DK0845939T3 - Lysosomale proteiner produceret i mælk fra ikke-humane transgene dyr - Google Patents

Lysosomale proteiner produceret i mælk fra ikke-humane transgene dyr

Info

Publication number
DK0845939T3
DK0845939T3 DK96928405T DK96928405T DK0845939T3 DK 0845939 T3 DK0845939 T3 DK 0845939T3 DK 96928405 T DK96928405 T DK 96928405T DK 96928405 T DK96928405 T DK 96928405T DK 0845939 T3 DK0845939 T3 DK 0845939T3
Authority
DK
Denmark
Prior art keywords
milk
lysosomal proteins
transgenic animals
human transgenic
proteins produced
Prior art date
Application number
DK96928405T
Other languages
English (en)
Inventor
Arnold J J Reuser
Der Ploeg Ans T Van
Frank R Pieper
Martin Ph Verbeet
Original Assignee
Genzyme Corp
Univ Erasmus
Academic Hospital Rotterdam
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=21697877&utm_source=***_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK0845939(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp, Univ Erasmus, Academic Hospital Rotterdam filed Critical Genzyme Corp
Application granted granted Critical
Publication of DK0845939T3 publication Critical patent/DK0845939T3/da

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    • 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
    • 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
    • A23FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23CDAIRY PRODUCTS, e.g. MILK, BUTTER OR CHEESE; MILK OR CHEESE SUBSTITUTES; MAKING THEREOF
    • A23C9/00Milk preparations; Milk powder or milk powder preparations
    • A23C9/20Dietetic milk products not covered by groups A23C9/12 - A23C9/18
    • 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
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4732Casein
    • CCHEMISTRY; METALLURGY
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    • 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
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • 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
    • A23FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23CDAIRY PRODUCTS, e.g. MILK, BUTTER OR CHEESE; MILK OR CHEESE SUBSTITUTES; MAKING THEREOF
    • A23C2230/00Aspects relating to animal feed or genotype
    • A23C2230/05Milk or milk products from transgenic animals
    • AHUMAN NECESSITIES
    • A23FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23CDAIRY PRODUCTS, e.g. MILK, BUTTER OR CHEESE; MILK OR CHEESE SUBSTITUTES; MAKING THEREOF
    • A23C2230/00Aspects relating to animal feed or genotype
    • A23C2230/15Animal milk with modified composition due to manipulation of the animal, e.g. animal milk comprising antibodies, selection of animals having specific genotypes
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/80Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85Vector systems having a special element relevant for transcription from vertebrates mammalian
    • YGENERAL 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
    • Y10TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10STECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S119/00Animal husbandry
    • Y10S119/01Milk digest

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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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DK96928405T 1995-08-02 1996-07-31 Lysosomale proteiner produceret i mælk fra ikke-humane transgene dyr DK0845939T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US179695P 1995-08-02 1995-08-02
PCT/EP1996/003421 WO1997005771A2 (en) 1995-08-02 1996-07-31 Lysosomal proteins produced in the milk of transgenic animals

Publications (1)

Publication Number Publication Date
DK0845939T3 true DK0845939T3 (da) 2003-09-22

Family

ID=21697877

Family Applications (1)

Application Number Title Priority Date Filing Date
DK96928405T DK0845939T3 (da) 1995-08-02 1996-07-31 Lysosomale proteiner produceret i mælk fra ikke-humane transgene dyr

Country Status (11)

Country Link
US (5) US6118045A (da)
EP (1) EP0845939B1 (da)
JP (1) JP2000501602A (da)
KR (1) KR19990036084A (da)
AT (1) ATE240637T1 (da)
AU (1) AU734953B2 (da)
DE (1) DE69628306T2 (da)
DK (1) DK0845939T3 (da)
ES (1) ES2200071T3 (da)
PT (1) PT845939E (da)
WO (1) WO1997005771A2 (da)

