DK2020438T3 - Treatment of Pompe disease - Google Patents

Treatment of Pompe disease Download PDF

Info

Publication number
DK2020438T3
DK2020438T3 DK08164825.5T DK08164825T DK2020438T3 DK 2020438 T3 DK2020438 T3 DK 2020438T3 DK 08164825 T DK08164825 T DK 08164825T DK 2020438 T3 DK2020438 T3 DK 2020438T3
Authority
DK
Denmark
Prior art keywords
glucosidase
lysosomal
human
acid
vol
Prior art date
Application number
DK08164825.5T
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=DK2020438(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 DK2020438T3 publication Critical patent/DK2020438T3/en

Links

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)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Husbandry (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Cardiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Dermatology (AREA)
  • Endocrinology (AREA)
DK08164825.5T 1998-12-07 1999-12-06 Treatment of Pompe disease DK2020438T3 (en)

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
DK2020438T3 true DK2020438T3 (en) 2018-07-23

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 Before (1)

Application Number Title Priority Date Filing Date
DK99965162T DK1137762T3 (da) 1998-12-07 1999-12-06 Behandling af Pompes sygdom

Country Status (16)

Country Link
US (7) US20030007963A1 (enExample)
EP (2) EP1137762B1 (enExample)
JP (7) JP4990434B2 (enExample)
KR (1) KR20010101131A (enExample)
AT (1) ATE410506T1 (enExample)
AU (1) AU3113700A (enExample)
BE (1) BE2018C038I2 (enExample)
CA (1) CA2353522A1 (enExample)
CY (2) CY1121113T1 (enExample)
DE (2) DE69939705D1 (enExample)
DK (2) DK1137762T3 (enExample)
ES (2) ES2677343T3 (enExample)
LU (1) LU91544I2 (enExample)
NL (1) NL300382I2 (enExample)
PT (2) PT2020438T (enExample)
WO (1) WO2000034451A1 (enExample)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2020438T (pt) * 1998-12-07 2018-07-13 Genzyme Corp Tratamento da doença de pompe
CA2398995C (en) 2000-02-04 2014-09-23 Children's Hospital Research Foundation Lipid hydrolysis therapy for atherosclerosis and related diseases
EP1782825B1 (en) * 2000-07-18 2014-09-10 Duke University Treatment of glycogen storage diseases type II
AU2016256663A1 (en) * 2000-07-18 2016-11-24 Duke University Treatment of clycogen storage disease type II
AU2001271941B2 (en) * 2000-07-18 2007-03-01 Duke University Treatment of glycogen storage disease 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
US7560424B2 (en) * 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
AU2002256423B2 (en) * 2001-04-30 2008-07-24 Zystor Therapeutics, Inc. Subcellular targeting of therapeutic proteins
US7629309B2 (en) * 2002-05-29 2009-12-08 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
US7658916B2 (en) 2002-04-05 2010-02-09 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy by modulation of cell surface receptor density
US20040248262A1 (en) * 2003-01-22 2004-12-09 Koeberl Dwight D. Constructs for expressing lysomal polypeptides
PT2444102E (pt) * 2003-01-31 2015-09-17 Sinai School Medicine Terapia combinada para o tratamento de distúrbios de deficiência proteica
US20050244400A1 (en) * 2004-02-10 2005-11-03 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
KR100697270B1 (ko) * 2004-12-10 2007-03-21 삼성전자주식회사 저전력 멀티칩 반도체 메모리 장치 및 그것의 칩 인에이블방법
EP2932982B1 (en) 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
WO2007030375A2 (en) * 2005-09-08 2007-03-15 Children's Hospital Medical Center Lysosomal acid lipase therapy for nafld and related diseases
EP2099523A2 (en) * 2006-11-13 2009-09-16 ZyStor Therapeutics , Inc. Methods for treating pompe disease
BRPI0912225A2 (pt) 2008-05-07 2018-03-20 Zystor Therapeutics, Inc. peptídeos de direcionamento lisossomais e usos dos mesmos
WO2010005565A2 (en) * 2008-07-08 2010-01-14 Duke University Method of treating glycogen storage disease
EP3075386B1 (en) 2009-06-17 2019-10-16 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
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
PL2561069T3 (pl) 2010-04-23 2017-08-31 Alexion Pharmaceuticals, Inc. Enzym lizosomalnej choroby spichrzeniowej
US9320711B2 (en) 2010-06-25 2016-04-26 Shire Human Genetic Therapies, Inc. Methods and compositions for CNS delivery of heparan N-sulfatase
HUE056884T2 (hu) 2010-06-25 2022-03-28 Shire Human Genetic Therapies Eljárások és készítmények arilszulfatáz-A CNS-be történõ beadására
MX2013000324A (es) 2010-06-25 2013-02-01 Shire Human Genetic Therapies Tratamiento del sindrome de sanfilippo tipo b.
EP3103469B1 (en) 2010-06-25 2020-12-09 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
PL2593131T3 (pl) 2010-06-25 2020-01-31 Shire Human Genetic Therapies, Inc. Sposoby i kompozycje do dostarczania sulfatazy-2-iduronianiu do oun
KR102537084B1 (ko) 2010-06-25 2023-05-26 샤이어 휴먼 지네틱 테라피즈 인크. 아릴설파타제 a의 cns 전달을 위한 방법들 및 조성물들
SG10201507199UA (en) 2010-09-09 2015-10-29 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
US9493753B2 (en) 2011-03-16 2016-11-15 Amano Enzyme Inc. Modified α-glucosidase and applications of same
CA2833371A1 (en) 2011-04-22 2012-10-26 Genzyme Corporation Modified acid alpha glucosidase with accelerated processing
EP2793922B1 (en) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
KR20140135222A (ko) 2012-03-07 2014-11-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
CA2901978A1 (en) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of pompe disease
US10603364B2 (en) 2014-08-11 2020-03-31 Shire Human Genetic Therapies, Inc. Lysosomal targeting and uses thereof
SI4273241T1 (sl) 2014-09-30 2025-03-31 Amicus Therapeutics, Inc. Zelo močna kislinska alfa-glukozidaza z okrepljenimi ogljikovimi hidrati
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
WO2017049157A1 (en) 2015-09-18 2017-03-23 Duke University Methods and compositions for the treatment of steatosis-associated disorders
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
NZ786723A (en) 2016-03-30 2025-07-25 Amicus Therapeutics Inc Method for selection of high m6p recombinant proteins
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
EP3293260A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
BR112019004785A2 (pt) 2016-09-12 2019-06-04 Genethon variantes de alfa-glicosidase ácida e usos das mesmas
DK3624831T5 (da) 2017-05-15 2024-09-02 Amicus Therapeutics Inc Rekombinant human sur alfa-glucosidase
US20220331408A1 (en) 2019-07-09 2022-10-20 Genethon Treatment of glycogen storage disease (gsd)
CN114829379B (zh) * 2019-12-17 2025-10-24 纳维格蛋白质有限公司 酶酸α葡萄糖苷酶(GAA)的结合蛋白及其用途

