JPWO2020186150A5 - - Google Patents
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- Publication number
- JPWO2020186150A5 JPWO2020186150A5 JP2021554695A JP2021554695A JPWO2020186150A5 JP WO2020186150 A5 JPWO2020186150 A5 JP WO2020186150A5 JP 2021554695 A JP2021554695 A JP 2021554695A JP 2021554695 A JP2021554695 A JP 2021554695A JP WO2020186150 A5 JPWO2020186150 A5 JP WO2020186150A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- cedna vector
- itrs
- stem
- nucleic acid
- 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 |
|---|---|---|---|
| US201962817771P | 2019-03-13 | 2019-03-13 | |
| US62/817,771 | 2019-03-13 | ||
| US201962857514P | 2019-06-05 | 2019-06-05 | |
| US62/857,514 | 2019-06-05 | ||
| PCT/US2020/022595 WO2020186150A2 (en) | 2019-03-13 | 2020-03-13 | Non-viral dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) therapeutics |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022525302A JP2022525302A (ja) | 2022-05-12 |
| JPWO2020186150A5 true JPWO2020186150A5 (ko) | 2023-03-22 |
Family
ID=72426457
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021554695A Pending JP2022525302A (ja) | 2019-03-13 | 2020-03-13 | フェニルアラニンヒドロキシラーゼ（ｐａｈ）治療薬を発現するための非ウイルス性ｄｎａベクターおよびその使用 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230024354A1 (ko) |
| EP (1) | EP3938515A4 (ko) |
| JP (1) | JP2022525302A (ko) |
| KR (1) | KR20210149702A (ko) |
| CN (1) | CN113874508A (ko) |
| AU (1) | AU2020235121A1 (ko) |
| BR (1) | BR112021017852A2 (ko) |
| CA (1) | CA3133330A1 (ko) |
| IL (1) | IL286283A (ko) |
| MX (1) | MX2021011037A (ko) |
| SG (1) | SG11202109830TA (ko) |
| WO (1) | WO2020186150A2 (ko) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4189098A1 (en) | 2020-07-27 | 2023-06-07 | Anjarium Biosciences AG | Compositions of dna molecules, methods of making therefor, and methods of use thereof |
| WO2022061000A1 (en) * | 2020-09-16 | 2022-03-24 | Generation Bio Co. | Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah) |
| CN115772503B (zh) * | 2022-08-16 | 2023-10-17 | 广东省南山医药创新研究院 | 一种表达pah的基因修饰细胞药物及其制备方法与应用 |
| CN116656740A (zh) * | 2023-05-30 | 2023-08-29 | 浙江大学 | 一种腺相关病毒基因治疗载体及其在制备治疗苯丙酮尿症的药物中的应用 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007149852A2 (en) * | 2006-06-19 | 2007-12-27 | Asklepios Biopharmaceutical, Inc. | Modified factor viii and factor ix genes and vectors for gene therapy |
| ES2898337T3 (es) * | 2016-03-03 | 2022-03-07 | Univ Massachusetts | ADN dúplex lineal de extremos cerrados para la transferencia de genes no víricos |
| KR20190100318A (ko) * | 2016-12-30 | 2019-08-28 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
| CA3056182A1 (en) * | 2017-03-16 | 2018-09-20 | Pfizer Inc. | Tyrosine prototrophy |
| US11820999B2 (en) * | 2017-04-03 | 2023-11-21 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| WO2018222925A1 (en) * | 2017-05-31 | 2018-12-06 | Ultragenyx Pharmaceutical Inc. | Therapeutics for phenylketonuria |
| TW201920255A (zh) * | 2017-08-09 | 2019-06-01 | 美商生物化學醫療公司 | 核酸分子及其用途 |
-
2020
- 2020-03-13 KR KR1020217029544A patent/KR20210149702A/ko unknown
- 2020-03-13 BR BR112021017852A patent/BR112021017852A2/pt unknown
- 2020-03-13 CN CN202080035845.8A patent/CN113874508A/zh active Pending
- 2020-03-13 JP JP2021554695A patent/JP2022525302A/ja active Pending
- 2020-03-13 SG SG11202109830T patent/SG11202109830TA/en unknown
- 2020-03-13 AU AU2020235121A patent/AU2020235121A1/en active Pending
- 2020-03-13 EP EP20768953.0A patent/EP3938515A4/en active Pending
- 2020-03-13 MX MX2021011037A patent/MX2021011037A/es unknown
- 2020-03-13 CA CA3133330A patent/CA3133330A1/en active Pending
- 2020-03-13 US US17/437,172 patent/US20230024354A1/en active Pending
- 2020-03-13 WO PCT/US2020/022595 patent/WO2020186150A2/en unknown
-
2021
- 2021-09-12 IL IL286283A patent/IL286283A/en unknown
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