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6118045A (en) * 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
US20040161837A1 (en) * 1995-08-02 2004-08-19 Reuser Arnold J. Methods of purifying human acid alpha-glucosidase
US20020013953A1 (en) * 1995-08-02 2002-01-31 Reuser Arnold J. Compositions and methods for treating enzyme deficiency
EP1793000A1 (en) * 1997-02-25 2007-06-06 GTC Biotherapeutics, Inc. Transgenically produced non-secreted proteins
EP0973934A4 (en) * 1997-02-25 2003-02-05 Genzyme Transgenics Corp NON-SECRETED PROTEINS PRODUCED TRANSGENICALLY
DK2020438T3 (da) * 1998-12-07 2018-07-23 Genzyme Corp Behandling af Pompes sygdom
CA2354638C (en) * 1999-01-06 2013-03-19 Atlantic Biopharmaceuticals, Inc. Expression of secreted human alpha-fetoprotein in transgenic animals
US7361481B2 (en) * 1999-04-09 2008-04-22 Women's And Children's Hospital Diagnosis of lysosomal storage disorders using saposins and other markers
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
US6770468B1 (en) * 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
DE60138535D1 (de) 2000-02-04 2009-06-10 Children S Hospital Res Founda Verwendung von lysosomal acid lipase zur behandlung von atherosklerose und ähnlichen krankheiten
SI3449934T1 (sl) 2000-07-18 2020-09-30 Duke University Zdravljenje bolezni kopičenja glikogena tip II
US7001994B2 (en) * 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
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
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
DE60224816T2 (de) * 2001-04-30 2009-01-22 ZyStor Therapeutics, Inc., Milwaukee Subzelluläres targeting von therapeutischen proteinen
US20030072761A1 (en) * 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US6800472B2 (en) 2001-12-21 2004-10-05 Genzyme Glycobiology Research Institute, Inc. Expression of lysosomal hydrolase in cells expressing pro-N-acetylglucosamine-1-phosphodiester α-N-acetyl glucosimanidase
US20040210953A1 (en) * 2003-01-09 2004-10-21 Murray James D. Lysozyme transgenic ungulates
US20040231010A1 (en) * 2003-01-09 2004-11-18 Murray James D. Lysozyme transgenic ungulates
WO2004064750A2 (en) 2003-01-22 2004-08-05 Duke University Improved constructs for expressing lysosomal polypeptides
KR20060123702A (ko) * 2003-08-21 2006-12-04 큐 세라퓨틱스, 아이엔씨. 리소좀성 축적병에서 결핍된 리소좀 효소전구체의 지속된분비를 위한 유전자 변형 체세포
ATE465250T1 (de) * 2004-02-10 2010-05-15 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
US7740861B2 (en) 2004-06-16 2010-06-22 University Of Massachusetts Drug delivery product and methods
WO2006007560A2 (en) 2004-07-01 2006-01-19 University Of Pennsylvania Targeted protein replacement for the treatment of lysosomal storage disorders
WO2006032039A2 (en) * 2004-09-17 2006-03-23 University Of Massachusetts Compositions and their uses for lysosomal enzyme deficiencies
EP2932982B1 (en) 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
ATE526991T1 (de) * 2005-10-24 2011-10-15 Univ Massachusetts Zusammensetzungen und ihre verwendungen für die gentherapeutische behandlung von knochenerkrankungen
WO2008063511A2 (en) * 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
NO2457920T3 (da) 2007-01-18 2018-03-24
JP2011517279A (ja) * 2007-10-29 2011-06-02 ユニバーシティ オブ マサチューセッツ 核酸(siRNA)送達用の酵母細胞壁粒子(YCWP)多層状ナノ粒子
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
BRPI0923047B8 (pt) 2008-12-16 2021-05-25 Genzyme Corp métodos para preparação de conjugados de proteínas- oligossacarídeos
EP2475376B1 (en) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
PT3205351T (pt) 2010-04-23 2023-06-28 Alexion Pharma Inc Enzima de doença de armazenamento lisossomal
PT3626258T (pt) 2010-06-25 2021-10-19 Shire Human Genetic Therapies Métodos e composições para fornecimento ao snc de iduronato-2-sulfatase
RU2012154576A (ru) 2010-06-25 2014-07-27 Шир Хьюман Дженетик Терапис, Инк. Способы и композиции для доставки к цнс гепаран-n-сульфатазы
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
EP2588130B1 (en) 2010-06-25 2016-08-17 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
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
ES2643015T3 (es) 2010-06-25 2017-11-21 Shire Human Genetic Therapies, Inc. Tratamiento del Síndrome de Sanfilippo Tipo B
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
WO2013023763A1 (en) 2011-08-16 2013-02-21 Instituto Nacional De Tecnologia Agropecuaria (Inta) Bitransgenic bovine
US10350277B2 (en) 2011-09-07 2019-07-16 Icahn School Of Medicine At Mount Sinai Ceramidase and cell differentiation
WO2013096899A2 (en) 2011-12-23 2013-06-27 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR20210062731A (ko) 2012-03-07 2021-05-31 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
HUE051020T2 (hu) 2012-06-01 2021-01-28 Icahn School Med Mount Sinai A ceramid szintjei a fertõzések kezelésében és megelõzésében
AR094780A1 (es) 2013-02-13 2015-08-26 Laboratoire Français Du Fractionnement Et Des Biotechnologies ANTICUERPOS ANTI-TNF-a ALTAMENTE GALACTOSILADOS Y SUS USOS
TW201446962A (zh) 2013-02-13 2014-12-16 Lab Francais Du Fractionnement 具有修飾的糖化作用之蛋白質及其製造方法
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
ES2739811T3 (es) 2013-03-14 2020-02-04 Icahn School Med Mount Sinai Composiciones terapéuticas de ceramidasa ácida y métodos de fabricación y uso de ellas
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
MA41022A (fr) 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
SG11201804965SA (en) 2015-12-30 2018-07-30 Amicus Therapeutics Inc Augmented acid alpha-glucosidase for the treatment of pompe disease
KR20240001291A (ko) 2016-03-30 2024-01-03 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
JP7046003B2 (ja) 2016-03-30 2022-04-01 アミカス セラピューティックス インコーポレイテッド 高m6p組換えタンパク質の選択方法

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0279582A3 (en) * 1987-02-17 1989-10-18 Pharming B.V. Dna sequences to target proteins to the mammary gland for efficient secretion
WO1993025567A1 (en) * 1992-06-15 1993-12-23 Gene Pharming Europe B.V. Production of recombinant polypeptides by bovine species and transgenic methods
US5356804A (en) * 1990-10-24 1994-10-18 Mount Sinai School Of Medicine Of The City Of New York Cloning and expression of biologically active human α-galactosidase A
US6118045A (en) * 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals

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EP0845939A2 (en) 1998-06-10
ATE240637T1 (de) 2003-06-15
EP0845939B1 (en) 2003-05-21
AU734953B2 (en) 2001-06-28
WO1997005771A3 (en) 1997-04-03
KR19990036084A (ko) 1999-05-25
US6118045A (en) 2000-09-12
US20040003421A1 (en) 2004-01-01
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US20070011754A1 (en) 2007-01-11
DE69628306D1 (de) 2003-06-26
JP2000501602A (ja) 2000-02-15
PT845939E (pt) 2003-10-31
US20040172665A1 (en) 2004-09-02
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US20030097665A1 (en) 2003-05-22

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