JP2023542131A5 - - Google Patents
Info
- Publication number
- JP2023542131A5 JP2023542131A5 JP2023517283A JP2023517283A JP2023542131A5 JP 2023542131 A5 JP2023542131 A5 JP 2023542131A5 JP 2023517283 A JP2023517283 A JP 2023517283A JP 2023517283 A JP2023517283 A JP 2023517283A JP 2023542131 A5 JP2023542131 A5 JP 2023542131A5
- Authority
- JP
- Japan
- Prior art keywords
- cedna vector
- nucleic acid
- acid sequence
- itrs
- adjacent
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063078954P | 2020-09-16 | 2020-09-16 | |
| US63/078,954 | 2020-09-16 | ||
| PCT/US2021/050695 WO2022061000A1 (en) | 2020-09-16 | 2021-09-16 | Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023542131A JP2023542131A (ja) | 2023-10-05 |
| JP2023542131A5 true JP2023542131A5 (https=) | 2024-09-25 |
Family
ID=80775707
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023517283A Pending JP2023542131A (ja) | 2020-09-16 | 2021-09-16 | フェニルアラニンヒドロキシラーゼ（ｐａｈ）を発現させるための閉端ｄｎａベクター及びその使用 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240026374A1 (https=) |
| EP (1) | EP4214309A4 (https=) |
| JP (1) | JP2023542131A (https=) |
| KR (1) | KR20230066615A (https=) |
| CN (1) | CN116507730A (https=) |
| AU (1) | AU2021345187A1 (https=) |
| CA (1) | CA3191765A1 (https=) |
| IL (1) | IL301196A (https=) |
| MX (1) | MX2023003019A (https=) |
| WO (1) | WO2022061000A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2023003805A (es) | 2020-10-01 | 2023-04-12 | Genzyme Corp | Casete de expresion de pah humana para el tratamiento de la pku mediante terapia de reemplazo genico dirigida al higado. |
| GB202307239D0 (en) * | 2023-05-15 | 2023-06-28 | Evox Therapeutics Ltd | Gene therapies for Phenylketonuria |
| WO2025064507A1 (en) * | 2023-09-19 | 2025-03-27 | Poseida Therapeutics, Inc. | Compositions and methods for integration of viral vectors |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11007280B2 (en) * | 2015-03-17 | 2021-05-18 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for FVIII and FIX |
| KR102738752B1 (ko) * | 2016-12-30 | 2024-12-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
| CA3075168A1 (en) * | 2017-09-08 | 2019-03-14 | Generation Bio Co. | Modified closed-ended dna (cedna) |
| CA3084185A1 (en) * | 2017-12-06 | 2019-06-13 | Generation Bio Co. | Gene editing using a modified closed-ended dna (cedna) |
| US12442015B2 (en) * | 2018-01-19 | 2025-10-14 | Generation Bio Co. | Closed-ended DNA vectors obtainable from cell-free synthesis and process for obtaining ceDNA vectors |
| MA51915A (fr) * | 2018-02-22 | 2020-12-30 | Generation Bio Co | Expression régulée de transgènes à l'aide de vecteurs adn à extrémités fermées (cedna) |
| TW202016314A (zh) * | 2018-05-09 | 2020-05-01 | 美商拜奧馬林製藥公司 | 治療苯丙酮尿症之方法 |
| CN113454232B (zh) * | 2019-02-15 | 2024-10-29 | 世代生物公司 | 封闭端dna（cedna）产生中对rep蛋白活性的调节 |
| EP3938515A4 (en) * | 2019-03-13 | 2022-12-28 | Generation Bio Co. | NON-VIRAL DNA VECTORS AND THEIR USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) THERAPEUTIC AGENTS |
| BR112021024124A2 (pt) * | 2019-05-31 | 2022-01-11 | American Gene Tech Int Inc | Expressão otimizada de fenilalanina hidroxilase |
-
2021
- 2021-09-16 EP EP21870225.6A patent/EP4214309A4/en active Pending
- 2021-09-16 US US18/025,926 patent/US20240026374A1/en active Pending
- 2021-09-16 CN CN202180076781.0A patent/CN116507730A/zh active Pending
- 2021-09-16 MX MX2023003019A patent/MX2023003019A/es unknown
- 2021-09-16 JP JP2023517283A patent/JP2023542131A/ja active Pending
- 2021-09-16 AU AU2021345187A patent/AU2021345187A1/en active Pending
- 2021-09-16 CA CA3191765A patent/CA3191765A1/en active Pending
- 2021-09-16 WO PCT/US2021/050695 patent/WO2022061000A1/en not_active Ceased
- 2021-09-16 KR KR1020237012572A patent/KR20230066615A/ko active Pending
- 2021-09-16 IL IL301196A patent/IL301196A/en unknown
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