ATE240637T1 - Lysomale proteine, hergestellt in der milch von nichtmenschlichen transgenen tieren - Google Patents

Lysomale proteine, hergestellt in der milch von nichtmenschlichen transgenen tieren

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Publication number
ATE240637T1
ATE240637T1 AT96928405T AT96928405T ATE240637T1 AT E240637 T1 ATE240637 T1 AT E240637T1 AT 96928405 T AT96928405 T AT 96928405T AT 96928405 T AT96928405 T AT 96928405T AT E240637 T1 ATE240637 T1 AT E240637T1
Authority
AT
Austria
Prior art keywords
milk
lysomal
transgenic animals
human transgenic
proteins produced
Prior art date
Application number
AT96928405T
Other languages
English (en)
Inventor
Arnold J J Reuser
Der Ploeg Ans T Van
Frank R Pieper
Martin Ph Verbeet
Original Assignee
Pharming Intellectual Pty Bv
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=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ATE240637(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Pharming Intellectual Pty Bv, Univ Erasmus, Academic Hospital Rotterdam filed Critical Pharming Intellectual Pty Bv
Application granted granted Critical
Publication of ATE240637T1 publication Critical patent/ATE240637T1/de

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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; PREPARATION 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
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    • 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
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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
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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
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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
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    • 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
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    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
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    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • AHUMAN NECESSITIES
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    • 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
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    • 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; PREPARATION 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; PREPARATION 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
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    • 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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    • 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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  • Proteomics, Peptides & Aminoacids (AREA)
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AT96928405T 1995-08-02 1996-07-31 Lysomale proteine, hergestellt in der milch von nichtmenschlichen transgenen tieren ATE240637T1 (de)

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
ATE240637T1 true ATE240637T1 (de) 2003-06-15

Family

ID=21697877

Family Applications (1)

Application Number Title Priority Date Filing Date
AT96928405T ATE240637T1 (de) 1995-08-02 1996-07-31 Lysomale proteine, hergestellt in der milch von nichtmenschlichen transgenen tieren

Country Status (11)

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

Families Citing this family (63)

