JP2014513952A5 - - Google Patents
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- Publication number
- JP2014513952A5 JP2014513952A5 JP2014506579A JP2014506579A JP2014513952A5 JP 2014513952 A5 JP2014513952 A5 JP 2014513952A5 JP 2014506579 A JP2014506579 A JP 2014506579A JP 2014506579 A JP2014506579 A JP 2014506579A JP 2014513952 A5 JP2014513952 A5 JP 2014513952A5
- Authority
- JP
- Japan
- Prior art keywords
- polypeptide
- amino acids
- kda
- seq
- polypeptide according
- 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.)
- Pending
Links
- 229920001184 polypeptide Polymers 0.000 claims 13
- 102000004196 processed proteins & peptides Human genes 0.000 claims 13
- 108090000765 processed proteins & peptides Proteins 0.000 claims 13
- 150000001413 amino acids Chemical class 0.000 claims 9
- 238000012986 modification Methods 0.000 claims 6
- 230000004048 modification Effects 0.000 claims 6
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims 3
- 239000012634 fragment Substances 0.000 claims 3
- 102000045921 human GAA Human genes 0.000 claims 3
- 230000002209 hydrophobic effect Effects 0.000 claims 3
- 229920002527 Glycogen Polymers 0.000 claims 2
- 238000009825 accumulation Methods 0.000 claims 2
- 229940096919 glycogen Drugs 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 108091005804 Peptidases Proteins 0.000 claims 1
- 239000004365 Protease Substances 0.000 claims 1
- 102100037486 Reverse transcriptase/ribonuclease H Human genes 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 208000007345 glycogen storage disease Diseases 0.000 claims 1
- 238000003780 insertion Methods 0.000 claims 1
- 230000037431 insertion Effects 0.000 claims 1
- 230000002132 lysosomal effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161478336P | 2011-04-22 | 2011-04-22 | |
| US61/478,336 | 2011-04-22 | ||
| PCT/US2012/034479 WO2012145644A1 (en) | 2011-04-22 | 2012-04-20 | Modified acid alpha glucosidase with accelerated processing |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016185075A Division JP2017035091A (ja) | 2011-04-22 | 2016-09-23 | 促進されたプロセシングを備えた修飾された酸性アルファグルコシダーゼ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2014513952A JP2014513952A (ja) | 2014-06-19 |
| JP2014513952A5 true JP2014513952A5 (enExample) | 2015-05-28 |
Family
ID=46000406
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014506579A Pending JP2014513952A (ja) | 2011-04-22 | 2012-04-20 | 促進されたプロセシングを備えた修飾された酸性アルファグルコシダーゼ |
| JP2016185075A Pending JP2017035091A (ja) | 2011-04-22 | 2016-09-23 | 促進されたプロセシングを備えた修飾された酸性アルファグルコシダーゼ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016185075A Pending JP2017035091A (ja) | 2011-04-22 | 2016-09-23 | 促進されたプロセシングを備えた修飾された酸性アルファグルコシダーゼ |
Country Status (24)
| Country | Link |
|---|---|
| US (1) | US20140186326A1 (enExample) |
| EP (1) | EP2699676A1 (enExample) |
| JP (2) | JP2014513952A (enExample) |
| KR (1) | KR20140037082A (enExample) |
| CN (1) | CN103797115A (enExample) |
| AU (1) | AU2012245280A1 (enExample) |
| BR (1) | BR112013026976A2 (enExample) |
| CA (1) | CA2833371A1 (enExample) |
| CL (1) | CL2013003010A1 (enExample) |
| CO (1) | CO6811810A2 (enExample) |
| CR (1) | CR20130555A (enExample) |
| EC (1) | ECSP13013036A (enExample) |
| GT (1) | GT201300252A (enExample) |
| IL (1) | IL228871A0 (enExample) |
| MA (1) | MA35125B1 (enExample) |
