DE60038104D1 - Arzneimittelzubereitungen für die behandlung von alpha-galactosidase a-mangel - Google Patents
Arzneimittelzubereitungen für die behandlung von alpha-galactosidase a-mangelInfo
- Publication number
- DE60038104D1 DE60038104D1 DE60038104T DE60038104T DE60038104D1 DE 60038104 D1 DE60038104 D1 DE 60038104D1 DE 60038104 T DE60038104 T DE 60038104T DE 60038104 T DE60038104 T DE 60038104T DE 60038104 D1 DE60038104 D1 DE 60038104D1
- Authority
- DE
- Germany
- Prior art keywords
- gal
- lack
- treatment
- methods
- preparations
- Prior art date
- Legal status (The legal status 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 status listed.)
- Expired - Lifetime
Links
- 238000002360 preparation method Methods 0.000 title abstract 5
- 102000005840 alpha-Galactosidase Human genes 0.000 title 1
- 108010030291 alpha-Galactosidase Proteins 0.000 title 1
- 238000000034 method Methods 0.000 abstract 4
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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/575—Hormones
- C07K14/61—Growth hormone [GH], i.e. somatotropin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- A61P9/00—Drugs for disorders of the cardiovascular system
-
- 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
-
- 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)
- C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
-
- 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)
- C12Y302/01022—Alpha-galactosidase (3.2.1.22)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Endocrinology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Diabetes (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Epidemiology (AREA)
- Toxicology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Urology & Nephrology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Emergency Medicine (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US266014 | 1988-11-02 | ||
| US09/266,014 US6458574B1 (en) | 1996-09-12 | 1999-03-11 | Treatment of a α-galactosidase a deficiency |
| PCT/US2000/006118 WO2000053730A2 (en) | 1999-03-11 | 2000-03-09 | Medical preparations for the treatment of alpha-galactosidase a deficiency |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| DE60038104D1 true DE60038104D1 (de) | 2008-04-03 |
| DE60038104T2 DE60038104T2 (de) | 2009-02-12 |
Family
ID=23012821
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DE60038104T Expired - Lifetime DE60038104T2 (de) | 1999-03-11 | 2000-03-09 | Arzneimittelzubereitungen für die behandlung von alpha-galactosidase a-mangel |
Country Status (22)
| Country | Link |
|---|---|
| US (4) | US6458574B1 (de) |
| EP (6) | EP1820862A3 (de) |
| JP (6) | JP2002538183A (de) |
| KR (2) | KR100961740B1 (de) |
| CN (4) | CN103585621B (de) |
| AT (1) | ATE386808T1 (de) |
| AU (1) | AU3519400A (de) |
| CA (3) | CA2365923A1 (de) |
| CY (3) | CY1107951T1 (de) |
| DE (1) | DE60038104T2 (de) |
| DK (3) | DK2314699T3 (de) |
| ES (3) | ES2391221T3 (de) |
| HK (2) | HK1043386B (de) |
| HU (1) | HU228743B1 (de) |
| IL (2) | IL145381A0 (de) |
| MX (1) | MXPA01009222A (de) |
| NO (2) | NO329689B1 (de) |
| NZ (1) | NZ514077A (de) |
| PL (1) | PL210833B1 (de) |
| PT (3) | PT2186902E (de) |
| RU (1) | RU2248213C2 (de) |
| WO (1) | WO2000053730A2 (de) |
Families Citing this family (71)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6458574B1 (en) * | 1996-09-12 | 2002-10-01 | Transkaryotic Therapies, Inc. | Treatment of a α-galactosidase a deficiency |
| US6083725A (en) * | 1996-09-13 | 2000-07-04 | Transkaryotic Therapies, Inc. | Tranfected human cells expressing human α-galactosidase A protein |
| IL155588A0 (en) * | 2003-04-27 | 2003-11-23 | Metabogal Ltd | Methods for expression of enzymatically active recombinant lysosomal enzymes in transgenic plant root cells and vectors used thereby |
| US20050032211A1 (en) * | 1996-09-26 | 2005-02-10 | Metabogal Ltd. | Cell/tissue culturing device, system and method |
| ATE384736T1 (de) | 2001-04-30 | 2008-02-15 | Zystor Therapeutics Inc | Subzelluläres targeting von therapeutischen proteinen |
| 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 |
| US20030072761A1 (en) | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| JP5570677B2 (ja) * | 2002-04-25 | 2014-08-13 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | α−ガラクトシダーゼA欠損症の治療 |
| WO2004069190A2 (en) | 2003-01-31 | 2004-08-19 | Mount Sinai School Of Medicine Of New York University | Combination therapy for treating protein deficiency disorders |
| US7422310B2 (en) * | 2003-04-25 | 2008-09-09 | Hewlett-Packard Development Company, L.P. | Methods and apparatus for selecting image enhancement techniques |
| US7951557B2 (en) * | 2003-04-27 | 2011-05-31 | Protalix Ltd. | Human lysosomal proteins from plant cell culture |
