending DE122009000018I1 (de) 1998-12-07 1999-12-06 Behandlung der pompeschen krankheit
DE69939705T Expired - Lifetime DE69939705D1 (de) 1998-12-07 1999-12-06 Behandlung der pompeschen krankheit

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DE122009000018C Pending DE122009000018I1 (de) 1998-12-07 1999-12-06 Behandlung der pompeschen krankheit

Country Status (16)

Country Link
US (7) US20030007963A1 (de)
EP (2) EP2020438B1 (de)
JP (7) JP4990434B2 (de)
KR (1) KR20010101131A (de)
AT (1) ATE410506T1 (de)
AU (1) AU3113700A (de)
BE (1) BE2018C038I2 (de)
CA (1) CA2353522A1 (de)
CY (2) CY1121113T1 (de)
DE (2) DE122009000018I1 (de)
DK (2) DK1137762T3 (de)
ES (2) ES2677343T3 (de)
LU (1) LU91544I2 (de)
NL (1) NL300382I2 (de)
PT (2) PT2020438T (de)
WO (1) WO2000034451A1 (de)

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BR0108077A (pt) * 2000-02-04 2002-10-22 Children S Hospital Res Founda Processo para reduzir placas ateroscleróticas em um mamìfero, método para tratamento da aterosclerose em um mamìfero, composição, métodos para prover proteìna ou polipeptìdeo hidrolisante de lipìdeo biologicamente ativo ou mistura deles, para células de um mamìfero tendo deficiência na proteìna ou polipeptìdeo hidrolisante de lipìdeo biologicamente ativo , para prover lipase de ácido lisossomal biologicamente ativa para células de um mamìfero tendo deficiência de lipase de ácido lisossomal biologicamente ativa, para prover lipase de ácido lisossoal biologicamente ativa para células de um mamìfero com aterosclerose, para tratamento da doença de wolman em um mamìfero, para tratamento de doença de armazenagem de colesteril éster em um mamìfero, e, para tratamento da aterosclerose em um mamìfero
EP1782825B1 (de) * 2000-07-18 2014-09-10 Duke University Behandlung der Glykogenspeicherkrankheit typ II
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ATE465250T1 (de) * 2004-02-10 2010-05-15 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
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AU2007322123A1 (en) * 2006-11-13 2008-05-29 Biomarin Pharmaceutical Inc. Methods for treating Pompe disease
EP2279210B1 (de) 2008-05-07 2017-04-12 BioMarin Pharmaceutical Inc. Auf lysosomen gerichtete peptide und ihre verwendung
SI2318037T1 (sl) 2008-07-08 2015-07-31 Duke University Metoda za zdravljenje bolezni glikogenoze
ES2569514T3 (es) 2009-06-17 2016-05-11 Biomarin Pharmaceutical Inc. Formulaciones para enzimas lisosómicas
WO2011028941A2 (en) 2009-09-04 2011-03-10 The United States Of America, As Represented By The Secretary Department Of Health & Human Services Disabling autophagy as a treatment for lysosomal storage diseases
SI2561069T1 (sl) 2010-04-23 2017-07-31 Alexion Pharmaceuticals, Inc. Encim za bolezen lizosomskega shranjevanja
MX2013000320A (es) 2010-06-25 2013-06-05 Shire Human Genetic Therapies Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa.
NZ702808A (en) 2010-06-25 2016-08-26 Shire Human Genetic Therapies Methods and compositions for cns delivery of iduronate-2-sulfatase
EP3964230A1 (de) 2010-06-25 2022-03-09 Shire Human Genetic Therapies, Inc. Verfahren und zusammensetzungen zur abgabe der arylsulfatase a in das zns
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KR20150038636A (ko) 2010-09-09 2015-04-08 시나게바 바이오파르마, 코포레이션 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도
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MX2018011951A (es) 2016-03-30 2019-02-13 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p.
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KR20240001721A (ko) 2016-09-12 2024-01-03 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
EP3293260A1 (de) 2016-09-12 2018-03-14 Genethon Saure alpha-glucosidase-varianten und verwendungen davon
EP3293203A1 (de) 2016-09-12 2018-03-14 Genethon Saure alpha-glucosidase-varianten und verwendungen davon
JP2022540632A (ja) 2019-07-09 2022-09-16 ジェネトン 糖原病(gsd)の処置

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EP1137762A1 (de) 2001-10-04
JP2002531581A (ja) 2002-09-24
ES2677343T3 (es) 2018-08-01
CY2018020I2 (el) 2019-07-10
EP1137762B1 (de) 2008-10-08
ES2312222T3 (es) 2009-02-16
US20100092449A1 (en) 2010-04-15
EP2020438A1 (de) 2009-02-04
KR20010101131A (ko) 2001-11-14
JP2012224643A (ja) 2012-11-15
JP2015134836A (ja) 2015-07-27
US7655226B2 (en) 2010-02-02
AU3113700A (en) 2000-06-26
DK1137762T3 (da) 2009-02-02
CA2353522A1 (en) 2000-06-15
NL300382I1 (nl) 2009-06-02
DK2020438T3 (en) 2018-07-23
JP2017066160A (ja) 2017-04-06
US20040081645A1 (en) 2004-04-29
PT2020438T (pt) 2018-07-13
WO2000034451A1 (en) 2000-06-15
EP1137762A4 (de) 2005-01-12
JP2019172685A (ja) 2019-10-10
DE122009000018I1 (de) 2009-09-17
ATE410506T1 (de) 2008-10-15
US20140037611A1 (en) 2014-02-06
JP2018115200A (ja) 2018-07-26
CY1121113T1 (el) 2019-07-10
JP4990434B2 (ja) 2012-08-01
US20080206223A1 (en) 2008-08-28
LU91544I2 (fr) 2009-05-25
US7351410B2 (en) 2008-04-01
PT1137762E (pt) 2009-01-14
BE2018C038I2 (de) 2019-03-05
EP2020438B1 (de) 2018-04-11
NL300382I2 (en) 2009-12-01
US20030007963A1 (en) 2003-01-09
US20130039901A1 (en) 2013-02-14
JP2009161562A (ja) 2009-07-23
US20160243203A1 (en) 2016-08-25

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