Family Cites Families (9)

* 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
DE69027683T2 (de) 1989-12-01 1997-02-13 Pharming Bv Herstellung rekombinanter polypeptide durch rinder und transgene methoden
US5612205A (en) 1990-08-29 1997-03-18 Genpharm International, Incorporated Homologous recombination in mammalian cells
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
TW319781B (enExample) * 1993-05-17 1997-11-11 Nippon Painting Co Ltd
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
PT2020438T (pt) * 1998-12-07 2018-07-13 Genzyme Corp Tratamento da doença de pompe

Also Published As

Publication number Publication date
US7351410B2 (en) 2008-04-01
CY1121113T1 (el) 2019-07-10
US20160243203A1 (en) 2016-08-25
DE69939705D1 (de) 2008-11-20
PT2020438T (pt) 2018-07-13
JP2018115200A (ja) 2018-07-26
JP2009161562A (ja) 2009-07-23
JP2002531581A (ja) 2002-09-24
US20130039901A1 (en) 2013-02-14
CY2018020I2 (el) 2019-07-10
US20100092449A1 (en) 2010-04-15
ATE410506T1 (de) 2008-10-15
WO2000034451A1 (en) 2000-06-15
EP1137762B1 (en) 2008-10-08
JP2012224643A (ja) 2012-11-15
PT1137762E (pt) 2009-01-14
CY2018020I1 (el) 2019-07-10
EP2020438A1 (en) 2009-02-04
CA2353522A1 (en) 2000-06-15
LU91544I2 (fr) 2009-05-25
JP2017066160A (ja) 2017-04-06
US7655226B2 (en) 2010-02-02
US20080206223A1 (en) 2008-08-28
AU3113700A (en) 2000-06-26
US20140037611A1 (en) 2014-02-06
DK1137762T3 (da) 2009-02-02
JP4990434B2 (ja) 2012-08-01
DE122009000018I1 (de) 2009-09-17
JP2015134836A (ja) 2015-07-27
JP2019172685A (ja) 2019-10-10
ES2312222T3 (es) 2009-02-16
EP2020438B1 (en) 2018-04-11
ES2677343T3 (es) 2018-08-01
US20030007963A1 (en) 2003-01-09
US20040081645A1 (en) 2004-04-29
KR20010101131A (ko) 2001-11-14
EP1137762A4 (en) 2005-01-12
NL300382I2 (en) 2009-12-01
BE2018C038I2 (enExample) 2019-03-05
NL300382I1 (nl) 2009-06-02
EP1137762A1 (en) 2001-10-04

Similar Documents

Publication Publication Date Title
DK2020438T3 (en) Treatment of Pompe disease
AU734953B2 (en) Lysosomal proteins produced in the milk of transgenic animals
US20020157123A1 (en) Compositions and methods for treating enzyme deficiency
US20020073438A1 (en) Methods of purifying human acid alpha-glucosidase
US20040161837A1 (en) Methods of purifying human acid alpha-glucosidase
AU2005200636B2 (en) Lysosomal proteins produced in the milk of transgenic animals
EP1262191A1 (en) Pharmaceutical compositions comprising recombinant human acid alpha-glucosidase containing mannose 6-phosphate
NZ501784A (en) Transgenic nonhuman mammals capable of secreting a lysosomal protein containing mannose 6-phosphate into milk