JP2013231081A5 - - Google Patents
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- Publication number
- JP2013231081A5 JP2013231081A5 JP2013162201A JP2013162201A JP2013231081A5 JP 2013231081 A5 JP2013231081 A5 JP 2013231081A5 JP 2013162201 A JP2013162201 A JP 2013162201A JP 2013162201 A JP2013162201 A JP 2013162201A JP 2013231081 A5 JP2013231081 A5 JP 2013231081A5
- Authority
- JP
- Japan
- Prior art keywords
- glycogenosis
- glucosidase
- type
- human
- medicament
- 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.)
- Granted
Links
- 239000003814 drug Substances 0.000 claims 15
- 208000007345 glycogen storage disease Diseases 0.000 claims 14
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims 9
- 102000045921 human GAA Human genes 0.000 claims 9
- 239000002253 acid Substances 0.000 claims 6
- 102000016679 alpha-Glucosidases Human genes 0.000 claims 6
- 108010028144 alpha-Glucosidases Proteins 0.000 claims 6
- 238000004113 cell culture Methods 0.000 claims 5
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims 5
- 208000031229 Cardiomyopathies Diseases 0.000 claims 4
- 241000282412 Homo Species 0.000 claims 4
- 239000000126 substance Substances 0.000 claims 4
- 230000037396 body weight Effects 0.000 claims 2
- 229940079593 drug Drugs 0.000 claims 2
- 238000002650 immunosuppressive therapy Methods 0.000 claims 2
- 238000009169 immunotherapy Methods 0.000 claims 2
- 230000000366 juvenile effect Effects 0.000 claims 1
- 239000002243 precursor Substances 0.000 claims 1
- 230000003442 weekly effect Effects 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US21923700P | 2000-07-18 | 2000-07-18 | |
| US60/219,237 | 2000-07-18 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2011173569A Division JP2012006953A (ja) | 2000-07-18 | 2011-08-09 | Ｉｉ型糖原病の治療 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014141336A Division JP2014185187A (ja) | 2000-07-18 | 2014-07-09 | Ｉｉ型糖原病の治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013231081A JP2013231081A (ja) | 2013-11-14 |
| JP2013231081A5 true JP2013231081A5 (enExample) | 2014-08-21 |
| JP5792774B2 JP5792774B2 (ja) | 2015-10-14 |
Family
ID=22818451
Family Applications (10)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2002511773A Expired - Lifetime JP5113312B2 (ja) | 2000-07-18 | 2001-07-10 | Ｉｉ型糖原病の治療 |
| JP2007312804A Pending JP2008081507A (ja) | 2000-07-18 | 2007-12-03 | Ｉｉ型糖原病の治療 |
| JP2011173569A Pending JP2012006953A (ja) | 2000-07-18 | 2011-08-09 | Ｉｉ型糖原病の治療 |
| JP2013162201A Expired - Lifetime JP5792774B2 (ja) | 2000-07-18 | 2013-08-05 | Ｉｉ型糖原病の治療 |
| JP2014141336A Pending JP2014185187A (ja) | 2000-07-18 | 2014-07-09 | Ｉｉ型糖原病の治療 |
| JP2016005132A Expired - Lifetime JP6163216B2 (ja) | 2000-07-18 | 2016-01-14 | Ｉｉ型糖原病の治療 |
| JP2017099515A Pending JP2017137357A (ja) | 2000-07-18 | 2017-05-19 | Ｉｉ型糖原病の治療 |
| JP2017201250A Pending JP2018002749A (ja) | 2000-07-18 | 2017-10-17 | Ｉｉ型糖原病の治療 |
| JP2018171154A Pending JP2018199721A (ja) | 2000-07-18 | 2018-09-13 | Ｉｉ型糖原病の治療 |
| JP2019202851A Pending JP2020019822A (ja) | 2000-07-18 | 2019-11-08 | Ｉｉ型糖原病の治療 |
Family Applications Before (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2002511773A Expired - Lifetime JP5113312B2 (ja) | 2000-07-18 | 2001-07-10 | Ｉｉ型糖原病の治療 |
| JP2007312804A Pending JP2008081507A (ja) | 2000-07-18 | 2007-12-03 | Ｉｉ型糖原病の治療 |
| JP2011173569A Pending JP2012006953A (ja) | 2000-07-18 | 2011-08-09 | Ｉｉ型糖原病の治療 |
Family Applications After (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014141336A Pending JP2014185187A (ja) | 2000-07-18 | 2014-07-09 | Ｉｉ型糖原病の治療 |
| JP2016005132A Expired - Lifetime JP6163216B2 (ja) | 2000-07-18 | 2016-01-14 | Ｉｉ型糖原病の治療 |
| JP2017099515A Pending JP2017137357A (ja) | 2000-07-18 | 2017-05-19 | Ｉｉ型糖原病の治療 |
