JP2024532758A5 - - Google Patents
Info
- Publication number
- JP2024532758A5 JP2024532758A5 JP2024508324A JP2024508324A JP2024532758A5 JP 2024532758 A5 JP2024532758 A5 JP 2024532758A5 JP 2024508324 A JP2024508324 A JP 2024508324A JP 2024508324 A JP2024508324 A JP 2024508324A JP 2024532758 A5 JP2024532758 A5 JP 2024532758A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- vector genome
- virus particle
- seq
- particle 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163231720P | 2021-08-11 | 2021-08-11 | |
| US63/231,720 | 2021-08-11 | ||
| PCT/US2022/040030 WO2023018854A2 (en) | 2021-08-11 | 2022-08-11 | Treatment of muscular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024532758A JP2024532758A (ja) | 2024-09-10 |
| JP2024532758A5 true JP2024532758A5 (https=) | 2025-08-20 |
Family
ID=85201128
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024508324A Pending JP2024532758A (ja) | 2021-08-11 | 2022-08-11 | 筋ジストロフィーの治療 |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240350674A1 (https=) |
| EP (1) | EP4384628A2 (https=) |
| JP (1) | JP2024532758A (https=) |
| KR (1) | KR20240095165A (https=) |
| CN (1) | CN118159656A (https=) |
| AU (1) | AU2022328215A1 (https=) |
| CA (1) | CA3228365A1 (https=) |
| IL (1) | IL310725A (https=) |
| TW (1) | TW202328446A (https=) |
| WO (1) | WO2023018854A2 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| WO2025221648A1 (en) * | 2024-04-18 | 2025-10-23 | Solid Biosciences Inc. | Low dose aav administration |
| WO2025250909A1 (en) | 2024-05-31 | 2025-12-04 | Sarepta Therapeutics, Inc. | Muscle-tropic recombinant aav |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| JP2001500497A (ja) | 1996-09-06 | 2001-01-16 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| 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 |
| US20070202587A1 (en) | 2002-09-23 | 2007-08-30 | Applied Genetic Technologies Corporation | Recombinant AAV production in mammalian cells |
| ATE453716T1 (de) * | 2004-08-03 | 2010-01-15 | Geneart Ag | Verfahren zur modulation der genexpression durch änderung des cpg gehalts |
| ES2751999T3 (es) | 2008-01-29 | 2020-04-02 | Applied Genetic Tech Corporation | Producción recombinante de virus adeno-asociados usando células BHK en suspensión |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| 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 |
| ES2977730T3 (es) | 2016-04-15 | 2024-08-29 | Res Inst Nationwide Childrens Hospital | Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular |
| 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 |
| US20250019721A1 (en) * | 2021-08-11 | 2025-01-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
-
2022
- 2022-08-11 WO PCT/US2022/040030 patent/WO2023018854A2/en not_active Ceased
- 2022-08-11 JP JP2024508324A patent/JP2024532758A/ja active Pending
- 2022-08-11 EP EP22856596.6A patent/EP4384628A2/en active Pending
- 2022-08-11 KR KR1020247008037A patent/KR20240095165A/ko active Pending
- 2022-08-11 US US18/682,359 patent/US20240350674A1/en active Pending
- 2022-08-11 CA CA3228365A patent/CA3228365A1/en active Pending
- 2022-08-11 AU AU2022328215A patent/AU2022328215A1/en active Pending
- 2022-08-11 TW TW111130260A patent/TW202328446A/zh unknown
- 2022-08-11 CN CN202280068850.8A patent/CN118159656A/zh active Pending
- 2022-08-11 IL IL310725A patent/IL310725A/en unknown
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