JP2026027271A5 - - Google Patents
Info
- Publication number
- JP2026027271A5 JP2026027271A5 JP2025175606A JP2025175606A JP2026027271A5 JP 2026027271 A5 JP2026027271 A5 JP 2026027271A5 JP 2025175606 A JP2025175606 A JP 2025175606A JP 2025175606 A JP2025175606 A JP 2025175606A JP 2026027271 A5 JP2026027271 A5 JP 2026027271A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- itr
- nucleotide sequence
- aav
- seq
- 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 (6)
| 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 | ||
| JP2022531343A 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 |
| PCT/US2020/062484 WO2021108755A2 (en) | 2019-11-28 | 2020-11-27 | Microdystrophin gene therapy constructs and uses thereof |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022531343A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2026027271A JP2026027271A (ja) | 2026-02-18 |
| JP2026027271A5 true JP2026027271A5 (enExample) | 2026-05-15 |
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 Before (1)
| 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 |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US20230270886A1 (enExample) |
| EP (1) | EP4065596A2 (enExample) |
| JP (2) | JP7822933B2 (enExample) |
| KR (1) | KR20220107222A (enExample) |
| AU (1) | AU2020392243A1 (enExample) |
| BR (1) | BR112022009895A2 (enExample) |
| CA (1) | CA3159516A1 (enExample) |
| CL (1) | CL2022001372A1 (enExample) |
| MX (1) | MX2022006427A (enExample) |
| TW (1) | TWI908746B (enExample) |
| WO (1) | WO2021108755A2 (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW202206447A (zh) | 2020-04-29 | 2022-02-16 | 美商必治妥美雅史谷比公司 | 具血影蛋白(spectrin)融合結構域之微型化肌肉萎縮蛋白及其用途 |
| WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
| US20240216543A1 (en) * | 2021-04-26 | 2024-07-04 | Regenxbio Inc. | Microdystrophin gene therapy administration for treatment of dystrophinopathies |
| 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 |
| US20240350674A1 (en) * | 2021-08-11 | 2024-10-24 | Solid Biosciences Inc. | Treatment of muscular dystrophy |
| JP2024531138A (ja) * | 2021-08-11 | 2024-08-29 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 筋ジストロフィーを処置するための組成物および方法 |
| JP2024536223A (ja) * | 2021-09-30 | 2024-10-04 | ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム | Ｓｌｃ１３ａ５遺伝子療法ベクターおよびその使用 |
| EP4413018A1 (en) | 2021-10-07 | 2024-08-14 | RegenxBio Inc. | Recombinant adeno-associated viruses for targeted delivery |
| WO2023064947A1 (en) | 2021-10-15 | 2023-04-20 | 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 |
| EP4499154A1 (en) | 2022-03-25 | 2025-02-05 | 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 |
| JP2025534666A (ja) | 2022-10-11 | 2025-10-17 | リジェネックスバイオ インコーポレイテッド | 操作された核酸調節エレメントならびにその使用方法 |
| 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 |
| WO2026075999A2 (en) * | 2024-10-01 | 2026-04-09 | Sarepta Therapeutics, Inc. | Dystrophin and micro-dystrophin antibodies and fragments thereof |
| 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 |
|---|---|---|---|---|
| EP2369002A1 (en) | 1999-08-09 | 2011-09-28 | 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 intrastrand base pairs |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| HUE054805T2 (hu) * | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| US7183969B2 (en) | 2004-12-22 | 2007-02-27 | Raytheon Company | System and technique for calibrating radar arrays |
| EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| WO2006132118A1 (ja) | 2005-06-09 | 2006-12-14 | Matsushita Electric Industrial Co., Ltd. | 振幅誤差補償装置及び直交度誤差補償装置 |
| US7892824B2 (en) * | 2007-01-18 | 2011-02-22 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma |
| ES2644880T3 (es) | 2008-02-19 | 2017-11-30 | Uniqure Ip B.V. | Optimización de expresión de proteínas Rep y Cap parvovirales en las células de insecto |
| ES2724122T3 (es) | 2009-04-30 | 2019-09-06 | Univ Pennsylvania | Composiciones para dirigir células de las vías respiratorias de conducción que comprenden construcciones de virus adenoasociado |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel 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 |
| CN107828820B (zh) | 2010-10-27 | 2022-06-07 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
| US9409953B2 (en) | 2011-02-10 | 2016-08-09 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| CN107012171B (zh) | 2011-04-22 | 2021-07-13 | 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 |
| EP3147295B2 (en) | 2011-08-24 | 2023-11-22 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
| EP3470523A1 (en) | 2012-05-09 | 2019-04-17 | 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 |
| EA201990558A3 (ru) | 2013-04-20 | 2021-02-26 | Рисёрч Инститъют Эт Нэйшнвайд Чилдрен'С Хоспитал | Доставка нацеленных на экзон 2 полинуклеотидных конструкций u7snrna при помощи рекомбинантного аденоассоциированного вируса |
| PT3024498T (pt) | 2013-07-22 | 2020-03-06 | Childrens Hospital Philadelphia | Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos |
| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
| NZ718926A (en) | 2013-10-11 | 2021-12-24 | Massachusetts Eye & Ear Infirmary | Methods of predicting ancestral virus sequences and uses thereof |
| US10746742B2 (en) | 2014-04-25 | 2020-08-18 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | 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 |
| US10876134B2 (en) | 2014-12-16 | 2020-12-29 | 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 |
| WO2016127057A1 (en) | 2015-02-06 | 2016-08-11 | The University Of North Carolina At Chapel Hill | Optimized human clotting factor viii gene expression cassettes and their use |
| 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 |
| CA3008142A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| DK3596222T5 (da) * | 2017-03-17 | 2024-09-02 | Res Inst Nationwide Childrens Hospital | Adenoassocieret virusvektorfremføring af muskelspecifik mikro-dystrophin til behandling af muskeldystrofi |
| CN120665951A (zh) | 2018-02-07 | 2025-09-19 | 吉尼松公司 | 杂合调控元件 |
-
2020
- 2020-11-27 US US17/778,651 patent/US20230270886A1/en active Pending
- 2020-11-27 EP EP20845454.6A patent/EP4065596A2/en active Pending
- 2020-11-27 MX MX2022006427A patent/MX2022006427A/es unknown
- 2020-11-27 CA CA3159516A patent/CA3159516A1/en active Pending
- 2020-11-27 TW TW109141932A patent/TWI908746B/zh active
- 2020-11-27 AU AU2020392243A patent/AU2020392243A1/en active Pending
- 2020-11-27 BR BR112022009895A patent/BR112022009895A2/pt unknown
- 2020-11-27 WO PCT/US2020/062484 patent/WO2021108755A2/en not_active Ceased
- 2020-11-27 JP JP2022531343A patent/JP7822933B2/ja active Active
- 2020-11-27 KR KR1020227021099A patent/KR20220107222A/ko active Pending
-
2022
- 2022-05-25 CL CL2022001372A patent/CL2022001372A1/es unknown
-
2025
- 2025-06-10 US US19/234,245 patent/US20250367327A1/en active Pending
- 2025-06-10 US US19/234,261 patent/US20250367328A1/en active Pending
- 2025-06-10 US US19/234,243 patent/US20250367326A1/en active Pending
- 2025-10-17 JP JP2025175606A patent/JP2026027271A/ja active Pending