| MX (1) | MX2013012345A (enExample) |
| NI (1) | NI201300110A (enExample) |
| PE (1) | PE20140617A1 (enExample) |
| PH (1) | PH12013502149A1 (enExample) |
| RU (1) | RU2013151875A (enExample) |
| SG (2) | SG10201605874TA (enExample) |
| TN (1) | TN2013000427A1 (enExample) |
| WO (1) | WO2012145644A1 (enExample) |
| ZA (1) | ZA201307696B (enExample) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2014218854B2 (en) | 2013-02-20 | 2019-01-24 | Valerion Therapeutics, Llc | Methods and compositions for treatment of Pompe disease |
| HRP20250058T1 (hr) | 2014-09-30 | 2025-03-28 | Amicus Therapeutics, Inc. | Vrlo snažna kisela alfa-glukozidaza s pojačanim ugljikohidratima |
| EP3371593B1 (en) * | 2015-11-06 | 2020-10-14 | BioMarin Pharmaceutical Inc. | Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes |
| EA201891507A1 (ru) * | 2015-12-30 | 2018-12-28 | Амикус Терапьютикс, Инк. | Кислая альфа-глюкозидаза усиленного действия для лечения болезни помпе |
| PE20190127A1 (es) | 2016-03-30 | 2019-01-17 | Amicus Therapeutics Inc | Metodo para seleccionar proteinas recombinantes ricas en m6p |
| IL301319A (en) | 2016-03-30 | 2023-05-01 | Amicus Therapeutics Inc | Formulations that include recombinant acid alpha-glucosidase |
| EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| AU2017322376B2 (en) | 2016-09-12 | 2023-08-17 | Association Institut De Myologie | Acid-alpha glucosidase variants and uses thereof |
| EP3293259A1 (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 |
| WO2018213340A1 (en) | 2017-05-15 | 2018-11-22 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase |
| EP3635009A1 (en) | 2017-06-07 | 2020-04-15 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
| KR101942093B1 (ko) * | 2018-01-05 | 2019-01-24 | 인하대학교 산학협력단 | 만노시다제 억제제를 포함하는 고만노즈 타입 n-당쇄를 갖는 인간 리소좀 효소 생산용 조성물 |
| SG11202007363TA (en) | 2018-02-07 | 2020-08-28 | Regeneron Pharma | Methods and compositions for therapeutic protein delivery |
| CA3100021A1 (en) | 2018-05-17 | 2019-11-21 | Regeneron Pharmaceuticals, Inc. | Anti-cd63 antibodies, conjugates, and uses thereof |
| EP3996696A1 (en) | 2019-07-09 | 2022-05-18 | Genethon | Treatment of glycogen storage disease (gsd) |
| MX2022005916A (es) * | 2019-11-19 | 2022-08-04 | Asklepios Biopharmaceutical Inc | Virus adenoasociado terapeutico que comprende promotores especificos del higado para el tratamiento de la enfermedad de pompe y los trastornos liposomales. |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6071890A (en) | 1994-12-09 | 2000-06-06 | Genzyme Corporation | Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy |
| US5952516A (en) | 1997-05-08 | 1999-09-14 | Genzyme Corporation | Cationic amphiphiles containing multiplesteroid lipophilic groups |
| ES2268799T3 (es) | 1997-10-29 | 2007-03-16 | Genzyme Corporation | Terapia genica para la enfermedad de gaucher. |
| US6287857B1 (en) | 1998-02-09 | 2001-09-11 | Genzyme Corporation | Nucleic acid delivery vehicles |
| GB9807464D0 (en) * | 1998-04-07 | 1998-06-10 | Pharming Bv | Purification of human acid µ-glucosidase |
| DK1137762T3 (da) | 1998-12-07 | 2009-02-02 | Genzyme Corp | Behandling af Pompes sygdom |
| US7138262B1 (en) | 2000-08-18 | 2006-11-21 | Shire Human Genetic Therapies, Inc. | High mannose proteins and methods of making high mannose proteins |
| 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 |
