JP2025510017A5 - - Google Patents
Info
- Publication number
- JP2025510017A5 JP2025510017A5 JP2024555349A JP2024555349A JP2025510017A5 JP 2025510017 A5 JP2025510017 A5 JP 2025510017A5 JP 2024555349 A JP2024555349 A JP 2024555349A JP 2024555349 A JP2024555349 A JP 2024555349A JP 2025510017 A5 JP2025510017 A5 JP 2025510017A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- aav vector
- recombinant aav
- nos
- signal peptide
- 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 |
|---|---|---|---|
| US202263321336P | 2022-03-18 | 2022-03-18 | |
| US63/321,336 | 2022-03-18 | ||
| US202263348862P | 2022-06-03 | 2022-06-03 | |
| US63/348,862 | 2022-06-03 | ||
| US202363444804P | 2023-02-10 | 2023-02-10 | |
| US63/444,804 | 2023-02-10 | ||
| PCT/US2023/015531 WO2023177885A2 (en) | 2022-03-18 | 2023-03-17 | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025510017A JP2025510017A (ja) | 2025-04-14 |
| JP2025510017A5 true JP2025510017A5 (enExample) | 2026-03-26 |
Family
ID=88024211
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024555349A Pending JP2025510017A (ja) | 2022-03-18 | 2023-03-17 | シグナルペプチド改変を有する、ポンペ病を処置するためのアルファ－グルコシダーゼ（ｇａａ）をコードするコドン最適化核酸を使用する治療用アデノ随伴ウイルス |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20250213726A1 (enExample) |
| EP (1) | EP4493704A4 (enExample) |
| JP (1) | JP2025510017A (enExample) |
| KR (1) | KR20250005569A (enExample) |
| CN (1) | CN119213137A (enExample) |
| AU (1) | AU2023235203A1 (enExample) |
| CA (1) | CA3254578A1 (enExample) |
| IL (1) | IL315711A (enExample) |
| MX (1) | MX2024011396A (enExample) |
| TW (1) | TW202403048A (enExample) |
| WO (1) | WO2023177885A2 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119095612A (zh) * | 2022-02-25 | 2024-12-06 | 阿斯克肋匹奥生物制药公司 | 用于在长期停止gaa酶替代疗法的情况下治疗庞贝病的治疗性腺相关病毒 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2371913T3 (es) * | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
| EP3293259A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| AU2019381803A1 (en) * | 2018-11-16 | 2021-06-10 | AskBio Inc. | Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence |
| 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 |
-
2023
- 2023-03-17 US US18/848,389 patent/US20250213726A1/en active Pending
- 2023-03-17 EP EP23771464.7A patent/EP4493704A4/en active Pending
- 2023-03-17 TW TW112110096A patent/TW202403048A/zh unknown
- 2023-03-17 WO PCT/US2023/015531 patent/WO2023177885A2/en not_active Ceased
- 2023-03-17 CN CN202380041220.6A patent/CN119213137A/zh active Pending
- 2023-03-17 JP JP2024555349A patent/JP2025510017A/ja active Pending
- 2023-03-17 KR KR1020247032558A patent/KR20250005569A/ko active Pending
- 2023-03-17 AU AU2023235203A patent/AU2023235203A1/en active Pending
- 2023-03-17 CA CA3254578A patent/CA3254578A1/en active Pending
- 2023-03-17 IL IL315711A patent/IL315711A/en unknown
-
2024
- 2024-09-17 MX MX2024011396A patent/MX2024011396A/es unknown
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