BE2018C038I2 - - Google Patents

Info

Publication number
BE2018C038I2
BE2018C038I2 BE2018C038C BE2018C038C BE2018C038I2 BE 2018C038 I2 BE2018C038 I2 BE 2018C038I2 BE 2018C038 C BE2018C038 C BE 2018C038C BE 2018C038 C BE2018C038 C BE 2018C038C BE 2018C038 I2 BE2018C038 I2 BE 2018C038I2
Authority
BE
Belgium
Prior art keywords
disease
patient
body weight
provides methods
comprises administering
Prior art date
Application number
BE2018C038C
Other languages
English (en)
French (fr)
Original Assignee
Genzyme Corp
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
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=BE2018C038(I2) "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
Publication of BE2018C038I2 publication Critical patent/BE2018C038I2/fr

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/107—Rabbit
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/01—Animal expressing industrially exogenous proteins
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal 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)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Husbandry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Pulmonology (AREA)
BE2018C038C 1998-12-07 2018-09-28 BE2018C038I2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11129198P 1998-12-07 1998-12-07
EP99965162A EP1137762B1 (en) 1998-12-07 1999-12-06 Treatment of pompe's disease

Publications (1)

Publication Number Publication Date
BE2018C038I2 true BE2018C038I2 (es) 2019-03-05

Family

ID=22337648

Family Applications (1)

Application Number Title Priority Date Filing Date
BE2018C038C BE2018C038I2 (es) 1998-12-07 2018-09-28

Country Status (16)

Country Link
US (7) US20030007963A1 (es)
EP (2) EP2020438B1 (es)
JP (7) JP4990434B2 (es)
KR (1) KR20010101131A (es)
AT (1) ATE410506T1 (es)
AU (1) AU3113700A (es)
BE (1) BE2018C038I2 (es)
CA (1) CA2353522A1 (es)
CY (2) CY1121113T1 (es)
DE (2) DE122009000018I1 (es)
DK (2) DK2020438T3 (es)
ES (2) ES2312222T3 (es)
LU (1) LU91544I2 (es)
NL (1) NL300382I2 (es)
PT (2) PT2020438T (es)
WO (1) WO2000034451A1 (es)

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DK2020438T3 (en) * 1998-12-07 2018-07-23 Genzyme Corp Treatment of Pompe disease
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SI3449934T1 (sl) 2000-07-18 2020-09-30 Duke University Zdravljenje bolezni kopičenja glikogena tip II
AU2016256663A1 (en) * 2000-07-18 2016-11-24 Duke University Treatment of clycogen storage disease type II
EP1782825B1 (en) * 2000-07-18 2014-09-10 Duke University Treatment of glycogen storage diseases type II
US7723296B2 (en) * 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
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US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
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US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
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AU2003224880A1 (en) 2002-04-05 2003-10-27 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
WO2004064750A2 (en) * 2003-01-22 2004-08-05 Duke University Improved constructs for expressing lysosomal polypeptides
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US20090297496A1 (en) * 2005-09-08 2009-12-03 Childrens Hospital Medical Center Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases
WO2008063511A2 (en) * 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
JP5627571B2 (ja) 2008-05-07 2014-11-19 バイオマリン ファーマシューティカル インコーポレイテッド リソソーム標的化ペプチドおよびその使用
DK2318037T3 (en) 2008-07-08 2015-05-04 Univ Duke Method for treating a glycogen storage-DISEASE
EP2475376B1 (en) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
US8536148B2 (en) 2009-09-04 2013-09-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Disabling autophagy as a treatment for lysosomal storage diseases
PT3205351T (pt) 2010-04-23 2023-06-28 Alexion Pharma Inc Enzima de doença de armazenamento lisossomal
EP2588130B1 (en) 2010-06-25 2016-08-17 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
IL284599B2 (en) 2010-06-25 2024-02-01 Shire Human Genetic Therapies Methods and preparations for administration of iduronate-2-sulfatase to the central nervous system
RU2012154576A (ru) 2010-06-25 2014-07-27 Шир Хьюман Дженетик Терапис, Инк. Способы и композиции для доставки к цнс гепаран-n-сульфатазы
ES2643015T3 (es) 2010-06-25 2017-11-21 Shire Human Genetic Therapies, Inc. Tratamiento del Síndrome de Sanfilippo Tipo B
PT3626258T (pt) 2010-06-25 2021-10-19 Shire Human Genetic Therapies Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase
IL291556B2 (en) 2010-06-25 2024-02-01 Shire Human Genetic Therapies Methods and compositions for administration of arylsulfatase A to the central nervous system
EP3650039A1 (en) 2010-09-09 2020-05-13 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
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BR112013026976A2 (pt) * 2011-04-22 2019-09-24 Genzyme Corp alfa glucosidade ácida modificada com processamento acelerado
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KR20210062731A (ko) 2012-03-07 2021-05-31 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
CN105189542A (zh) 2013-02-20 2015-12-23 瓦莱里昂治疗有限责任公司 用于治疗庞贝氏症的方法和组合物
EP3193942B1 (en) 2014-08-11 2020-03-25 Shire Human Genetic Therapies, Inc. Lysosomal targeting and uses thereof
CA2961762C (en) 2014-09-30 2024-03-05 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
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Also Published As

Publication number Publication date
ATE410506T1 (de) 2008-10-15
JP2017066160A (ja) 2017-04-06
US20130039901A1 (en) 2013-02-14
US20080206223A1 (en) 2008-08-28
DE69939705D1 (de) 2008-11-20
AU3113700A (en) 2000-06-26
JP2018115200A (ja) 2018-07-26
JP2012224643A (ja) 2012-11-15
CY2018020I2 (el) 2019-07-10
EP1137762B1 (en) 2008-10-08
US20100092449A1 (en) 2010-04-15
US7655226B2 (en) 2010-02-02
US20160243203A1 (en) 2016-08-25
CY2018020I1 (el) 2019-07-10
ES2312222T3 (es) 2009-02-16
ES2677343T3 (es) 2018-08-01
EP1137762A4 (en) 2005-01-12
JP2019172685A (ja) 2019-10-10
JP2015134836A (ja) 2015-07-27
DK1137762T3 (da) 2009-02-02
PT2020438T (pt) 2018-07-13
JP2009161562A (ja) 2009-07-23
US20030007963A1 (en) 2003-01-09
CA2353522A1 (en) 2000-06-15
NL300382I1 (nl) 2009-06-02
EP2020438B1 (en) 2018-04-11
LU91544I2 (fr) 2009-05-25
JP2002531581A (ja) 2002-09-24
CY1121113T1 (el) 2019-07-10
EP2020438A1 (en) 2009-02-04
PT1137762E (pt) 2009-01-14
JP4990434B2 (ja) 2012-08-01
US20040081645A1 (en) 2004-04-29
US7351410B2 (en) 2008-04-01
KR20010101131A (ko) 2001-11-14
DE122009000018I1 (de) 2009-09-17
DK2020438T3 (en) 2018-07-23
NL300382I2 (en) 2009-12-01
WO2000034451A1 (en) 2000-06-15
EP1137762A1 (en) 2001-10-04
US20140037611A1 (en) 2014-02-06

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