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 (https=) | 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 (https=) |
| EP (1) | EP2699676A1 (https=) |
| JP (2) | JP2014513952A (https=) |
| KR (1) | KR20140037082A (https=) |
| CN (1) | CN103797115A (https=) |
| AU (1) | AU2012245280A1 (https=) |
| BR (1) | BR112013026976A2 (https=) |
| CA (1) | CA2833371A1 (https=) |
| CL (1) | CL2013003010A1 (https=) |
| CO (1) | CO6811810A2 (https=) |
| CR (1) | CR20130555A (https=) |
| EC (1) | ECSP13013036A (https=) |
| GT (1) | GT201300252A (https=) |
| IL (1) | IL228871A0 (https=) |
| MA (1) | MA35125B1 (https=) |
| MX (1) | MX2013012345A (https=) |
| NI (1) | NI201300110A (https=) |
| PE (1) | PE20140617A1 (https=) |
| PH (1) | PH12013502149A1 (https=) |
| RU (1) | RU2013151875A (https=) |
| SG (2) | SG10201605874TA (https=) |
| TN (1) | TN2013000427A1 (https=) |
| WO (1) | WO2012145644A1 (https=) |
| ZA (1) | ZA201307696B (https=) |
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 |
| CN114540327B (zh) | 2014-09-30 | 2026-03-24 | 阿米库斯治疗学公司 | 具有增强的碳水化合物的高强度酸性α-葡糖苷酶 |
| US11262348B2 (en) | 2015-11-06 | 2022-03-01 | Biomarin Pharmaceutical Inc. | Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes |
| KR20250007693A (ko) * | 2015-12-30 | 2025-01-14 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| AU2017239641A1 (en) | 2016-03-30 | 2018-10-18 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| CA3019354A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
| EP3293260A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293259A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| WO2018046774A1 (en) | 2016-09-12 | 2018-03-15 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| EP3293203A1 (en) | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| SMT202300187T1 (it) | 2017-05-15 | 2023-07-20 | Amicus Therapeutics Inc | Alfa-glucosidasi acida umana ricombinante |
| DK3635009T3 (da) | 2017-06-07 | 2026-03-30 | Regeneron Pharma | Sammensætninger og fremgangsmåder til internalisering af enzymer |
| KR101942093B1 (ko) * | 2018-01-05 | 2019-01-24 | 인하대학교 산학협력단 | 만노시다제 억제제를 포함하는 고만노즈 타입 n-당쇄를 갖는 인간 리소좀 효소 생산용 조성물 |
| US12258597B2 (en) | 2018-02-07 | 2025-03-25 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| EP3996696A1 (en) | 2019-07-09 | 2022-05-18 | Genethon | Treatment of glycogen storage disease (gsd) |
| KR20220098384A (ko) * | 2019-11-19 | 2022-07-12 | 아스클레피오스 바이오파마슈티컬, 인크. | 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스 |
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 |
| IL135578A0 (en) | 1997-10-29 | 2001-05-20 | Genzyme Corp | Compositions and methods for treating lysosomal storage disease |
| 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 |
| ES2312222T3 (es) * | 1998-12-07 | 2009-02-16 | Genzyme Corporation | Tratamiento de la enfermedad de pompe. |
| US7138262B1 (en) | 2000-08-18 | 2006-11-21 | Shire Human Genetic Therapies, Inc. | High mannose proteins and methods of making high mannose proteins |
| US7001994B2 (en) | 2001-01-18 | 2006-02-21 | Genzyme Corporation | Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins |
| 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 |
| 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 |
| EP2067775B1 (en) | 2001-07-16 | 2012-04-25 | Genzyme Corporation | A N-Acylsphingosine glucosyltransferase inhibitor |
| AU2002348261A1 (en) | 2001-11-26 | 2003-06-10 | 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 |
| ES2371913T3 (es) * | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
| US20100196345A1 (en) | 2003-04-27 | 2010-08-05 | Protalix | Production of high mannose proteins in plant culture |
| HUE036449T2 (hu) | 2003-06-24 | 2018-07-30 | Genzyme Corp | Új béta-aktin és RPS21 promóterek és alkalmazásaik |
| CA2553955C (en) * | 2004-02-10 | 2012-08-28 | Zystor Therapeutics, Inc. | Acid alpha-glucosidase and fragments thereof |
| EP2099523A2 (en) * | 2006-11-13 | 2009-09-16 | ZyStor Therapeutics , Inc. | Methods for treating pompe disease |
| CN102369023A (zh) | 2008-12-16 | 2012-03-07 | 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 MX MX2013012345A patent/MX2013012345A/es not_active Application Discontinuation
- 2012-04-20 CN CN201280030662.2A patent/CN103797115A/zh active Pending
- 2012-04-20 JP JP2014506579A patent/JP2014513952A/ja active Pending
- 2012-04-20 AU AU2012245280A patent/AU2012245280A1/en not_active Abandoned
- 2012-04-20 WO PCT/US2012/034479 patent/WO2012145644A1/en not_active Ceased
- 2012-04-20 EP EP12717025.6A patent/EP2699676A1/en not_active Withdrawn
- 2012-04-20 RU RU2013151875/10A patent/RU2013151875A/ru not_active Application Discontinuation
- 2012-04-20 SG SG10201605874TA patent/SG10201605874TA/en unknown
- 2012-04-20 KR KR1020137029936A patent/KR20140037082A/ko not_active Withdrawn
- 2012-04-20 PE PE2013002377A patent/PE20140617A1/es not_active Application Discontinuation
- 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 SG SG2013076526A patent/SG194486A1/en unknown
- 2012-04-20 BR BR112013026976A patent/BR112013026976A2/pt not_active IP Right Cessation
- 2012-04-20 PH PH1/2013/502149A patent/PH12013502149A1/en unknown
-
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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