DK1137762T3 - Behandling af Pompes sygdom - Google Patents

Behandling af Pompes sygdom

Info

Publication number
DK1137762T3
DK1137762T3 DK99965162T DK99965162T DK1137762T3 DK 1137762 T3 DK1137762 T3 DK 1137762T3 DK 99965162 T DK99965162 T DK 99965162T DK 99965162 T DK99965162 T DK 99965162T DK 1137762 T3 DK1137762 T3 DK 1137762T3
Authority
DK
Denmark
Prior art keywords
treatment
pompe disease
disease
patient
body weight
Prior art date
Application number
DK99965162T
Other languages
Danish (da)
English (en)
Inventor
Bree Johannes Brenardus Mathias Marie Van
Edna Henriette Germaine Venneker
David P Meeker
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=DK1137762(T3) "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 DK1137762T3 publication Critical patent/DK1137762T3/da

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
    • 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
    • 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)
  • Environmental Sciences (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Husbandry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Endocrinology (AREA)
DK99965162T 1998-12-07 1999-12-06 Behandling af Pompes sygdom DK1137762T3 (da)

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
DK1137762T3 true DK1137762T3 (da) 2009-02-02

Family

ID=22337648

Family Applications (2)

Application Number Title Priority Date Filing Date
DK99965162T DK1137762T3 (da) 1998-12-07 1999-12-06 Behandling af Pompes sygdom
DK08164825.5T DK2020438T3 (en) 1998-12-07 1999-12-06 Treatment of Pompe disease

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK08164825.5T DK2020438T3 (en) 1998-12-07 1999-12-06 Treatment of Pompe disease

Country Status (16)

Country Link
US (7) US20030007963A1 (ja)
EP (2) EP1137762B1 (ja)
JP (7) JP4990434B2 (ja)
KR (1) KR20010101131A (ja)
AT (1) ATE410506T1 (ja)
AU (1) AU3113700A (ja)
BE (1) BE2018C038I2 (ja)
CA (1) CA2353522A1 (ja)
CY (2) CY1121113T1 (ja)
DE (2) DE122009000018I1 (ja)
DK (2) DK1137762T3 (ja)
ES (2) ES2677343T3 (ja)
LU (1) LU91544I2 (ja)
NL (1) NL300382I2 (ja)
PT (2) PT1137762E (ja)
WO (1) WO2000034451A1 (ja)

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2677343T3 (es) * 1998-12-07 2018-08-01 Genzyme Corporation Tratamiento de la enfermedad de Pompe
US6849257B2 (en) * 2000-02-04 2005-02-01 Children's Hospital Research Foundation Lipid hydrolysis therapy for atherosclerosis and related diseases
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
LT2767291T (lt) 2000-07-18 2016-11-25 Duke University Ii tipo glikogenozės gydymas
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
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
ES2300439T3 (es) * 2001-04-30 2008-06-16 Zystor Therapeutics , Inc. Reconocimiento subcelular de proteinas terapeuticas.
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, 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
WO2003086452A2 (en) 2002-04-05 2003-10-23 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy
PT1587923E (pt) * 2003-01-22 2011-12-07 Univ Duke Construções melhoradas para a expressão de polipéptidos lisossomais
MX344587B (es) 2003-01-31 2016-12-20 Mount Sinai School Of Medicine Of New York Univ Metodo para incrementar la estabilidad in vitro e in vivo de enzimas asociadas al transtorno lisosomal.
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
KR100697270B1 (ko) * 2004-12-10 2007-03-21 삼성전자주식회사 저전력 멀티칩 반도체 메모리 장치 및 그것의 칩 인에이블방법
WO2006125141A2 (en) 2005-05-17 2006-11-23 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin derivatives
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
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
US9050333B2 (en) 2008-07-08 2015-06-09 Duke University Method of treating glycogen storage disease type III with human acid α-glucosidase
EP3075386B1 (en) 2009-06-17 2019-10-16 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
SI3205351T1 (sl) 2010-04-23 2023-09-29 Alexion Pharmaceuticals, Inc., Encim za bolezen lizosomskega shranjevanja
MX2013000324A (es) 2010-06-25 2013-02-01 Shire Human Genetic Therapies Tratamiento del sindrome de sanfilippo tipo b.
DK2588130T3 (en) 2010-06-25 2016-10-24 Shire Human Genetic Therapies Cns delivery of therapeutic agents
ES2754234T3 (es) 2010-06-25 2020-04-16 Shire Human Genetic Therapies Métodos y composiciones para la administración SNC de iduronato-2-sulfatasa
LT2585104T (lt) 2010-06-25 2019-10-25 Shire Human Genetic Therapies Būdai ir kompozicijos, skirti arilsulfatazės a pristatymui į cns
RU2012154576A (ru) 2010-06-25 2014-07-27 Шир Хьюман Дженетик Терапис, Инк. Способы и композиции для доставки к цнс гепаран-n-сульфатазы
LT3626258T (lt) 2010-06-25 2021-12-10 Shire Human Genetic Therapies, Inc. Būdai ir kompozicijos, skirti iduronato-2-sulfatazės įvedimui į cns
SG188456A1 (en) 2010-09-09 2013-04-30 Synageva Biopharma Corp 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
WO2012124520A1 (ja) * 2011-03-16 2012-09-20 天野エンザイム株式会社 改変型α－グルコシダーゼ及びその用途
EP2699676A1 (en) * 2011-04-22 2014-02-26 Genzyme Corporation Modified acid alpha glucosidase with accelerated processing
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
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
US10603364B2 (en) 2014-08-11 2020-03-31 Shire Human Genetic Therapies, Inc. Lysosomal targeting and uses thereof
EA038986B1 (ru) 2014-09-30 2021-11-18 Амикус Терапьютикс, Инк. Высокоактивная кислая альфа-глюкозидаза с повышенным содержанием углеводов
MA41022A (fr) 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
MA44747A (fr) * 2015-08-31 2019-03-06 Univ Duke Méthodes et compositions pour le traitement de troubles associés à une accumulation du glycogène cytoplasmique
US10940125B2 (en) 2015-09-18 2021-03-09 Duke University Methods and compositions for the treatment of steatosis-associated disorders
BR112018013151A2 (pt) 2015-12-30 2018-12-18 Amicus Therapeutics Inc alfa-glicosidase ácida aumentada para o tratamento da doença de pompe
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293260A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
SG11201901494UA (en) 2016-09-12 2019-03-28 Genethon Acid-alpha glucosidase variants and uses thereof
WO2021005176A1 (en) 2019-07-09 2021-01-14 Genethon Treatment of glycogen storage disease (gsd)

