JPWO2020223362A5 - - Google Patents
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- Publication number
- JPWO2020223362A5 JPWO2020223362A5 JP2021564730A JP2021564730A JPWO2020223362A5 JP WO2020223362 A5 JPWO2020223362 A5 JP WO2020223362A5 JP 2021564730 A JP2021564730 A JP 2021564730A JP 2021564730 A JP2021564730 A JP 2021564730A JP WO2020223362 A5 JPWO2020223362 A5 JP WO2020223362A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- expression cassette
- composition
- sequence
- seq
- 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
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962840911P | 2019-04-30 | 2019-04-30 | |
| US62/840,911 | 2019-04-30 | ||
| US201962913401P | 2019-10-10 | 2019-10-10 | |
| US62/913,401 | 2019-10-10 | ||
| PCT/US2020/030493 WO2020223362A1 (en) | 2019-04-30 | 2020-04-29 | Compositions useful for treatment of pompe disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022530824A JP2022530824A (ja) | 2022-07-01 |
| JPWO2020223362A5 true JPWO2020223362A5 (de) | 2023-05-08 |
Family
ID=73029176
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021565001A Pending JP2022530833A (ja) | 2019-04-30 | 2020-04-29 | ポンペ病の治療のために有用な組成物 |
| JP2021564730A Pending JP2022530824A (ja) | 2019-04-30 | 2020-04-29 | ポンペ病の治療のために有用な組成物 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021565001A Pending JP2022530833A (ja) | 2019-04-30 | 2020-04-29 | ポンペ病の治療のために有用な組成物 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20220193261A1 (de) |
| EP (2) | EP3980548A4 (de) |
| JP (2) | JP2022530833A (de) |
| KR (2) | KR20220008280A (de) |
| CN (2) | CN114072515A (de) |
| AU (2) | AU2020266552A1 (de) |
| BR (2) | BR112021021792A2 (de) |
| CA (2) | CA3134523A1 (de) |
| CL (2) | CL2021002754A1 (de) |
| CO (2) | CO2021016198A2 (de) |
| IL (2) | IL287522A (de) |
| MX (2) | MX2021013365A (de) |
| SG (2) | SG11202111400TA (de) |
| TW (1) | TW202100541A (de) |
| WO (2) | WO2020223362A1 (de) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2022014255A (es) * | 2020-05-14 | 2022-12-07 | Univ Pennsylvania | Composiciones utiles para el tratamiento de la enfermedad de pompe. |
| CA3183153A1 (en) | 2020-06-17 | 2021-12-23 | Christian HINDERER | Compositions and methods for treatment of gene therapy patients |
| WO2023086928A2 (en) * | 2021-11-12 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of mucopolysaccharidosis iiia |
| NL2031676B1 (en) * | 2022-04-22 | 2023-11-07 | Univ Erasmus Med Ct Rotterdam | Gene therapy for Pompe Disease |
| WO2024003687A1 (en) * | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| CN116693633B (zh) * | 2023-02-21 | 2023-12-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040248262A1 (en) * | 2003-01-22 | 2004-12-09 | Koeberl Dwight D. | Constructs for expressing lysomal polypeptides |
| JP4914224B2 (ja) * | 2004-02-10 | 2012-04-11 | バイオマリン ファーマシューティカル インコーポレイテッド | 酸性αグルコシダーゼおよびそのフラグメント |
| WO2008063511A2 (en) * | 2006-11-13 | 2008-05-29 | Zystor Therapeutics, Inc. | Methods for treating pompe disease |
| EP2623599B1 (de) * | 2007-10-04 | 2019-01-02 | Roche Innovation Center Copenhagen A/S | Mikromir-Moleküle |
| US8466340B2 (en) * | 2009-02-18 | 2013-06-18 | Amicus Therapeutics, Inc. | Mouse model for pompe disease and methods of use thereof |
