JP2023526923A5 - - Google Patents
Info
- Publication number
- JP2023526923A5 JP2023526923A5 JP2022569264A JP2022569264A JP2023526923A5 JP 2023526923 A5 JP2023526923 A5 JP 2023526923A5 JP 2022569264 A JP2022569264 A JP 2022569264A JP 2022569264 A JP2022569264 A JP 2022569264A JP 2023526923 A5 JP2023526923 A5 JP 2023526923A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- pharmaceutical composition
- patient
- composition according
- approximately
- 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 (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063024941P | 2020-05-14 | 2020-05-14 | |
| US63/024,941 | 2020-05-14 | ||
| US202063109677P | 2020-11-04 | 2020-11-04 | |
| US63/109,677 | 2020-11-04 | ||
| US202163180379P | 2021-04-27 | 2021-04-27 | |
| US63/180,379 | 2021-04-27 | ||
| PCT/US2021/032451 WO2021231863A1 (en) | 2020-05-14 | 2021-05-14 | Compositions useful for treatment of pompe disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023526923A JP2023526923A (ja) | 2023-06-26 |
| JP2023526923A5 true JP2023526923A5 (https=) | 2024-05-23 |
Family
ID=78525047
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022569264A Pending JP2023526923A (ja) | 2020-05-14 | 2021-05-14 | ポンペ病の治療に有用な組成物 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20230173108A1 (https=) |
| EP (1) | EP4149519A4 (https=) |
| JP (1) | JP2023526923A (https=) |
| KR (1) | KR20230010255A (https=) |
| CN (1) | CN115916334A (https=) |
| AU (1) | AU2021273273A1 (https=) |
| BR (1) | BR112022022704A2 (https=) |
| CA (1) | CA3177954A1 (https=) |
| CL (1) | CL2022003132A1 (https=) |
| CO (1) | CO2022017224A2 (https=) |
| IL (1) | IL298178A (https=) |
| MX (1) | MX2022014255A (https=) |
| WO (1) | WO2021231863A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024003687A1 (en) * | 2022-06-28 | 2024-01-04 | Pfizer Inc. | Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy |
| WO2024151982A1 (en) * | 2023-01-13 | 2024-07-18 | Amicus Therapeutics, Inc. | Gene therapy constructs for the treatment of pompe disease |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2099523A2 (en) * | 2006-11-13 | 2009-09-16 | ZyStor Therapeutics , Inc. | Methods for treating pompe disease |
| US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| KR20230066482A (ko) * | 2012-03-07 | 2023-05-15 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 |
| PL3097197T3 (pl) * | 2014-01-21 | 2021-06-28 | Vrije Universiteit Brussel | Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie |
| US20170037431A1 (en) * | 2014-05-01 | 2017-02-09 | University Of Washington | In vivo Gene Engineering with Adenoviral Vectors |
| EP2960336A1 (en) * | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| WO2018178067A1 (en) * | 2017-03-27 | 2018-10-04 | Vrije Universiteit Brussel | Diaphragm-specific nucleic acid regulatory elements and methods and use thereof |
| RU2770922C2 (ru) * | 2017-09-20 | 2022-04-25 | 4Д Молекьюлар Терапьютикс Инк. | Капсиды вариантов аденоассоциированных вирусов и методы их применения |
| BR112020021962A2 (pt) * | 2018-04-30 | 2021-01-26 | Amicus Therapeutics, Inc. | construtos para terapia gênica e métodos de uso |
| JP7585233B2 (ja) * | 2019-04-30 | 2024-11-18 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ポンペ病の治療のために有用な組成物 |
-
2021
- 2021-05-14 WO PCT/US2021/032451 patent/WO2021231863A1/en not_active Ceased
- 2021-05-14 IL IL298178A patent/IL298178A/en unknown
- 2021-05-14 US US17/998,371 patent/US20230173108A1/en active Pending
- 2021-05-14 AU AU2021273273A patent/AU2021273273A1/en not_active Abandoned
- 2021-05-14 CN CN202180049054.5A patent/CN115916334A/zh active Pending
- 2021-05-14 MX MX2022014255A patent/MX2022014255A/es unknown
- 2021-05-14 BR BR112022022704A patent/BR112022022704A2/pt unknown
- 2021-05-14 KR KR1020227043641A patent/KR20230010255A/ko not_active Withdrawn
- 2021-05-14 CA CA3177954A patent/CA3177954A1/en active Pending
- 2021-05-14 JP JP2022569264A patent/JP2023526923A/ja active Pending
- 2021-05-14 EP EP21804731.4A patent/EP4149519A4/en not_active Withdrawn
-
2022
- 2022-11-10 CL CL2022003132A patent/CL2022003132A1/es unknown
- 2022-11-30 CO CONC2022/0017224A patent/CO2022017224A2/es unknown
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