JP2022525302A5 - - Google Patents
Info
- Publication number
- JP2022525302A5 JP2022525302A5 JP2021554695A JP2021554695A JP2022525302A5 JP 2022525302 A5 JP2022525302 A5 JP 2022525302A5 JP 2021554695 A JP2021554695 A JP 2021554695A JP 2021554695 A JP2021554695 A JP 2021554695A JP 2022525302 A5 JP2022525302 A5 JP 2022525302A5
- Authority
- JP
- Japan
- Prior art keywords
- cedna vector
- prior
- itrs
- vector according
- 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.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024193194A JP2025016685A (ja) | 2019-03-13 | 2024-11-01 | 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 |
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024193194A Division JP2025016685A (ja) | 2019-03-13 | 2024-11-01 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022525302A JP2022525302A (ja) | 2022-05-12 |
| JP2022525302A5 true JP2022525302A5 (https=) | 2023-03-22 |
| JP7583732B2 JP7583732B2 (ja) | 2024-11-14 |
Family
ID=72426457
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021554695A Active JP7583732B2 (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 |
| JP2024193194A Pending JP2025016685A (ja) | 2019-03-13 | 2024-11-01 | 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024193194A Pending JP2025016685A (ja) | 2019-03-13 | 2024-11-01 | 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) | US12415002B2 (https=) |
| EP (1) | EP3938515A4 (https=) |
| JP (2) | JP7583732B2 (https=) |
| KR (1) | KR20210149702A (https=) |
| CN (1) | CN113874508A (https=) |
| AU (1) | AU2020235121A1 (https=) |
| BR (1) | BR112021017852A2 (https=) |
| CA (1) | CA3133330A1 (https=) |
| IL (1) | IL286283A (https=) |
| MX (1) | MX2021011037A (https=) |
| SG (1) | SG11202109830TA (https=) |
| WO (1) | WO2020186150A2 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4165196A1 (en) * | 2020-06-11 | 2023-04-19 | Sangamo Therapeutics, Inc. | Methods and compositions for expressing phenylalanine hydroxylase |
| JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Ｄｎａ分子の組成物、その作製方法、及びその使用方法 |
| EP4214309A4 (en) * | 2020-09-16 | 2025-07-16 | Generation Bio Co | CLOSED-END DNA VECTORS AND RELATED USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) |
| GB202014751D0 (en) * | 2020-09-18 | 2020-11-04 | Lightbio Ltd | Targeting vector |
| 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. |
| 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 |
| AU2024257493A1 (en) * | 2023-04-18 | 2025-12-04 | Siren Biotechnology, Inc. | Adeno-associated viral vector compositions and methods of use |
| 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 |
| CN119876192B (zh) * | 2024-07-02 | 2025-11-07 | 浙江大学滨江研究院 | 用于治疗苯丙酮尿症的多核苷酸 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2535877T3 (es) | 2006-06-19 | 2015-05-18 | Asklepios Biopharmaceutical, Inc. | Genes de Factor VIII y Factor IX modificados y vectores para la terapia génica |
| EP2500434A1 (en) | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| MX2018010633A (es) | 2016-03-03 | 2019-06-13 | Univ Massachusetts | Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral. |
| KR102738752B1 (ko) | 2016-12-30 | 2024-12-06 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 페닐케톤뇨증을 치료하기 위한 유전자 치료 |
| WO2018167621A1 (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 |
| EP3630200A4 (en) | 2017-05-31 | 2021-02-24 | Arcturus Therapeutics, Inc. | THERAPEUTICS FOR PHENYLKETONURIA |
| NZ762100A (en) * | 2017-08-09 | 2025-12-19 | Bioverativ Therapeutics Inc | Nucleic acid molecules and uses thereof |
-
2020
- 2020-03-13 EP EP20768953.0A patent/EP3938515A4/en active Pending
- 2020-03-13 JP JP2021554695A patent/JP7583732B2/ja active Active
- 2020-03-13 US US17/437,172 patent/US12415002B2/en active Active
- 2020-03-13 MX MX2021011037A patent/MX2021011037A/es unknown
- 2020-03-13 BR BR112021017852A patent/BR112021017852A2/pt unknown
- 2020-03-13 AU AU2020235121A patent/AU2020235121A1/en active Pending
- 2020-03-13 CN CN202080035845.8A patent/CN113874508A/zh active Pending
- 2020-03-13 KR KR1020217029544A patent/KR20210149702A/ko active Pending
- 2020-03-13 WO PCT/US2020/022595 patent/WO2020186150A2/en not_active Ceased
- 2020-03-13 CA CA3133330A patent/CA3133330A1/en active Pending
- 2020-03-13 SG SG11202109830T patent/SG11202109830TA/en unknown
-
2021
- 2021-09-12 IL IL286283A patent/IL286283A/en unknown
-
2024
- 2024-11-01 JP JP2024193194A patent/JP2025016685A/ja active Pending
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