JP2023551874A5 - - Google Patents
Info
- Publication number
- JP2023551874A5 JP2023551874A5 JP2023533279A JP2023533279A JP2023551874A5 JP 2023551874 A5 JP2023551874 A5 JP 2023551874A5 JP 2023533279 A JP2023533279 A JP 2023533279A JP 2023533279 A JP2023533279 A JP 2023533279A JP 2023551874 A5 JP2023551874 A5 JP 2023551874A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- capsid protein
- sequence
- vector according
- protein
- 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 (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063119977P | 2020-12-01 | 2020-12-01 | |
| US63/119,977 | 2020-12-01 | ||
| US202063130092P | 2020-12-23 | 2020-12-23 | |
| US63/130,092 | 2020-12-23 | ||
| US202163278746P | 2021-11-12 | 2021-11-12 | |
| US63/278,746 | 2021-11-12 | ||
| PCT/US2021/061490 WO2022119979A1 (en) | 2020-12-01 | 2021-12-01 | Rna-targeting compositions and methods for treating myotonic dystrophy type 1 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023551874A JP2023551874A (ja) | 2023-12-13 |
| JP2023551874A5 true JP2023551874A5 (enExample) | 2024-12-09 |
Family
ID=79731176
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023533279A Pending JP2023551874A (ja) | 2020-12-01 | 2021-12-01 | 筋強直性ジストロフィー１型を処置するためのｒｎａ標的化組成物および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240108751A1 (enExample) |
| EP (1) | EP4255471A1 (enExample) |
| JP (1) | JP2023551874A (enExample) |
| KR (1) | KR20230129162A9 (enExample) |
| AU (1) | AU2021391645A1 (enExample) |
| CA (1) | CA3200588A1 (enExample) |
| WO (1) | WO2022119979A1 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020205889A1 (en) | 2019-04-01 | 2020-10-08 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| JP7415042B2 (ja) | 2020-10-09 | 2024-01-16 | テナヤ セラピューティクス， インコーポレイテッド | プラコフィリン２遺伝子治療の方法および組成物 |
| JP2023551873A (ja) * | 2020-12-01 | 2023-12-13 | ロックアネイビオ， インコーポレイテッド | 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 |
| WO2022221278A1 (en) * | 2021-04-12 | 2022-10-20 | Locanabio, Inc. | Compositions and methods comprising hybrid promoters |
| GB202207026D0 (en) * | 2022-05-13 | 2022-06-29 | Univ Oxford Innovation Ltd | Method |
| WO2025101968A1 (en) * | 2023-11-10 | 2025-05-15 | Repeat Therapeutics, Inc. | Treatment of neurodegenerative diseases caused by repeat expansion with nucleoside administration |
| TW202546236A (zh) | 2024-01-16 | 2025-12-01 | 美商安斯泰來基因治療股份有限公司 | 用於治療第1型肌強直性營養不良之肌肉選擇性混合調節組合及其使用方法 |
| WO2026015609A1 (en) * | 2024-07-10 | 2026-01-15 | The Johns Hopkins University | Vectors and methods for increased non-coding rna expression |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| HUE030719T2 (en) | 2007-04-09 | 2017-05-29 | Univ Florida | Raav vector compositions having tyrosine-modified capsid proteins and methods for use |
| 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 |
| US9580714B2 (en) | 2010-11-24 | 2017-02-28 | 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 |
| US10476825B2 (en) | 2017-08-22 | 2019-11-12 | Salk Institue for Biological Studies | RNA targeting methods and compositions |
| US20200071718A1 (en) * | 2018-06-08 | 2020-03-05 | Locana, Inc. | Rna-targeting fusion protein compositions and methods for use |
| JP2023551873A (ja) * | 2020-12-01 | 2023-12-13 | ロックアネイビオ， インコーポレイテッド | 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 |
-
2021
- 2021-12-01 EP EP21845129.2A patent/EP4255471A1/en active Pending
- 2021-12-01 JP JP2023533279A patent/JP2023551874A/ja active Pending
- 2021-12-01 WO PCT/US2021/061490 patent/WO2022119979A1/en not_active Ceased
- 2021-12-01 AU AU2021391645A patent/AU2021391645A1/en active Pending
- 2021-12-01 US US18/039,812 patent/US20240108751A1/en active Pending
- 2021-12-01 KR KR1020237021514A patent/KR20230129162A9/ko active Pending
- 2021-12-01 CA CA3200588A patent/CA3200588A1/en active Pending
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