* 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
US20030007963A1 (en) * 1998-12-07 2003-01-09 Van Bree Johannes B. M. M. Treatment of pompe's disease
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
CA2281949A1 (en) * 1997-02-25 1998-08-27 Genzyme Transgenics Corporation Transgenically produced non-secreted proteins
EP1793000A1 (de) * 1997-02-25 2007-06-06 GTC Biotherapeutics, Inc. Transgen produzierte nicht-sekretierte Proteine
EP1958503A3 (de) * 1999-01-06 2008-11-26 Merrimack Pharmaceuticals, Inc. Expression von ausgeschiedenem menschlichen Alpha-Fetoprotein in transgenen Tieren
US7361481B2 (en) * 1999-04-09 2008-04-22 Women's And Children's Hospital Diagnosis of lysosomal storage disorders using saposins and other markers
US6642038B1 (en) 1999-09-14 2003-11-04 Genzyme Glycobiology Research Institute, Inc. GlcNAc phosphotransferase of the lysosomal targeting pathway
US6770468B1 (en) * 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
EP1267914B2 (de) 2000-02-04 2013-11-06 Children's Hospital Research Foundation Verwendung von lysosomal acid lipase zur behandlung von atherosklerose und ähnlichen krankheiten
DK1782825T3 (da) 2000-07-18 2014-11-03 Univ Duke Behandling af glycogenlagringssygdom type 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
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
ES2300439T3 (es) * 2001-04-30 2008-06-16 Zystor Therapeutics , Inc. Reconocimiento subcelular de proteinas terapeuticas.
US7560424B2 (en) * 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
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
US6905856B2 (en) 2001-12-21 2005-06-14 Genzyme Glycobiology Research Institute, Inc. Soluble GlcNAc phosphotransferase
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
US20040248262A1 (en) * 2003-01-22 2004-12-09 Koeberl Dwight D. Constructs for expressing lysomal polypeptides
CA2535816A1 (en) * 2003-08-21 2005-03-10 Q Therapeutics, Inc. Genetically modified somatic cells for sustained secretion of lysosomal proenzymes deficient in lysosomal storage disorders
CN1922313B (zh) * 2004-02-10 2011-10-26 生物马林医药公司 酸性α-糖苷酶及其片段
US7740861B2 (en) * 2004-06-16 2010-06-22 University Of Massachusetts Drug delivery product and methods
US8889127B2 (en) * 2004-07-01 2014-11-18 Icahn School Of Medicine At Mount Sinai Targeted protein replacement for the treatment of lysosomal storage disorders
CN101052383B (zh) * 2004-09-17 2013-01-30 马萨诸塞大学 用于溶酶体酶缺乏症的组合物和其用途
EP1896083B1 (de) 2005-05-17 2016-03-02 Amicus Therapeutics, Inc. Verfahren zur behandlung von pompe-krankheit mit 1-desoxynojirimycin und derivaten
JP5489462B2 (ja) * 2005-10-24 2014-05-14 ユニバーシティ オブ マサチューセッツ 骨病状の遺伝子治療のための組成物およびそれらの使用法
US20090117091A1 (en) * 2006-11-13 2009-05-07 Lebowitz Jonathan Methods for treating pompe disease
DK2121713T3 (da) 2007-01-18 2013-06-24 Genzyme Corp Oligosaccharider, omfattende en aminooxygruppe, og konjugater deraf
EP2222283A2 (de) * 2007-10-29 2010-09-01 University of Massachusetts Zur freisetzung von nukleinsäuren (sirna) mit hefe-zellwandproteinen verkapselte mehrlagige nanopatikel
BRPI0912225A2 (pt) 2008-05-07 2018-03-20 Zystor Therapeutics, Inc. peptídeos de direcionamento lisossomais e usos dos mesmos
HUE044381T2 (hu) 2008-12-16 2019-10-28 Genzyme Corp Szintetikus intermedierek oligoszacharid-fehérje konjugátumok elõállításához
EP2475376B1 (de) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulierungen für lysosomale enzyme
JP5925187B2 (ja) 2010-04-23 2016-05-25 シナジーバ バイオファーマ コープ リソソーム蓄積症酵素
WO2011163648A1 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
CN103269709A (zh) 2010-06-25 2013-08-28 夏尔人类遗传性治疗公司 Sanfilippo综合症类型B的治疗
PT3626257T (pt) 2010-06-25 2021-11-12 Shire Human Genetic Therapies Métodos e composições de arilsulfatase a para a administração no snc
CN103260637B (zh) 2010-06-25 2016-04-06 夏尔人类遗传性治疗公司 乙酰肝素n-硫酸酯酶cns递送的方法和组合物
AU2011270669B2 (en) 2010-06-25 2017-01-05 Takeda Pharmaceutical Company Limited Methods and compositions for CNS delivery of iduronate-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
IL225095A (en) 2010-09-09 2016-05-31 Synageva Biopharma Corp Lysosomal acidic lipase for use in treating lysosomal acidic 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
HUE051021T2 (hu) 2011-09-07 2021-01-28 Sinai School Medicine Ceramidáz és sejtek differenciálódása
US20140377244A1 (en) 2011-12-23 2014-12-25 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR20200032244A (ko) 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
ES2897825T3 (es) 2012-06-01 2022-03-02 Icahn School Med Mount Sinai Niveles de ceramida en el tratamiento y prevención de infecciones
BR112015019348A2 (pt) 2013-02-13 2017-08-22 Lab Francais Du Fractionnement Métodos para produção de proteína com glicosilação modificada e com sialilação aumentada, para aumentar a atividade de sialil transferase na glândula mamária e para produzir sialil transferase, proteína com glicosilação modificada ou proteína com sialilação aumentada, composição, sialil transferase, mamífero transgênico, e, célula epitelial mamária
KR20160002713A (ko) 2013-02-13 2016-01-08 라보라토이레 프란카이즈 듀 프락티온네먼트 에트 데스 바이오테크놀로지스 고도로 갈락토실화된 항-tnf-알파 항체 및 이의 용도
CA2901978A1 (en) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of pompe disease
PL2968479T3 (pl) 2013-03-14 2019-10-31 Icahn School Med Mount Sinai Kompozycje terapeutyczne ceramidazy kwasowej i sposoby ich wytwarzania i stosowania
EP3193942B1 (de) 2014-08-11 2020-03-25 Shire Human Genetic Therapies, Inc. Lysosomales targeting und verwendungen davon
PE20170906A1 (es) 2014-09-30 2017-07-12 Amicus Therapeutics Inc Alfa-glucosidasa acida muy potente con hidratos de carbono potenciados
MA41022A (fr) 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
FR3038517B1 (fr) 2015-07-06 2020-02-28 Laboratoire Francais Du Fractionnement Et Des Biotechnologies Utilisation de fragments fc modifies en immunotherapie
MX394968B (es) 2015-12-30 2025-03-24 Amicus Therapeutics Inc Alfa-glucosidasa con mayor cantidad de acido para el tratamiento de la enfermedad de pompe.
MX2018011951A (es) 2016-03-30 2019-02-13 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p.
WO2017173060A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
AU2018270925B2 (en) 2017-05-15 2025-02-20 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0832981A1 (de) * 1987-02-17 1998-04-01 Pharming B.V. DNA-Sequenzen die Proteine zwecks effizienter Abscheidung zur Milchdrüse leiten
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
JP3670003B2 (ja) * 1992-06-15 2005-07-13 ファーミング ビー.ブイ. ウシ種による組み換えポリペプチドの製造及びトランスジェニック法
US6118045A (en) * 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals

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KR19990036084A (ko) 1999-05-25
US20030097665A1 (en) 2003-05-22
US20040003421A1 (en) 2004-01-01
PT845939E (pt) 2003-10-31
EP0845939B1 (de) 2003-05-21
ES2200071T3 (es) 2004-03-01
DE69628306T2 (de) 2004-04-01
DK0845939T3 (da) 2003-09-22
AU6789096A (en) 1997-03-05
JP2000501602A (ja) 2000-02-15
US6118045A (en) 2000-09-12
AU734953B2 (en) 2001-06-28
DE69628306D1 (de) 2003-06-26
US20070011754A1 (en) 2007-01-11

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