JPWO2022119974A5 - - Google Patents
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- Publication number
- JPWO2022119974A5 JPWO2022119974A5 JP2023533276A JP2023533276A JPWO2022119974A5 JP WO2022119974 A5 JPWO2022119974 A5 JP WO2022119974A5 JP 2023533276 A JP2023533276 A JP 2023533276A JP 2023533276 A JP2023533276 A JP 2023533276A JP WO2022119974 A5 JPWO2022119974 A5 JP WO2022119974A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- aav
- composition
- sequence
- 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
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063119977P | 2020-12-01 | 2020-12-01 | |
| US63/119,977 | 2020-12-01 | ||
| US202063130060P | 2020-12-23 | 2020-12-23 | |
| US63/130,060 | 2020-12-23 | ||
| PCT/US2021/061482 WO2022119974A1 (en) | 2020-12-01 | 2021-12-01 | Rna-targeting compositions and methods for treating cag repeat diseases |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023551873A JP2023551873A (ja) | 2023-12-13 |
| JPWO2022119974A5 true JPWO2022119974A5 (https=) | 2024-10-29 |
| JP2023551873A5 JP2023551873A5 (https=) | 2024-10-29 |
Family
ID=79731103
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023533276A Pending JP2023551873A (ja) | 2020-12-01 | 2021-12-01 | Ｃａｇリピート病を処置するためのｒｎａ標的化組成物および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240000972A1 (https=) |
| EP (1) | EP4255470A1 (https=) |
| JP (1) | JP2023551873A (https=) |
| KR (1) | KR20230127221A9 (https=) |
| AU (1) | AU2021391643A1 (https=) |
| CA (1) | CA3200453A1 (https=) |
| WO (1) | WO2022119974A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN114450411A (zh) | 2019-04-01 | 2022-05-06 | 特纳亚治疗股份有限公司 | 具有工程化衣壳的腺相关病毒 |
| IL301675A (en) | 2020-10-09 | 2023-05-01 | Tenaya Therapeutics Inc | Methods and preparations for placophilin-2 gene therapy |
| US20240108751A1 (en) * | 2020-12-01 | 2024-04-04 | Locanabio, Inc. | Rna-targeting compositions and methods for treating myotonic dystrophy type 1 |
| WO2022221278A1 (en) * | 2021-04-12 | 2022-10-20 | Locanabio, Inc. | Compositions and methods comprising hybrid promoters |
| JP2025525378A (ja) * | 2022-06-21 | 2025-08-05 | リーゲル セラピューティクス，インコーポレイティッド | 遺伝子調節エレメント及びその使用 |
| CN119320798A (zh) * | 2023-07-17 | 2025-01-17 | 中国科学院上海营养与健康研究所 | 一种可用于筛选针对rna重复序列药物的报告系统 |
| WO2025155722A1 (en) * | 2024-01-16 | 2025-07-24 | Astellas Gene Therapies, Inc. | Muscle selective hybrid regulatory combinations and methods of use thereof for the treatment of myotonic dystrophy type 1 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| DK2191001T3 (en) | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| CN101895633A (zh) | 2010-07-14 | 2010-11-24 | 中兴通讯股份有限公司 | 一种移动终端及其解锁方法 |
| WO2012068627A1 (en) | 2010-11-24 | 2012-05-31 | The University Of Western Australia | Peptides for the specific binding of rna targets |
| WO2013058404A1 (ja) | 2011-10-21 | 2013-04-25 | 国立大学法人九州大学 | Pprモチーフを利用したrna結合性蛋白質の設計方法及びその利用 |
| US10330674B2 (en) | 2015-01-13 | 2019-06-25 | Massachusetts Institute Of Technology | Pumilio domain-based modular protein architecture for RNA binding |
| US10876101B2 (en) | 2017-03-28 | 2020-12-29 | Locanabio, Inc. | CRISPR-associated (Cas) protein |
| US11168322B2 (en) | 2017-06-30 | 2021-11-09 | Arbor Biotechnologies, Inc. | CRISPR RNA targeting enzymes and systems and uses thereof |
| US10476825B2 (en) | 2017-08-22 | 2019-11-12 | Salk Institue for Biological Studies | RNA targeting methods and compositions |
| JP2021526858A (ja) * | 2018-06-08 | 2021-10-11 | ロックアネイビオ， インコーポレイテッド | Ｒｎａ標的化融合タンパク質組成物および使用方法 |
| US20240108751A1 (en) * | 2020-12-01 | 2024-04-04 | Locanabio, Inc. | Rna-targeting compositions and methods for treating myotonic dystrophy type 1 |
-
2021
- 2021-12-01 KR KR1020237021515A patent/KR20230127221A9/ko active Pending
- 2021-12-01 CA CA3200453A patent/CA3200453A1/en active Pending
- 2021-12-01 EP EP21845128.4A patent/EP4255470A1/en active Pending
- 2021-12-01 AU AU2021391643A patent/AU2021391643A1/en active Pending
- 2021-12-01 WO PCT/US2021/061482 patent/WO2022119974A1/en not_active Ceased
- 2021-12-01 JP JP2023533276A patent/JP2023551873A/ja active Pending
- 2021-12-01 US US18/039,813 patent/US20240000972A1/en active Pending
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