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0832981A1 (en) * 1987-02-17 1998-04-01 Pharming B.V. DNA sequences to target proteins to the mammary gland for efficient secretion
DE69027683T2 (de) 1989-12-01 1997-02-13 Pharming Bv Herstellung rekombinanter polypeptide durch rinder und transgene methoden
WO1993025567A1 (en) 1992-06-15 1993-12-23 Gene Pharming Europe B.V. Production of recombinant polypeptides by bovine species and transgenic methods
DE69133557D1 (de) 1990-08-29 2007-03-15 Pharming Intellectual Pty Bv Homologe rekombination in säugetier-zellen
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
EP0625530A1 (en) * 1993-05-17 1994-11-23 Nippon Paint Co., Ltd. Oxazolidone ring-containing modified epoxy resins and cathodic electrodeposition paints containing same
AT1470U1 (de) 1995-08-01 1997-05-26 Austria Card Laminierte karte und verfahren zu ihrer herstellung
US6118045A (en) * 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
ES2677343T3 (es) * 1998-12-07 2018-08-01 Genzyme Corporation Tratamiento de la enfermedad de Pompe

Also Published As

Publication number Publication date
EP1137762A4 (en) 2005-01-12
JP4990434B2 (ja) 2012-08-01
JP2002531581A (ja) 2002-09-24
EP1137762A1 (en) 2001-10-04
CY1121113T1 (el) 2019-07-10
PT1137762E (pt) 2009-01-14
EP1137762B1 (en) 2008-10-08
NL300382I2 (en) 2009-12-01
DE122009000018I1 (de) 2009-09-17
CY2018020I2 (el) 2019-07-10
ES2312222T3 (es) 2009-02-16
DE69939705D1 (de) 2008-11-20
ATE410506T1 (de) 2008-10-15
US20140037611A1 (en) 2014-02-06
CY2018020I1 (el) 2019-07-10
NL300382I1 (nl) 2009-06-02
AU3113700A (en) 2000-06-26
JP2012224643A (ja) 2012-11-15
ES2677343T3 (es) 2018-08-01
EP2020438A1 (en) 2009-02-04
US20130039901A1 (en) 2013-02-14
JP2017066160A (ja) 2017-04-06
DK2020438T3 (en) 2018-07-23
US20040081645A1 (en) 2004-04-29
PT2020438T (pt) 2018-07-13
US7351410B2 (en) 2008-04-01
US20100092449A1 (en) 2010-04-15
KR20010101131A (ko) 2001-11-14
JP2018115200A (ja) 2018-07-26
US20030007963A1 (en) 2003-01-09
US20160243203A1 (en) 2016-08-25
US20080206223A1 (en) 2008-08-28
LU91544I2 (fr) 2009-05-25
JP2019172685A (ja) 2019-10-10
US7655226B2 (en) 2010-02-02
EP2020438B1 (en) 2018-04-11
CA2353522A1 (en) 2000-06-15
JP2009161562A (ja) 2009-07-23
WO2000034451A1 (en) 2000-06-15
BE2018C038I2 (ja) 2019-03-05
JP2015134836A (ja) 2015-07-27

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