DE60126947T2 - Behandlung der glykogenspeicherkrankheit typ ii - Google Patents
Behandlung der glykogenspeicherkrankheit typ ii Download PDFInfo
- Publication number
- DE60126947T2 DE60126947T2 DE60126947T DE60126947T DE60126947T2 DE 60126947 T2 DE60126947 T2 DE 60126947T2 DE 60126947 T DE60126947 T DE 60126947T DE 60126947 T DE60126947 T DE 60126947T DE 60126947 T2 DE60126947 T2 DE 60126947T2
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- Prior art keywords
- glucosidase
- gaa
- medicament
- treatment
- storage disease
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- 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.)
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Classifications
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- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
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- 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
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- 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
-
- 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
- A61P9/02—Non-specific cardiovascular stimulants, e.g. drugs for syncope, antihypotensives
-
- 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
- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Cardiology (AREA)
- Diabetes (AREA)
- Heart & Thoracic Surgery (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Transplantation (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Hospice & Palliative Care (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US21923700P | 2000-07-18 | 2000-07-18 | |
| US219237P | 2000-07-18 | ||
| PCT/US2001/021651 WO2002005841A1 (en) | 2000-07-18 | 2001-07-10 | Treatment of glycogen storage disease type ii |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| DE60126947D1 DE60126947D1 (de) | 2007-04-12 |
| DE60126947T2 true DE60126947T2 (de) | 2007-10-31 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DE60126947T Expired - Lifetime DE60126947T2 (de) | 2000-07-18 | 2001-07-10 | Behandlung der glykogenspeicherkrankheit typ ii |
Country Status (17)
| Country | Link |
|---|---|
| US (10) | US7056712B2 (https=) |
| EP (4) | EP1301201B1 (https=) |
| JP (10) | JP5113312B2 (https=) |
| AT (1) | ATE355075T1 (https=) |
| AU (3) | AU7194101A (https=) |
| BR (1) | BR0113006A (https=) |
| CA (1) | CA2416492C (https=) |
| CY (2) | CY1120977T1 (https=) |
| DE (1) | DE60126947T2 (https=) |
| DK (5) | DK1301201T3 (https=) |
| ES (5) | ES2599401T3 (https=) |
| LT (3) | LT3449934T (https=) |
| MX (1) | MXPA03000474A (https=) |
| PT (5) | PT1782825E (https=) |
| SI (3) | SI3108895T1 (https=) |
| TW (1) | TWI280882B (https=) |
| WO (1) | WO2002005841A1 (https=) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU7194101A (en) * | 2000-07-18 | 2002-01-30 | Univ Duke | Treatment of glycogen storage disease type ii |
| US20040005309A1 (en) * | 2002-05-29 | 2004-01-08 | Symbiontics, Inc. | Targeted therapeutic proteins |
| ATE384736T1 (de) * | 2001-04-30 | 2008-02-15 | Zystor Therapeutics Inc | Subzelluläres targeting von therapeutischen proteinen |
| 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 |
| US6981964B2 (en) | 2001-05-22 | 2006-01-03 | Boston Scientific Scimed, Inc. | Draining bodily fluids with a stent |
| US20030072761A1 (en) * | 2001-10-16 | 2003-04-17 | Lebowitz Jonathan | Methods and compositions for targeting proteins across the blood brain barrier |
| US6733536B1 (en) | 2002-10-22 | 2004-05-11 | Scimed Life Systems | Male urethral stent device |
| ATE521701T1 (de) | 2003-01-22 | 2011-09-15 | Univ Duke | Verbesserte konstrukte zur expression lysosomaler polypeptide |
| US7442372B2 (en) | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US7129049B2 (en) * | 2003-12-22 | 2006-10-31 | Regents Of The University Of Minnesota | Method of detecting equine glycogen storage disease IV |
| 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 |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| JP2008503590A (ja) * | 2004-06-21 | 2008-02-07 | メドトロニック・インコーポレーテッド | 組成物を細胞にデリバリーするための医学用システム及び方法 |
| US20090117091A1 (en) * | 2006-11-13 | 2009-05-07 | Lebowitz Jonathan | Methods for treating pompe disease |
| WO2009075815A1 (en) | 2007-12-07 | 2009-06-18 | Duke University | Immunomodulating gene therapy |
| HRP20170698T1 (hr) | 2008-05-07 | 2017-09-22 | Biomarin Pharmaceutical Inc. | Lizosomski usmjereni peptidi i njihove uporabe |
| ES2535461T3 (es) * | 2008-07-08 | 2015-05-11 | Duke University | Método de tratamiento de la enfermedad por almacenamiento de glucógeno |
| EP2475376B1 (en) * | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| WO2011139379A2 (en) | 2010-05-06 | 2011-11-10 | Duke University | A method of treating patients undergoing protein replacement therapy, gene replacement therapy, or other therapeutic modalities |
| EP2981551B1 (en) | 2013-02-20 | 2020-06-03 | Valerion Therapeutics, LLC | Methods and compositions for treatment of pompe disease |
| CN105377039A (zh) * | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| WO2016054231A1 (en) | 2014-09-30 | 2016-04-07 | Amicus Therapeutics, Inc. | Highly potent acid alpha-glucosidase with enhanced carbohydrates |
| WO2016178431A1 (ja) * | 2015-05-07 | 2016-11-10 | 国立大学法人 東京大学 | ポリイオンコンプレックス型ポリマーソームを用いたナノリアクタとその製造方法 |