| US20100196345A1 (en) * | 2003-04-27 | 2010-08-05 | Protalix | Production of high mannose proteins in plant culture |
| US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| ES2344302T3 (es) | 2004-02-10 | 2010-08-24 | Zystor Therapeutics , Inc. | Alfa glucosidasa acida y fragmentos de la misma. |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| EP1773401B1 (de) * | 2004-06-21 | 2013-01-02 | Medtronic, Inc. | Medizinische systeme und verfahren zur abgabe von zusammensetzungen an zellen |
| CN1308444C (zh) * | 2005-04-15 | 2007-04-04 | 中国人民解放军军事医学科学院野战输血研究所 | 基因重组α-半乳糖苷酶大批量发酵液的纯化方法 |
| ES2572148T3 (es) | 2005-05-17 | 2016-05-30 | Amicus Therapeutics Inc | Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados |
| AU2006316122A1 (en) | 2005-11-18 | 2007-05-24 | Altif Laboratories Inc. | Novel highly functional enzyme having modified substrate-specificity |
| US7776557B2 (en) * | 2006-01-30 | 2010-08-17 | Diagnostic Technologies Ltd. | Method for monitoring tocolytic treatment |
| JP2010509344A (ja) * | 2006-11-13 | 2010-03-25 | ザイストール セラピューティクス, インコーポレイテッド | ポンペ病を治療するための方法 |
| BRPI0810343B1 (pt) * | 2007-05-07 | 2021-03-30 | Protalix Ltd | Dispositivo descartável para o cultivo e colheita de tecidos vegetais e/ou células, método de cultivo e colheita de um tecido vegetal e/ou células vegetais em um volume maior do que 400 litros e sistema de cultivo da célula vegetal |
| EP2185187A1 (de) * | 2007-08-29 | 2010-05-19 | Shire Human Genetic Therapies, Inc. | Subkutane verabreichung von alpha-galatosidase a |
| US20110223147A1 (en) | 2008-05-07 | 2011-09-15 | Zystor Therapeutics, Inc. | Lysosomal targeting peptides and uses thereof |
| US9050276B2 (en) | 2009-06-16 | 2015-06-09 | The Trustees Of Columbia University In The City Of New York | Autism-associated biomarkers and uses thereof |
| EP3679942A1 (de) | 2009-06-17 | 2020-07-15 | BioMarin Pharmaceutical Inc. | Formulierungen für lysosomale enzyme |
| US9194011B2 (en) | 2009-11-17 | 2015-11-24 | Protalix Ltd. | Stabilized alpha-galactosidase and uses thereof |
| BR112012022029B1 (pt) * | 2010-03-02 | 2022-10-11 | Protalix Ltd | Estrutura de proteina multimérica, seu uso e processo para preparação da estrutura de proteína multimérica |
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| ES3037386T3 (en) | 2010-07-08 | 2025-10-01 | Takeda Pharmaceuticals Co | Method of producing recombinant high molecular weight vwf in cell culture |
| CN108404115A (zh) | 2010-10-15 | 2018-08-17 | 纽约市哥伦比亚大学理事会 | 肥胖症-相关的基因和它们的蛋白和其用途 |
| EP2635299B1 (de) | 2010-11-02 | 2019-07-31 | The Trustees of Columbia University in the City of New York | Verfahren zur behandlung von haarausfall |
| WO2012121041A1 (ja) | 2011-03-04 | 2012-09-13 | 株式会社糖鎖工学研究所 | シアル酸含有糖鎖の製造方法 |
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| KR20140135222A (ko) | 2012-03-07 | 2014-11-25 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
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| KR102135601B1 (ko) | 2012-03-29 | 2020-07-20 | 더 트러스티스 오브 콜롬비아 유니버시티 인 더 시티 오브 뉴욕 | 탈모 질환의 치료 방법 |
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| JP6226435B2 (ja) * | 2012-07-26 | 2017-11-08 | Jcrファーマ株式会社 | 組換えヒトα−ガラクトシダーゼAの製造方法 |
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| WO2014152659A1 (en) | 2013-03-14 | 2014-09-25 | Shire Human Genetic Therapies, Inc. | Quantitative assessment for cap efficiency of messenger rna |
| MX2015011945A (es) | 2013-03-14 | 2015-12-01 | Shire Human Genetic Therapies | Evaluacion cuantitativa para la eficacia de los casquetes de arn mensajero. |
| TW202332774A (zh) * | 2013-10-23 | 2023-08-16 | 美商健臻公司 | 重組醣蛋白及其用途 |
| US9682123B2 (en) | 2013-12-20 | 2017-06-20 | The Trustees Of Columbia University In The City Of New York | Methods of treating metabolic disease |
| US9457067B2 (en) | 2014-03-05 | 2016-10-04 | Ultragenyx Pharmaceutical Inc. | Sialylated glycoprotein compositions and uses thereof |
| DK3201320T5 (da) | 2014-09-30 | 2024-10-14 | Amicus Therapeutics Inc | Højpotent sur alfa-glucosidase med forbedrede carbohydrater |
| KR102438885B1 (ko) * | 2014-12-22 | 2022-09-01 | 코덱시스, 인코포레이티드 | 인간 알파-갈락토시다제 변이체 |
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| CA2997947A1 (en) | 2015-09-09 | 2017-03-16 | The Trustees Of Columbia University In The City Of New York | Reduction of er-mam-localized app-c99 and methods of treating alzheimer's disease |
| 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. |
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| KR102343162B1 (ko) * | 2016-03-30 | 2021-12-23 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 고 m6p 재조합 단백질의 선택 방법 |
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