| US7001994B2 (en) | 2001-01-18 | 2006-02-21 | Genzyme Corporation | Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins |
| JP4742191B2 (ja) * | 2001-06-14 | 2011-08-10 | 独立行政法人産業技術総合研究所 | 糖蛋白質およびその製造方法 |
| DK2266968T3 (da) | 2001-07-16 | 2013-04-08 | Genzyme Corp | Syntese af UDP-glucose: N-acylsphingosin-glucosyltransferase-hæmmere |
| US6835831B2 (en) | 2001-11-26 | 2004-12-28 | Genzyme Corporation | Diastereoselective synthesis of UDP-glucose: N-acylsphingosine glucosyltransferase inhibitors |
| US6916802B2 (en) | 2002-04-29 | 2005-07-12 | Genzyme Corporation | Amino ceramide-like compounds and therapeutic methods of use |
| PT1587923E (pt) * | 2003-01-22 | 2011-12-07 | Univ Duke | Construções melhoradas para a expressão de polipéptidos lisossomais |
| US20100196345A1 (en) | 2003-04-27 | 2010-08-05 | Protalix | Production of high mannose proteins in plant culture |
| CN107058313A (zh) | 2003-06-24 | 2017-08-18 | 建新公司 | 新的β‑肌动蛋白和RPS21启动子及其应用 |
| DE602005020745D1 (de) * | 2004-02-10 | 2010-06-02 | Zystor Therapeutics Inc | Saure alpha-glukosidase und fragmente davon |
| EP2099523A2 (en) * | 2006-11-13 | 2009-09-16 | ZyStor Therapeutics , Inc. | Methods for treating pompe disease |
| CN105879047A (zh) | 2008-12-16 | 2016-08-24 | 建新公司 | 寡糖-蛋白缀合物 |
| WO2010096369A1 (en) * | 2009-02-18 | 2010-08-26 | Amicus Therapeutics, Inc. | Mouse model for pompe disease and methods of use thereof |
-
2012
- 2012-04-20 BR BR112013026976A patent/BR112013026976A2/pt not_active IP Right Cessation
- 2012-04-20 CN CN201280030662.2A patent/CN103797115A/zh active Pending
- 2012-04-20 MX MX2013012345A patent/MX2013012345A/es not_active Application Discontinuation
- 2012-04-20 SG SG10201605874TA patent/SG10201605874TA/en unknown
- 2012-04-20 PE PE2013002377A patent/PE20140617A1/es not_active Application Discontinuation
- 2012-04-20 EP EP12717025.6A patent/EP2699676A1/en not_active Withdrawn
- 2012-04-20 CA CA2833371A patent/CA2833371A1/en not_active Abandoned
- 2012-04-20 US US14/113,360 patent/US20140186326A1/en not_active Abandoned
- 2012-04-20 WO PCT/US2012/034479 patent/WO2012145644A1/en not_active Ceased
- 2012-04-20 JP JP2014506579A patent/JP2014513952A/ja active Pending
- 2012-04-20 PH PH1/2013/502149A patent/PH12013502149A1/en unknown
- 2012-04-20 AU AU2012245280A patent/AU2012245280A1/en not_active Abandoned
- 2012-04-20 RU RU2013151875/10A patent/RU2013151875A/ru not_active Application Discontinuation
- 2012-04-20 SG SG2013076526A patent/SG194486A1/en unknown
- 2012-04-20 KR KR1020137029936A patent/KR20140037082A/ko not_active Withdrawn
-
2013
- 2013-10-14 IL IL228871A patent/IL228871A0/en unknown
- 2013-10-16 NI NI201300110A patent/NI201300110A/es unknown
- 2013-10-16 ZA ZA2013/07696A patent/ZA201307696B/en unknown
- 2013-10-17 CL CL2013003010A patent/CL2013003010A1/es unknown
- 2013-10-18 GT GT201300252A patent/GT201300252A/es unknown
- 2013-10-18 TN TNP2013000427A patent/TN2013000427A1/fr unknown
- 2013-10-29 CR CR20130555A patent/CR20130555A/es unknown
- 2013-11-14 MA MA36431A patent/MA35125B1/fr unknown
- 2013-11-20 CO CO13272849A patent/CO6811810A2/es not_active Application Discontinuation
- 2013-11-21 EC ECSP13013036 patent/ECSP13013036A/es unknown
-
2016
- 2016-09-23 JP JP2016185075A patent/JP2017035091A/ja active Pending
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