| EP3344337A4 (en) * | 2015-08-31 | 2019-03-06 | Duke University | METHOD AND COMPOSITIONS FOR TREATING CYTOPLASMATIC GLYCOGEN MEMORY DISEASES |
| 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 |
| IL325155A (en) | 2016-03-30 | 2026-02-01 | Amicus Therapeutics Inc | Formulations containing recombinant acid alpha-glucosidase |
| US10227577B2 (en) | 2016-03-30 | 2019-03-12 | Amicus Therapeutics, Inc. | Method for selection of high M6P recombinant proteins |
| IL305449B2 (en) | 2016-04-15 | 2026-01-01 | Univ Pennsylvania | Gene therapy for the treatment of mucocutaneous type ii diabetes |
| EP3624831B1 (en) | 2017-05-15 | 2023-03-29 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| WO2020163480A1 (en) * | 2019-02-05 | 2020-08-13 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase and uses thereof |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3063911A (en) * | 1961-04-03 | 1962-11-13 | Taisho Pharmaceutical Co Ltd | Acid-stable digestive enzyme preparation and process of making same |
| US5464778A (en) | 1989-03-08 | 1995-11-07 | Board Of Regents Of The University Of Oklahoma | Glycoprotein ligand for P-selectin and methods of use thereof |
| 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 |
| CA2328518A1 (en) | 1998-05-13 | 1999-11-18 | Harbor-Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| DK1137762T3 (da) | 1998-12-07 | 2009-02-02 | Genzyme Corp | Behandling af Pompes sygdom |
| US6537785B1 (en) | 1999-09-14 | 2003-03-25 | Genzyme Glycobiology Research Institute, Inc. | Methods of treating lysosomal storage diseases |
| AU7194101A (en) * | 2000-07-18 | 2002-01-30 | Univ Duke | Treatment of glycogen storage disease type ii |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
| 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 |
| US8470552B2 (en) | 2009-10-12 | 2013-06-25 | Keck Graduate Institute | Strategy to reduce lactic acid production and control PH in animal cell culture |
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2001
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- 2001-07-10 DK DK01951000T patent/DK1301201T3/da active
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- 2001-07-10 EP EP01951000A patent/EP1301201B1/en not_active Revoked
- 2001-07-10 EP EP18194771.4A patent/EP3449934B1/en not_active Expired - Lifetime
- 2001-07-10 PT PT70010913T patent/PT1782825E/pt unknown
- 2001-07-10 SI SI200131068T patent/SI3108895T1/sl unknown
- 2001-07-10 JP JP2002511773A patent/JP5113312B2/ja not_active Expired - Lifetime
- 2001-07-10 DK DK07001091.3T patent/DK1782825T3/da active
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- 2001-07-10 DK DK18194771.4T patent/DK3449934T3/da active
- 2001-07-10 CA CA002416492A patent/CA2416492C/en not_active Expired - Lifetime
- 2001-07-10 BR BR0113006-4A patent/BR0113006A/pt not_active Application Discontinuation
- 2001-07-10 ES ES07001091.3T patent/ES2523024T3/es not_active Expired - Lifetime
- 2001-07-10 DK DK14167882.1T patent/DK2767291T3/en active
- 2001-07-10 LT LTEP18194771.4T patent/LT3449934T/lt unknown
- 2001-07-10 EP EP14167882.1A patent/EP2767291B1/en not_active Expired - Lifetime
- 2001-07-10 US US09/902,461 patent/US7056712B2/en not_active Expired - Lifetime
- 2001-07-10 PT PT16178390T patent/PT3108895T/pt unknown
- 2001-07-10 PT PT141678821T patent/PT2767291T/pt unknown
- 2001-07-10 MX MXPA03000474A patent/MXPA03000474A/es active IP Right Grant
- 2001-07-10 ES ES16178390T patent/ES2700865T3/es not_active Expired - Lifetime
- 2001-07-10 DK DK16178390.7T patent/DK3108895T3/en active
- 2001-07-10 AU AU2001271941A patent/AU2001271941B2/en not_active Expired
- 2001-07-10 SI SI200131073T patent/SI3449934T1/sl unknown
- 2001-07-10 EP EP16178390.7A patent/EP3108895B1/en not_active Expired - Lifetime
- 2001-07-10 SI SI200131057A patent/SI2767291T1/sl unknown
- 2001-07-10 PT PT01951000T patent/PT1301201E/pt unknown
- 2001-07-10 LT LTEP14167882.1T patent/LT2767291T/lt unknown
- 2001-07-10 PT PT181947714T patent/PT3449934T/pt unknown
- 2001-07-10 WO PCT/US2001/021651 patent/WO2002005841A1/en not_active Ceased
- 2001-07-10 ES ES01951000T patent/ES2277931T3/es not_active Expired - Lifetime
- 2001-07-10 LT LTEP16178390.7T patent/LT3108895T/lt unknown
- 2001-07-10 DE DE60126947T patent/DE60126947T2/de not_active Expired - Lifetime
- 2001-07-10 AT AT01951000T patent/ATE355075T1/de active
- 2001-07-10 TW TW090116828A patent/TWI280882B/zh not_active IP Right Cessation
-
2005
- 2005-01-20 US US11/039,281 patent/US20050123531A1/en not_active Abandoned
-
2007
- 2007-08-13 US US11/889,457 patent/US20080175833A1/en not_active Abandoned
- 2007-12-03 JP JP2007312804A patent/JP2008081507A/ja active Pending
-
2009
- 2009-10-22 US US12/604,267 patent/US20100254966A1/en not_active Abandoned
-
2010
- 2010-02-10 AU AU2010200487A patent/AU2010200487A1/en not_active Ceased
-
2011
- 2011-03-30 US US13/064,556 patent/US20120058132A1/en not_active Abandoned
- 2011-08-09 JP JP2011173569A patent/JP2012006953A/ja active Pending
-
2012
- 2012-08-23 US US13/593,148 patent/US8900552B2/en not_active Expired - Fee Related
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2013
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