JP2023503637A5 - - Google Patents
Info
- Publication number
- JP2023503637A5 JP2023503637A5 JP2022531343A JP2022531343A JP2023503637A5 JP 2023503637 A5 JP2023503637 A5 JP 2023503637A5 JP 2022531343 A JP2022531343 A JP 2022531343A JP 2022531343 A JP2022531343 A JP 2022531343A JP 2023503637 A5 JP2023503637 A5 JP 2023503637A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid sequence
- sequence encoding
- seq
- 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.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025175606A JP2026027271A (ja) | 2019-11-28 | 2025-10-17 | マイクロジストロフィン遺伝子治療コンストラクト及びその使用 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962941719P | 2019-11-28 | 2019-11-28 | |
| US62/941,719 | 2019-11-28 | ||
| US202063024933P | 2020-05-14 | 2020-05-14 | |
| US63/024,933 | 2020-05-14 | ||
| PCT/US2020/062484 WO2021108755A2 (en) | 2019-11-28 | 2020-11-27 | Microdystrophin gene therapy constructs and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025175606A Division JP2026027271A (ja) | 2019-11-28 | 2025-10-17 | マイクロジストロフィン遺伝子治療コンストラクト及びその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023503637A JP2023503637A (ja) | 2023-01-31 |
| JP2023503637A5 true JP2023503637A5 (https=) | 2024-01-09 |
| JP7822933B2 JP7822933B2 (ja) | 2026-03-03 |
Family
ID=74206146
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022531343A Active JP7822933B2 (ja) | 2019-11-28 | 2020-11-27 | マイクロジストロフィン遺伝子治療コンストラクト及びその使用 |
| JP2025175606A Pending JP2026027271A (ja) | 2019-11-28 | 2025-10-17 | マイクロジストロフィン遺伝子治療コンストラクト及びその使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025175606A Pending JP2026027271A (ja) | 2019-11-28 | 2025-10-17 | マイクロジストロフィン遺伝子治療コンストラクト及びその使用 |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US20230270886A1 (https=) |
| EP (1) | EP4065596A2 (https=) |
| JP (2) | JP7822933B2 (https=) |
| KR (1) | KR20220107222A (https=) |
| AU (1) | AU2020392243A1 (https=) |
| BR (1) | BR112022009895A2 (https=) |
| CA (1) | CA3159516A1 (https=) |
| CL (1) | CL2022001372A1 (https=) |
| MX (1) | MX2022006427A (https=) |
| TW (1) | TWI908746B (https=) |
| WO (1) | WO2021108755A2 (https=) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL297753A (en) | 2020-04-29 | 2022-12-01 | Bristol Myers Squibb Co | Minimized dystrophins with spectrin fusion domains and uses thereof |
| WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
| JP2024517143A (ja) * | 2021-04-26 | 2024-04-19 | リジェネックスバイオ インコーポレイテッド | 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 |
| US20240269329A1 (en) * | 2021-06-07 | 2024-08-15 | University Of Florida Research Foundation, Incorporated | Gene therapy for duchenne muscular dystrophy |
| AU2022297795A1 (en) * | 2021-06-23 | 2024-01-04 | AskBio Inc. | Regulatory nucleic acid sequences |
| US20240409955A1 (en) * | 2021-07-19 | 2024-12-12 | New York University | Auf1 combination therapies for treatment of muscle degenerative disease |
| WO2023018854A2 (en) * | 2021-08-11 | 2023-02-16 | Solid Biosciences Inc. | Treatment of muscular dystrophy |
| US20250019721A1 (en) * | 2021-08-11 | 2025-01-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
| WO2023056367A1 (en) * | 2021-09-30 | 2023-04-06 | The Board Of Regents Of The Universityof Texas System | Slc13a5 gene therapy vectors and uses thereof |
| WO2023060269A1 (en) | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for targeted delivery |
| US20240425572A1 (en) | 2021-10-15 | 2024-12-26 | Regenxbio Inc. | Antibodies and methods of using thereof |
