JP2025521111A5 - - Google Patents
Info
- Publication number
- JP2025521111A5 JP2025521111A5 JP2024568194A JP2024568194A JP2025521111A5 JP 2025521111 A5 JP2025521111 A5 JP 2025521111A5 JP 2024568194 A JP2024568194 A JP 2024568194A JP 2024568194 A JP2024568194 A JP 2024568194A JP 2025521111 A5 JP2025521111 A5 JP 2025521111A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- raav
- seq
- arsa
- expression cassette
- 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 |
|---|---|---|---|
| US202263342590P | 2022-05-16 | 2022-05-16 | |
| US63/342,590 | 2022-05-16 | ||
| US202363459564P | 2023-04-14 | 2023-04-14 | |
| US63/459,564 | 2023-04-14 | ||
| PCT/US2023/067013 WO2023225481A1 (en) | 2022-05-16 | 2023-05-15 | Methods of treating metachromatic leukodystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025521111A JP2025521111A (ja) | 2025-07-08 |
| JP2025521111A5 true JP2025521111A5 (cg-RX-API-DMAC10.html) | 2026-05-22 |
Family
ID=87155607
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024568194A Pending JP2025521111A (ja) | 2022-05-16 | 2023-05-15 | 異染性白質ジストロフィーを治療する方法 |
Country Status (12)
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN122206466A (zh) * | 2023-11-15 | 2026-06-12 | 建新公司 | 治疗神经变性障碍的方法 |
| WO2026142957A1 (en) * | 2024-12-24 | 2026-07-02 | Genzyme Corporation | Aav genomes encoding engineered human frataxin transgene |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| US7465583B2 (en) | 2000-06-01 | 2008-12-16 | The University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| US7510872B2 (en) | 2003-02-26 | 2009-03-31 | Nationwide Children's Hospital | Recombinant adeno-associated virus production |
| JP4559429B2 (ja) | 2003-05-21 | 2010-10-06 | ジェンザイム・コーポレーション | 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法 |
| US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
| JP5956331B2 (ja) | 2009-06-16 | 2016-07-27 | ジェンザイム・コーポレーション | 組換えａａｖベクターの改良された精製方法 |
| JP7715640B2 (ja) * | 2019-05-03 | 2025-07-30 | 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 | 異染性白質ジストロフィーの治療に有用な組成物 |
| PE20220233A1 (es) * | 2019-06-10 | 2022-02-07 | Homology Medicines Inc | Composiciones de virus adenoasociados para la transferencia del gen de arsa y metodos de uso de las mismas |
| CN115023242B (zh) | 2019-11-22 | 2025-03-14 | 费城儿童医院 | 腺相关病毒载体变体 |
-
2023
- 2023-05-15 CN CN202380040795.6A patent/CN119213120A/zh active Pending
- 2023-05-15 EP EP23738590.1A patent/EP4526439A1/en active Pending
- 2023-05-15 IL IL316984A patent/IL316984A/en unknown
- 2023-05-15 TW TW112117950A patent/TW202403049A/zh unknown
- 2023-05-15 CA CA3253612A patent/CA3253612A1/en active Pending
- 2023-05-15 US US18/317,772 patent/US20230398192A1/en active Pending
- 2023-05-15 AU AU2023273809A patent/AU2023273809A1/en active Pending
- 2023-05-15 KR KR1020247041243A patent/KR20250011933A/ko active Pending
- 2023-05-15 JP JP2024568194A patent/JP2025521111A/ja active Pending
- 2023-05-15 WO PCT/US2023/067013 patent/WO2023225481A1/en not_active Ceased
-
2024
- 2024-11-14 MX MX2024014114A patent/MX2024014114A/es unknown
- 2024-12-10 CO CONC2024/0016901A patent/CO2024016901A2/es unknown