JP2023506030A5 - - Google Patents
Info
- Publication number
- JP2023506030A5 JP2023506030A5 JP2022535865A JP2022535865A JP2023506030A5 JP 2023506030 A5 JP2023506030 A5 JP 2023506030A5 JP 2022535865 A JP2022535865 A JP 2022535865A JP 2022535865 A JP2022535865 A JP 2022535865A JP 2023506030 A5 JP2023506030 A5 JP 2023506030A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- item
- sarcoglycan
- nos
- 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 |
|---|---|---|---|
| JP2025087309A JP2025139591A (ja) | 2019-12-16 | 2025-05-26 | ジストロフィン関連タンパク質複合体（ｄａｐｃ）を回復および維持するための組成物および方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962948586P | 2019-12-16 | 2019-12-16 | |
| US62/948,586 | 2019-12-16 | ||
| US202063023144P | 2020-05-11 | 2020-05-11 | |
| US63/023,144 | 2020-05-11 | ||
| PCT/US2020/065157 WO2021126880A1 (en) | 2019-12-16 | 2020-12-15 | Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc) |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025087309A Division JP2025139591A (ja) | 2019-12-16 | 2025-05-26 | ジストロフィン関連タンパク質複合体（ｄａｐｃ）を回復および維持するための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023506030A JP2023506030A (ja) | 2023-02-14 |
| JP2023506030A5 true JP2023506030A5 (https=) | 2023-12-22 |
| JP7847084B2 JP7847084B2 (ja) | 2026-04-16 |
Family
ID=76478281
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022535865A Active JP7847084B2 (ja) | 2019-12-16 | 2020-12-15 | ジストロフィン関連タンパク質複合体（ｄａｐｃ）を回復および維持するための組成物および方法 |
| JP2025087309A Pending JP2025139591A (ja) | 2019-12-16 | 2025-05-26 | ジストロフィン関連タンパク質複合体（ｄａｐｃ）を回復および維持するための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025087309A Pending JP2025139591A (ja) | 2019-12-16 | 2025-05-26 | ジストロフィン関連タンパク質複合体（ｄａｐｃ）を回復および維持するための組成物および方法 |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20230040544A1 (https=) |
| EP (1) | EP4117783A4 (https=) |
| JP (2) | JP7847084B2 (https=) |
| WO (1) | WO2021126880A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102877920B1 (ko) | 2015-11-16 | 2025-10-30 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법 |
| TWI887479B (zh) * | 2020-09-08 | 2025-06-21 | 美商薩羅塔治療公司 | 表現γ-肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療 |
| EP4198047A1 (en) | 2021-12-16 | 2023-06-21 | Genethon | Fukutin related protein gene transfer increase using modified itr sequences |
| EP4198046A1 (en) | 2021-12-16 | 2023-06-21 | Genethon | Alpha-sarcoglycan gene transfer increase using modified itr sequences |
| EP4198048A1 (en) | 2021-12-16 | 2023-06-21 | Genethon | Calpain-3 gene transfer increase using modified itr sequences |
| CA3259572A1 (en) * | 2022-06-30 | 2024-01-04 | Sarepta Therapeutics, Inc. | TREATMENT METHODS FOR MUSCULAR DYSTROPHY |
| CA3257440A1 (en) * | 2022-07-06 | 2024-01-11 | Adverum Biotechnologies, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF ACHROMOTOPSY |
| 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 |
| WO2025226842A1 (en) | 2024-04-24 | 2025-10-30 | Kate Therapeutics, Inc. | Expression control by drg-expressed mirnas |
| WO2026011009A1 (en) | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1999043360A1 (en) * | 1998-02-26 | 1999-09-02 | The Trustees Of The University Of Pennsylvania | Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex |
| US6262035B1 (en) * | 1998-10-01 | 2001-07-17 | University Of Iowa Research Foundation | Gene replacement therapy for muscular dystrophy |
| US9434928B2 (en) * | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| WO2017165859A1 (en) * | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Modified viral capsid proteins |
| LT3442600T (lt) * | 2016-04-15 | 2024-05-27 | Research Institute At Nationwide Children's Hospital | Su adeno virusu susijusio viruso vektoriaus iš β-sarkoglikano ir mikro- rnr-29 pristatymas ir raumenų distrofijos gydymas |
| 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 |
| AR114350A1 (es) * | 2018-01-31 | 2020-08-26 | Res Institute At Nationwide ChildrenS Hospital | Terapia genética para la distrofia muscular de cinturas del tipo 2c |
-
2020
- 2020-12-15 WO PCT/US2020/065157 patent/WO2021126880A1/en not_active Ceased
- 2020-12-15 JP JP2022535865A patent/JP7847084B2/ja active Active
- 2020-12-15 EP EP20902648.3A patent/EP4117783A4/en active Pending
- 2020-12-15 US US17/785,879 patent/US20230040544A1/en active Pending
-
2025
- 2025-05-26 JP JP2025087309A patent/JP2025139591A/ja active Pending
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