| CN119233831A (zh) * | 2022-01-17 | 2024-12-31 | 迪纳柯公司 | 用于与肌营养不良蛋白有关的心肌病的基因疗法组合物和治疗 |
| WO2023141582A1 (en) | 2022-01-21 | 2023-07-27 | Regenxbio Inc. | Engineered promoters |
| EP4230196A1 (en) | 2022-02-21 | 2023-08-23 | Som Innovation Biotech, S.A. | Compounds for use in the treatment of dystrophinopathies |
| US20250213727A1 (en) | 2022-03-25 | 2025-07-03 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
| WO2023248251A1 (en) * | 2022-06-24 | 2023-12-28 | Indian Institute Of Technology Kanpur | An optimized aav vector for gene therapy of muscular dystrophy |
| US20260055144A1 (en) | 2022-08-24 | 2026-02-26 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
| EP4601710A2 (en) | 2022-10-11 | 2025-08-20 | RegenxBio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
| WO2024091495A1 (en) * | 2022-10-24 | 2024-05-02 | Biogen Ma Inc. | Compositions and methods for treating retinitis pigmentosa |
| WO2024138158A2 (en) | 2022-12-23 | 2024-06-27 | Ultragenyx Pharmaceutical Inc. | Method of producing soluble microdystrophin protein and uses of the same |
| CN115838725B (zh) * | 2022-12-30 | 2023-09-08 | 广州派真生物技术有限公司 | 在哺乳动物心脏中特异性启动基因的启动子序列及其应用 |
| WO2024216244A2 (en) | 2023-04-13 | 2024-10-17 | Regenxbio Inc. | Targeting aav capsids, methods of manufacturing and using same |
| AR133921A1 (es) | 2023-09-25 | 2025-11-12 | Sarepta Therapeutics Inc | Distrofinas humanas adaptadas con mayor afinidad de unión a la actina para el tratamiento de las distrofias musculares |
| WO2025250909A1 (en) | 2024-05-31 | 2025-12-04 | Sarepta Therapeutics, Inc. | Muscle-tropic recombinant aav |
| WO2026080823A1 (en) | 2024-10-10 | 2026-04-16 | Research Institute At Nationwide Children's Hospital | Methods for diagnosing and treating muscular dystrophy diseases |
Family Cites Families (35)
| 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 |
| WO2016100575A1 (en) | 2014-12-16 | 2016-06-23 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
| 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 |
| MX388739B (es) | 2015-02-06 | 2025-03-20 | Univ North Carolina Chapel Hill | Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso. |
| 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 |
| AU2016366560A1 (en) | 2015-12-11 | 2018-06-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP7676111B2 (ja) | 2017-03-17 | 2025-05-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| CN111902539B (zh) | 2018-02-07 | 2025-05-16 | 吉尼松公司 | 杂合调控元件 |
-
2020
- 2020-11-27 TW TW109141932A patent/TWI908746B/zh active
- 2020-11-27 JP JP2022531343A patent/JP7822933B2/ja active Active
- 2020-11-27 US US17/778,651 patent/US20230270886A1/en active Pending
- 2020-11-27 CA CA3159516A patent/CA3159516A1/en active Pending
- 2020-11-27 WO PCT/US2020/062484 patent/WO2021108755A2/en not_active Ceased
- 2020-11-27 KR KR1020227021099A patent/KR20220107222A/ko active Pending
- 2020-11-27 AU AU2020392243A patent/AU2020392243A1/en active Pending
- 2020-11-27 EP EP20845454.6A patent/EP4065596A2/en active Pending
- 2020-11-27 BR BR112022009895A patent/BR112022009895A2/pt unknown
- 2020-11-27 MX MX2022006427A patent/MX2022006427A/es unknown
-
2022
- 2022-05-25 CL CL2022001372A patent/CL2022001372A1/es unknown
-
2025
- 2025-06-10 US US19/234,243 patent/US20250367326A1/en active Pending
- 2025-06-10 US US19/234,245 patent/US20250367327A1/en active Pending
- 2025-06-10 US US19/234,261 patent/US20250367328A1/en active Pending
- 2025-10-17 JP JP2025175606A patent/JP2026027271A/ja active Pending
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