JP2021520196A5 - - Google Patents
Info
- Publication number
- JP2021520196A5 JP2021520196A5 JP2020554102A JP2020554102A JP2021520196A5 JP 2021520196 A5 JP2021520196 A5 JP 2021520196A5 JP 2020554102 A JP2020554102 A JP 2020554102A JP 2020554102 A JP2020554102 A JP 2020554102A JP 2021520196 A5 JP2021520196 A5 JP 2021520196A5
- Authority
- JP
- Japan
- Prior art keywords
- adeno
- associated virus
- avv
- muscular dystrophy
- vector
- 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 |
|---|---|---|---|
| US201862651772P | 2018-04-03 | 2018-04-03 | |
| US62/651,772 | 2018-04-03 | ||
| PCT/US2019/025496 WO2019195362A1 (en) | 2018-04-03 | 2019-04-03 | Hinges 1 and/or 4 modified dystrophins for dystrophinopathy therapy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021520196A JP2021520196A (ja) | 2021-08-19 |
| JP2021520196A5 true JP2021520196A5 (enExample) | 2022-04-05 |
Family
ID=68101401
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020554102A Pending JP2021520196A (ja) | 2018-04-03 | 2019-04-03 | ジストロフィン異常症（ｄｙｓｔｒｏｐｈｉｎｏｐａｔｈｙ）治療のためのヒンジ１および／または４の改変ジストロフィン |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20210139550A1 (enExample) |
| EP (1) | EP3773605A4 (enExample) |
| JP (1) | JP2021520196A (enExample) |
| CN (1) | CN111971050A (enExample) |
| WO (1) | WO2019195362A1 (enExample) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN108463211B (zh) | 2015-11-16 | 2022-09-27 | 全国儿童医院研究所 | 用于治疗肌联蛋白类肌病和其它肌联蛋白病变的材料和方法 |
| US11338045B2 (en) | 2017-03-17 | 2022-05-24 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| AU2019216257B2 (en) | 2018-01-31 | 2025-11-20 | Research Institute At Nationwide Children's Hospital | Gene therapy for limb-girdle muscular dystrophy type 2C |
| IL324506A (en) | 2018-06-18 | 2026-01-01 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector for delivery of muscle-specific microdystrophin for the treatment of muscular dystrophy |
| IL279789B2 (en) | 2018-06-29 | 2026-04-01 | Res Inst Nationwide Childrens Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A |
| CN113613668A (zh) | 2018-10-24 | 2021-11-05 | 百时美施贵宝公司 | 小型化肌营养不良蛋白及其用途 |
| KR20210133242A (ko) | 2019-02-26 | 2021-11-05 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료 |
| CA3150964A1 (en) | 2019-08-21 | 2021-02-25 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| AU2020392243A1 (en) | 2019-11-28 | 2022-06-16 | Regenxbio Inc. | Microdystrophin gene therapy constructs and uses thereof |
| US11535868B2 (en) * | 2020-04-29 | 2022-12-27 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
| EP4164668A1 (en) | 2020-06-15 | 2023-04-19 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery for muscular dystrophies |
| WO2022029543A1 (en) * | 2020-08-06 | 2022-02-10 | Intas Pharmaceuticals Ltd. | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
| EP4108263A3 (en) | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| US12285497B2 (en) | 2021-10-15 | 2025-04-29 | Research Institute At Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7771993B2 (en) * | 2004-01-23 | 2010-08-10 | The Trustees Of The University Of Pennsylvania | Microutrophin and uses thereof |
| US9624282B2 (en) * | 2012-11-26 | 2017-04-18 | The Curators Of The University Of Missouri | Microdystrophin peptides and methods for treating muscular dystrophy using the same |
| WO2017223128A1 (en) * | 2016-06-21 | 2017-12-28 | The Curators Of The University Of Missouri | Modified dystrophin proteins |
-
2019
- 2019-04-03 WO PCT/US2019/025496 patent/WO2019195362A1/en not_active Ceased
- 2019-04-03 CN CN201980022798.0A patent/CN111971050A/zh active Pending
- 2019-04-03 EP EP19782410.5A patent/EP3773605A4/en not_active Withdrawn
- 2019-04-03 JP JP2020554102A patent/JP2021520196A/ja active Pending
- 2019-04-03 US US17/045,266 patent/US20210139550A1/en not_active Abandoned