| JP2017201250A Pending JP2018002749A (ja) | 2000-07-18 | 2017-10-17 | Ｉｉ型糖原病の治療 |
| JP2018171154A Pending JP2018199721A (ja) | 2000-07-18 | 2018-09-13 | Ｉｉ型糖原病の治療 |
| JP2019202851A Pending JP2020019822A (ja) | 2000-07-18 | 2019-11-08 | Ｉｉ型糖原病の治療 |
Country Status (17)
| Country | Link |
|---|---|
| US (10) | US7056712B2 (enExample) |
| EP (4) | EP3108895B1 (enExample) |
| JP (10) | JP5113312B2 (enExample) |
| AT (1) | ATE355075T1 (enExample) |
| AU (3) | AU2001271941B2 (enExample) |
| BR (1) | BR0113006A (enExample) |
| CA (1) | CA2416492C (enExample) |
| CY (2) | CY1120977T1 (enExample) |
| DE (1) | DE60126947T2 (enExample) |
| DK (5) | DK1301201T3 (enExample) |
| ES (5) | ES2599401T3 (enExample) |
| LT (3) | LT3449934T (enExample) |
| MX (1) | MXPA03000474A (enExample) |
| PT (5) | PT2767291T (enExample) |
| SI (3) | SI3449934T1 (enExample) |
| TW (1) | TWI280882B (enExample) |
| WO (1) | WO2002005841A1 (enExample) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2001271941B2 (en) * | 2000-07-18 | 2007-03-01 | Duke University | Treatment of glycogen storage disease type II |
| US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, 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 |
| US20040005309A1 (en) * | 2002-05-29 | 2004-01-08 | Symbiontics, 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 |
| WO2006002283A1 (en) * | 2004-06-21 | 2006-01-05 | Medtronic, Inc. | Medical systems and methods for delivering compositions to cells |
| WO2004064750A2 (en) | 2003-01-22 | 2004-08-05 | Duke University | Improved constructs for expressing lysosomal polypeptides |
| 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 |
| WO2008063511A2 (en) * | 2006-11-13 | 2008-05-29 | Zystor Therapeutics, Inc. | 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 |
| HUE025212T2 (en) * | 2008-07-08 | 2016-03-29 | Univ Duke | A method for treating glycogen storage disease |
| EP2475376B1 (en) | 2009-06-17 | 2016-03-30 | BioMarin Pharmaceutical Inc. | Formulations for lysosomal enzymes |
| US8809282B2 (en) | 2010-05-06 | 2014-08-19 | Duke University | Method of reducing titers of antibodies specific for a therapeutic agent |
| HK1220980A1 (zh) | 2013-02-20 | 2017-05-19 | Valerion Therapeutics, Llc | 用於治疗庞贝氏症的方法和组合物 |
| HK1222093A1 (zh) * | 2013-05-15 | 2017-06-23 | 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 |
| MY186336A (en) | 2014-09-30 | 2021-07-13 | 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 |
| US20180326021A1 (en) * | 2015-08-31 | 2018-11-15 | Duke University | Methods and Compositions for the Treatment of Cytoplasmic Glycogen Storage Disorders |
| US10940125B2 (en) | 2015-09-18 | 2021-03-09 | Duke University | Methods and compositions for the treatment of steatosis-associated disorders |
| CA3010205A1 (en) | 2015-12-30 | 2017-07-06 | Amicus Therapeutics, Inc. | Augmented acid alpha-glucosidase for the treatment of pompe disease |
| IL301319A (en) | 2016-03-30 | 2023-05-01 | Amicus Therapeutics Inc | Formulations that include recombinant acid alpha-glucosidase |
| MX2018011951A (es) | 2016-03-30 | 2019-02-13 | Amicus Therapeutics Inc | Metodo para seleccionar proteinas recombinantes ricas en m6p. |
| SG11201808176TA (en) | 2016-04-15 | 2018-10-30 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| EP3624831B1 (en) | 2017-05-15 | 2023-03-29 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| CA3076036A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| 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 |
| CN1300323A (zh) | 1998-05-13 | 2001-06-20 | 加州大学洛杉矶分校港口研究和教育院 | 重组α－L－艾杜糖苷酸酶,其生产和纯化的方法以及治疗其缺乏导致的疾病的方法 |
| AU3113700A (en) | 1998-12-07 | 2000-06-26 | Genzyme Corporation | Treatment of pompe's disease |
