JP2022548800A5 - - Google Patents
Info
- Publication number
- JP2022548800A5 JP2022548800A5 JP2022543368A JP2022543368A JP2022548800A5 JP 2022548800 A5 JP2022548800 A5 JP 2022548800A5 JP 2022543368 A JP2022543368 A JP 2022543368A JP 2022543368 A JP2022543368 A JP 2022543368A JP 2022548800 A5 JP2022548800 A5 JP 2022548800A5
- Authority
- JP
- Japan
- Prior art keywords
- regulator
- nucleic acid
- nanoparticles
- fxn
- recombinant rna
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025135712A JP2025169372A (ja) | 2019-09-23 | 2025-08-18 | フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962904391P | 2019-09-23 | 2019-09-23 | |
| US62/904,391 | 2019-09-23 | ||
| PCT/US2020/052101 WO2021061698A1 (en) | 2019-09-23 | 2020-09-23 | Methods and compositions for modulating frataxin expression and treating friedrich's ataxia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025135712A Division JP2025169372A (ja) | 2019-09-23 | 2025-08-18 | フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022548800A JP2022548800A (ja) | 2022-11-21 |
| JP2022548800A5 true JP2022548800A5 (https=) | 2023-10-02 |
Family
ID=75166421
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022543368A Pending JP2022548800A (ja) | 2019-09-23 | 2020-09-23 | フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物 |
| JP2025135712A Pending JP2025169372A (ja) | 2019-09-23 | 2025-08-18 | フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025135712A Pending JP2025169372A (ja) | 2019-09-23 | 2025-08-18 | フラタキシン発現を調節し、フリードライヒ運動失調症を治療するための方法及び組成物 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20220348893A1 (https=) |
| EP (1) | EP4034660A4 (https=) |
| JP (2) | JP2022548800A (https=) |
| CN (1) | CN114787353A (https=) |
| AU (1) | AU2020352984A1 (https=) |
| CA (1) | CA3155417A1 (https=) |
| WO (1) | WO2021061698A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20190255106A1 (en) | 2016-09-07 | 2019-08-22 | Flagship Pioneering Inc. | Methods and compositions for modulating gene expression |
| CN114729376A (zh) | 2019-09-23 | 2022-07-08 | 欧米茄治疗公司 | 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法 |
| WO2023275892A1 (en) * | 2021-06-29 | 2023-01-05 | Council Of Scientific & Industrial Research | Engineered fncas9 and uses thereof |
| WO2024163678A2 (en) * | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6150091A (en) * | 1996-03-06 | 2000-11-21 | Baylor College Of Medicine | Direct molecular diagnosis of Friedreich ataxia |
| KR102043422B1 (ko) * | 2011-06-09 | 2019-11-11 | 큐알엔에이, 인크. | 프라탁신 (fxn)에 대한 자연 안티센스 전사체의 저해에 의한 프라탁신 (fxn) 관련된 질환의 치료 |
| EP2780376B1 (en) * | 2011-11-18 | 2017-03-08 | Université Laval | Methods and products for increasing frataxin levels and uses thereof |
| EP2954042B1 (en) * | 2013-02-07 | 2017-12-06 | The General Hospital Corporation | Tale transcriptional activators |
| AU2015234204A1 (en) * | 2014-03-20 | 2016-10-06 | Universite Laval | CRISPR-based methods and products for increasing frataxin levels and uses thereof |
| CN109476716A (zh) * | 2016-03-23 | 2019-03-15 | 加利福尼亚大学董事会 | 治疗线粒体障碍的方法 |
| EP4484443A3 (en) * | 2016-06-29 | 2025-03-26 | CRISPR Therapeutics AG | Materials and methods for treatment of friedreich ataxia and other related disorders |
| US11866469B2 (en) * | 2019-02-06 | 2024-01-09 | Klogenix Llc | DNA binding proteins and uses thereof |
-
2020
- 2020-09-23 US US17/754,051 patent/US20220348893A1/en active Pending
- 2020-09-23 CA CA3155417A patent/CA3155417A1/en active Pending
- 2020-09-23 CN CN202080078749.1A patent/CN114787353A/zh active Pending
- 2020-09-23 WO PCT/US2020/052101 patent/WO2021061698A1/en not_active Ceased
- 2020-09-23 JP JP2022543368A patent/JP2022548800A/ja active Pending
- 2020-09-23 EP EP20870270.4A patent/EP4034660A4/en active Pending
- 2020-09-23 AU AU2020352984A patent/AU2020352984A1/en active Pending
-
2025
- 2025-08-18 JP JP2025135712A patent/JP2025169372A/ja active Pending
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