JP2023501897A5 - - Google Patents
Info
- Publication number
- JP2023501897A5 JP2023501897A5 JP2022523436A JP2022523436A JP2023501897A5 JP 2023501897 A5 JP2023501897 A5 JP 2023501897A5 JP 2022523436 A JP2022523436 A JP 2022523436A JP 2022523436 A JP2022523436 A JP 2022523436A JP 2023501897 A5 JP2023501897 A5 JP 2023501897A5
- Authority
- JP
- Japan
- Prior art keywords
- c9orf72
- nucleic acid
- vector
- pharmaceutical composition
- expression cassette
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962924351P | 2019-10-22 | 2019-10-22 | |
| US62/924,351 | 2019-10-22 | ||
| PCT/US2020/056905 WO2021081236A1 (en) | 2019-10-22 | 2020-10-22 | Triple function adeno-associated virus (aav) vectors for the treatment of c9orf72 associated diseases |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023501897A JP2023501897A (ja) | 2023-01-20 |
| JP2023501897A5 true JP2023501897A5 (enExample) | 2023-10-30 |
Family
ID=75620858
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022523436A Pending JP2023501897A (ja) | 2019-10-22 | 2020-10-22 | Ｃ９ｏｒｆ７２関連疾患の治療のための三重機能アデノ随伴ウイルス（ａａｖ）ベクター |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20210147873A1 (enExample) |
| EP (1) | EP4048794A4 (enExample) |
| JP (1) | JP2023501897A (enExample) |
| KR (1) | KR20230019063A (enExample) |
| CN (1) | CN116134134A (enExample) |
| AU (1) | AU2020370291A1 (enExample) |
| CA (1) | CA3158518A1 (enExample) |
| IL (1) | IL292384A (enExample) |
| MX (1) | MX2022004771A (enExample) |
| WO (1) | WO2021081236A1 (enExample) |
Families Citing this family (8)
| 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 |
| 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 |
| MX2023014168A (es) * | 2021-06-04 | 2024-02-27 | Alnylam Pharmaceuticals Inc | Marco de lectura abierto 72 del cromosoma humano 9 (c9orf72) composiciones de agente de arni y metodos de uso de este. |
| EP4427049A4 (en) * | 2021-11-01 | 2025-08-20 | Univ Florida | POLY-GA PROTEINS IN ALZHEIMER'S DISEASE |
| TW202340467A (zh) * | 2022-01-10 | 2023-10-16 | 賓州大學委員會 | 有用於治療c9orf72介導之病症之組成物及方法 |
| WO2024073592A2 (en) * | 2022-09-28 | 2024-04-04 | Atalanta Therapeutics, Inc. | Compositions and methods for treatment of neurological disorders |
| CN121057823A (zh) * | 2023-04-10 | 2025-12-02 | 耶鲁大学 | 体内修饰神经元以治疗和/或预防肌萎缩性侧索硬化(als)的方法 |
| CN122374456A (zh) * | 2023-10-20 | 2026-07-10 | 斯帕克医疗有限公司 | 靶向 α 突触核蛋白表达的抑制性核酸 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10738326B2 (en) * | 2010-10-27 | 2020-08-11 | Jichi Medical University | Adeno-associated virus vector for gene transfer to nervous system cells |
| US9096671B2 (en) * | 2011-06-29 | 2015-08-04 | Consejo Superior De Investigaciones Cientificas (Csic) | LRP1 as key receptor for the transfer of sterified cholesterol from very-low-density lipoproteins (VLDL) to ischaemic cardiac muscle |
| WO2013030588A1 (en) * | 2011-08-31 | 2013-03-07 | The University Of Manchester | Method for diagnosing a neurodegenerative disease |
| WO2016168592A2 (en) * | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| AU2017234929B2 (en) * | 2016-03-18 | 2024-05-02 | The Children's Hospital Of Philadelphia | Therapeutic for treatment of diseases including the central nervous system |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| AU2018354195A1 (en) * | 2017-10-23 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
-
2020
- 2020-10-22 EP EP20878214.4A patent/EP4048794A4/en not_active Withdrawn
- 2020-10-22 KR KR1020227017065A patent/KR20230019063A/ko not_active Ceased
- 2020-10-22 MX MX2022004771A patent/MX2022004771A/es unknown
- 2020-10-22 JP JP2022523436A patent/JP2023501897A/ja active Pending
- 2020-10-22 IL IL292384A patent/IL292384A/en unknown
- 2020-10-22 WO PCT/US2020/056905 patent/WO2021081236A1/en not_active Ceased
- 2020-10-22 US US17/077,682 patent/US20210147873A1/en not_active Abandoned
- 2020-10-22 AU AU2020370291A patent/AU2020370291A1/en not_active Abandoned
- 2020-10-22 CN CN202080089426.2A patent/CN116134134A/zh active Pending
- 2020-10-22 CA CA3158518A patent/CA3158518A1/en active Pending
-
2023
- 2023-04-24 US US18/138,361 patent/US20240067984A1/en not_active Abandoned
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