JP2025508841A5 - - Google Patents
Info
- Publication number
- JP2025508841A5 JP2025508841A5 JP2024550218A JP2024550218A JP2025508841A5 JP 2025508841 A5 JP2025508841 A5 JP 2025508841A5 JP 2024550218 A JP2024550218 A JP 2024550218A JP 2024550218 A JP2024550218 A JP 2024550218A JP 2025508841 A5 JP2025508841 A5 JP 2025508841A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- administration
- gaa
- ert
- 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 |
|---|---|---|---|
| US202263314093P | 2022-02-25 | 2022-02-25 | |
| US63/314,093 | 2022-02-25 | ||
| US202263319034P | 2022-03-11 | 2022-03-11 | |
| US63/319,034 | 2022-03-11 | ||
| US202263340972P | 2022-05-12 | 2022-05-12 | |
| US63/340,972 | 2022-05-12 | ||
| PCT/US2023/013713 WO2023164060A2 (en) | 2022-02-25 | 2023-02-23 | Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025508841A JP2025508841A (ja) | 2025-04-10 |
| JP2025508841A5 true JP2025508841A5 (https=) | 2026-03-03 |
Family
ID=87766839
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024550218A Pending JP2025508841A (ja) | 2022-02-25 | 2023-02-23 | Ｇａａ酵素補充療法の長期休止を伴うポンペ病を処置するための治療用アデノ随伴ウイルス |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20250163457A1 (https=) |
| EP (1) | EP4482516A4 (https=) |
| JP (1) | JP2025508841A (https=) |
| CN (1) | CN119095612A (https=) |
| AU (1) | AU2023224856A1 (https=) |
| CA (1) | CA3252501A1 (https=) |
| TW (1) | TW202342109A (https=) |
| WO (1) | WO2023164060A2 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025053118A1 (ja) * | 2023-09-04 | 2025-03-13 | アステラス製薬株式会社 | 短縮型ｃｍｖプロモーター配列、及び／又は短縮型ポリアデニル化配列を含む組換えアデノ随伴ウイルス |
| WO2025235807A1 (en) * | 2024-05-10 | 2025-11-13 | Solid Biosciences, Inc. | Compositions and methods to improve graft survival |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2001269923A1 (en) * | 2000-06-19 | 2002-01-02 | Genzyme Corporation | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases |
| US8679478B2 (en) * | 2010-10-04 | 2014-03-25 | Duke University | Methods of lysosomal storage disease therapy |
| EP3293259A1 (en) * | 2016-09-12 | 2018-03-14 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| US20200063160A1 (en) * | 2018-08-07 | 2020-02-27 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type ii |
| CN113423419A (zh) * | 2019-01-04 | 2021-09-21 | 桑格摩生物治疗股份有限公司 | 用于治疗法布里病的方法和组合物 |
| 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 |
| US20250213726A1 (en) * | 2022-03-18 | 2025-07-03 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications |
-
2023
- 2023-02-23 CN CN202380036185.9A patent/CN119095612A/zh active Pending
- 2023-02-23 EP EP23760645.4A patent/EP4482516A4/en active Pending
- 2023-02-23 AU AU2023224856A patent/AU2023224856A1/en active Pending
- 2023-02-23 WO PCT/US2023/013713 patent/WO2023164060A2/en not_active Ceased
- 2023-02-23 JP JP2024550218A patent/JP2025508841A/ja active Pending
- 2023-02-23 CA CA3252501A patent/CA3252501A1/en active Pending
- 2023-02-23 US US18/840,822 patent/US20250163457A1/en active Pending
- 2023-02-24 TW TW112107060A patent/TW202342109A/zh unknown
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