| US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| AU2001271941B2 (en) * | 2000-07-18 | 2007-03-01 | Duke University | 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 |
-
2001
- 2001-07-10 AU AU2001271941A patent/AU2001271941B2/en not_active Expired
- 2001-07-10 ES ES14167882.1T patent/ES2599401T3/es not_active Expired - Lifetime
- 2001-07-10 ES ES18194771T patent/ES2799882T3/es not_active Expired - Lifetime
- 2001-07-10 TW TW090116828A patent/TWI280882B/zh not_active IP Right Cessation
- 2001-07-10 SI SI200131073T patent/SI3449934T1/sl unknown
- 2001-07-10 ES ES01951000T patent/ES2277931T3/es not_active Expired - Lifetime
- 2001-07-10 LT LTEP18194771.4T patent/LT3449934T/lt unknown
- 2001-07-10 BR BR0113006-4A patent/BR0113006A/pt not_active Application Discontinuation
- 2001-07-10 PT PT141678821T patent/PT2767291T/pt unknown
- 2001-07-10 ES ES16178390T patent/ES2700865T3/es not_active Expired - Lifetime
- 2001-07-10 PT PT181947714T patent/PT3449934T/pt 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 DK DK01951000T patent/DK1301201T3/da active
- 2001-07-10 AU AU7194101A patent/AU7194101A/xx active Pending
- 2001-07-10 LT LTEP16178390.7T patent/LT3108895T/lt unknown
- 2001-07-10 SI SI200131068T patent/SI3108895T1/sl unknown
- 2001-07-10 PT PT16178390T patent/PT3108895T/pt unknown
- 2001-07-10 DK DK16178390.7T patent/DK3108895T3/en active
- 2001-07-10 EP EP18194771.4A patent/EP3449934B1/en not_active Expired - Lifetime
- 2001-07-10 PT PT01951000T patent/PT1301201E/pt unknown
- 2001-07-10 PT PT70010913T patent/PT1782825E/pt unknown
- 2001-07-10 LT LTEP14167882.1T patent/LT2767291T/lt unknown
- 2001-07-10 MX MXPA03000474A patent/MXPA03000474A/es active IP Right Grant
- 2001-07-10 DK DK14167882.1T patent/DK2767291T3/en active
- 2001-07-10 CA CA002416492A patent/CA2416492C/en not_active Expired - Lifetime
- 2001-07-10 ES ES07001091.3T patent/ES2523024T3/es not_active Expired - Lifetime
- 2001-07-10 DE DE60126947T patent/DE60126947T2/de not_active Expired - Lifetime
- 2001-07-10 DK DK18194771.4T patent/DK3449934T3/da active
- 2001-07-10 WO PCT/US2001/021651 patent/WO2002005841A1/en not_active Ceased
- 2001-07-10 US US09/902,461 patent/US7056712B2/en not_active Expired - Lifetime
- 2001-07-10 AT AT01951000T patent/ATE355075T1/de active
- 2001-07-10 EP EP14167882.1A patent/EP2767291B1/en not_active Expired - Lifetime
- 2001-07-10 EP EP01951000A patent/EP1301201B1/en not_active Revoked
- 2001-07-10 DK DK07001091.3T patent/DK1782825T3/da active
- 2001-07-10 JP JP2002511773A patent/JP5113312B2/ja not_active Expired - Lifetime
-
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
-
2013
- 2013-08-05 JP JP2013162201A patent/JP5792774B2/ja not_active Expired - Lifetime
-
2014
- 2014-07-09 JP JP2014141336A patent/JP2014185187A/ja active Pending
- 2014-09-12 US US14/485,071 patent/US9370556B2/en not_active Expired - Fee Related
-
2016
- 2016-01-14 JP JP2016005132A patent/JP6163216B2/ja not_active Expired - Lifetime
- 2016-03-10 US US15/066,211 patent/US9907839B2/en not_active Expired - Fee Related
-
2017
- 2017-05-19 JP JP2017099515A patent/JP2017137357A/ja active Pending
- 2017-10-17 JP JP2017201250A patent/JP2018002749A/ja active Pending
-
2018
- 2018-01-19 US US15/876,086 patent/US10792341B2/en not_active Expired - Fee Related
- 2018-09-13 JP JP2018171154A patent/JP2018199721A/ja active Pending
- 2018-12-07 CY CY181101308T patent/CY1120977T1/el unknown
-
2019
- 2019-11-08 JP JP2019202851A patent/JP2020019822A/ja active Pending
-
2020
- 2020-06-24 CY CY20201100587T patent/CY1123084T1/el unknown
- 2020-07-27 US US16/940,054 patent/US20210169996A1/en not_active Abandoned
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