| WO2012070014A2 (en) * | 2010-11-26 | 2012-05-31 | Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. | Identification of novel cell surface markers for pancreatic progenitor cells and definite endodermal cells |
| HUE038172T2 (hu) * | 2011-05-27 | 2018-09-28 | Amicus Therapeutics Inc | Eljárások célzó peptidek rekombináns lizoszómális enzimekre történõ kapcsolására lizoszómális tárolási betegségek javított kezelésében |
| WO2013013019A2 (en) * | 2011-07-21 | 2013-01-24 | Alnylam Pharmaceuticals, Inc. | Lysosomal polypeptides, methods of making and using |
| EP3387137B1 (de) * | 2015-12-11 | 2021-02-03 | California Institute of Technology | Zielpeptide zum leiten von adeno-assoziierten viren (aavs) |
| EP3293260A1 (de) * | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
| KR20240001721A (ko) * | 2016-09-12 | 2024-01-03 | 제네똥 | 산-알파 글루코시다제 변이체 및 이의 용도 |
| EP3293259A1 (de) * | 2016-09-12 | 2018-03-14 | Genethon | Saure alpha-glucosidase-varianten und verwendungen davon |
| US11920149B2 (en) * | 2017-03-27 | 2024-03-05 | Vrije Universiteit Brussel | Diaphragm-specific nucleic acid regulatory elements and methods and use thereof |
-
2020
- 2020-04-29 KR KR1020217038223A patent/KR20220008280A/ko unknown
- 2020-04-29 AU AU2020266552A patent/AU2020266552A1/en active Pending
- 2020-04-29 BR BR112021021792A patent/BR112021021792A2/pt unknown
- 2020-04-29 AU AU2020266829A patent/AU2020266829A1/en active Pending
- 2020-04-29 CA CA3134523A patent/CA3134523A1/en active Pending
- 2020-04-29 MX MX2021013365A patent/MX2021013365A/es unknown
- 2020-04-29 KR KR1020217038231A patent/KR20220004696A/ko unknown
- 2020-04-29 EP EP20798851.0A patent/EP3980548A4/de active Pending
- 2020-04-29 US US17/606,414 patent/US20220193261A1/en active Pending
- 2020-04-29 TW TW109114314A patent/TW202100541A/zh unknown
- 2020-04-29 WO PCT/US2020/030493 patent/WO2020223362A1/en unknown
- 2020-04-29 CA CA3134485A patent/CA3134485A1/en active Pending
- 2020-04-29 WO PCT/US2020/030484 patent/WO2020223356A1/en unknown
- 2020-04-29 SG SG11202111400TA patent/SG11202111400TA/en unknown
- 2020-04-29 EP EP20799541.6A patent/EP3963063A4/de active Pending
- 2020-04-29 JP JP2021565001A patent/JP2022530833A/ja active Pending
- 2020-04-29 BR BR112021021720A patent/BR112021021720A2/pt unknown
- 2020-04-29 CN CN202080049015.0A patent/CN114072515A/zh active Pending
- 2020-04-29 MX MX2021013364A patent/MX2021013364A/es unknown
- 2020-04-29 US US17/606,410 patent/US20220193207A1/en active Pending
- 2020-04-29 CN CN202080048355.1A patent/CN114127275A/zh active Pending
- 2020-04-29 JP JP2021564730A patent/JP2022530824A/ja active Pending
- 2020-04-29 SG SG11202111380VA patent/SG11202111380VA/en unknown
-
2021
- 2021-10-20 CL CL2021002754A patent/CL2021002754A1/es unknown
- 2021-10-20 CL CL2021002755A patent/CL2021002755A1/es unknown
- 2021-10-24 IL IL287522A patent/IL287522A/en unknown
- 2021-10-24 IL IL287523A patent/IL287523A/en unknown
- 2021-11-29 CO CONC2021/0016198A patent/CO2021016198A2/es unknown
- 2021-11-29 CO CONC2021/0016200A patent/CO2021016200A2/es unknown
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