JP2024517143A5 - - Google Patents
Info
- Publication number
- JP2024517143A5 JP2024517143A5 JP2023565530A JP2023565530A JP2024517143A5 JP 2024517143 A5 JP2024517143 A5 JP 2024517143A5 JP 2023565530 A JP2023565530 A JP 2023565530A JP 2023565530 A JP2023565530 A JP 2023565530A JP 2024517143 A5 JP2024517143 A5 JP 2024517143A5
- Authority
- JP
- Japan
- Prior art keywords
- dystrophin
- region
- pharmaceutical composition
- raav
- composition according
- 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 (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163180064P | 2021-04-26 | 2021-04-26 | |
| US63/180,064 | 2021-04-26 | ||
| US202163245909P | 2021-09-19 | 2021-09-19 | |
| US63/245,909 | 2021-09-19 | ||
| US202263314436P | 2022-02-27 | 2022-02-27 | |
| US63/314,436 | 2022-02-27 | ||
| US202263319363P | 2022-03-13 | 2022-03-13 | |
| US63/319,363 | 2022-03-13 | ||
| PCT/US2022/026341 WO2022232141A1 (en) | 2021-04-26 | 2022-04-26 | Microdystrophin gene therapy administration for treatment of dystrophinopathies |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024517143A JP2024517143A (ja) | 2024-04-19 |
| JP2024517143A5 true JP2024517143A5 (https=) | 2025-05-12 |
Family
ID=81654620
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023565530A Pending JP2024517143A (ja) | 2021-04-26 | 2022-04-26 | ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US20240216543A1 (https=) |
| EP (1) | EP4329821A1 (https=) |
| JP (1) | JP2024517143A (https=) |
| KR (1) | KR20240004564A (https=) |
| BR (1) | BR112023021999A2 (https=) |
| CA (1) | CA3216744A1 (https=) |
| IL (1) | IL307902A (https=) |
| TW (1) | TW202309293A (https=) |
| WO (1) | WO2022232141A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023178053A1 (en) * | 2022-03-13 | 2023-09-21 | Regenxbio Inc. | Modified muscle-specific promoters |
| WO2024138158A2 (en) | 2022-12-23 | 2024-06-27 | Ultragenyx Pharmaceutical Inc. | Method of producing soluble microdystrophin protein and uses of the same |
| WO2025225719A1 (en) * | 2024-04-26 | 2025-10-30 | Chugai Seiyaku Kabushiki Kaisha | Treatment and prevention of muscular dystrophy and its related diseases or disorders with satralizumab |
| CN118490245B (zh) * | 2024-05-11 | 2025-12-12 | 北京邮电大学 | 一种基于心电波形监督的压力识别方法 |
| WO2026006341A1 (en) | 2024-06-24 | 2026-01-02 | Regenxbio Inc. | Microdystrophin gene therapy administration for treatment of dystrophinopathies |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| US7183969B2 (en) | 2004-12-22 | 2007-02-27 | Raytheon Company | System and technique for calibrating radar arrays |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| CN101711164B (zh) * | 2007-01-18 | 2014-06-04 | 密苏里-哥伦比亚大学 | 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 |
| JP2011512156A (ja) | 2008-02-19 | 2011-04-21 | アムステルダム モレキュラー セラピューティクス ビー．ブイ． | 昆虫細胞におけるパルボウイルスｒｅｐ及びｃａｐタンパク質の発現の最適化 |
| EP2425000B1 (en) | 2009-04-30 | 2019-02-20 | The Trustees Of The University Of Pennsylvania | Compositions for targeting conducting airway cells comprising adeno-associated virus constructs |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| WO2012057363A1 (ja) | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| CN105755044A (zh) | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| WO2013170078A1 (en) | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| CA2905952A1 (en) | 2013-03-13 | 2014-10-02 | The Children's Hospital Of Philadelphia | Adeno-associated virus vectors and methods of use thereof |
| MX388221B (es) | 2013-04-20 | 2025-03-19 | Res Institute At Nationwide Children´S Hospital | ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2. |
| IL297919A (en) | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| CN106232618A (zh) | 2013-10-11 | 2016-12-14 | 马萨诸塞眼科耳科诊所 | 预测祖先病毒序列的方法及其用途 |
| WO2015164757A1 (en) | 2014-04-25 | 2015-10-29 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| JP6832280B2 (ja) * | 2015-01-16 | 2021-02-24 | ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| GB201507842D0 (en) * | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| JP6665466B2 (ja) | 2015-09-26 | 2020-03-13 | 日亜化学工業株式会社 | 半導体発光素子及びその製造方法 |
| WO2017070491A1 (en) | 2015-10-23 | 2017-04-27 | Applied Genetic Technologies Corporation | Ophthalmic formulations |
| CN111902539B (zh) | 2018-02-07 | 2025-05-16 | 吉尼松公司 | 杂合调控元件 |
| TWI908746B (zh) * | 2019-11-28 | 2025-12-21 | 美商銳進科斯生物股份有限公司 | 微小肌縮蛋白基因療法之構築體及其用途 |
-
2022
- 2022-04-26 JP JP2023565530A patent/JP2024517143A/ja active Pending
- 2022-04-26 EP EP22723884.7A patent/EP4329821A1/en active Pending
- 2022-04-26 US US18/557,155 patent/US20240216543A1/en active Pending
- 2022-04-26 TW TW111115911A patent/TW202309293A/zh unknown
- 2022-04-26 CA CA3216744A patent/CA3216744A1/en active Pending
- 2022-04-26 IL IL307902A patent/IL307902A/en unknown
- 2022-04-26 BR BR112023021999A patent/BR112023021999A2/pt unknown
- 2022-04-26 WO PCT/US2022/026341 patent/WO2022232141A1/en not_active Ceased
- 2022-04-26 KR KR1020237039920A patent/KR20240004564A/ko active Pending
-
2025
- 2025-06-20 US US19/245,006 patent/US20250312492A1/en active Pending
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