JP2022513456A5 - - Google Patents
Info
- Publication number
- JP2022513456A5 JP2022513456A5 JP2021533367A JP2021533367A JP2022513456A5 JP 2022513456 A5 JP2022513456 A5 JP 2022513456A5 JP 2021533367 A JP2021533367 A JP 2021533367A JP 2021533367 A JP2021533367 A JP 2021533367A JP 2022513456 A5 JP2022513456 A5 JP 2022513456A5
- Authority
- JP
- Japan
- Prior art keywords
- viral vector
- recombinant viral
- vector according
- sequence
- gene
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862778646P | 2018-12-12 | 2018-12-12 | |
| US62/778,646 | 2018-12-12 | ||
| PCT/US2019/065718 WO2020123645A1 (en) | 2018-12-12 | 2019-12-11 | Combination therapy for treating muscular dystrophy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022513456A JP2022513456A (ja) | 2022-02-08 |
| JP2022513456A5 true JP2022513456A5 (https=) | 2022-12-20 |
| JP7788855B2 JP7788855B2 (ja) | 2025-12-19 |
Family
ID=71076668
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021533367A Active JP7788855B2 (ja) | 2018-12-12 | 2019-12-11 | 筋ジストロフィーの治療のための併用療法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220031865A1 (https=) |
| EP (1) | EP3893940A4 (https=) |
| JP (1) | JP7788855B2 (https=) |
| KR (1) | KR20210124969A (https=) |
| CN (1) | CN113646004A (https=) |
| AU (1) | AU2019395388A1 (https=) |
| CA (1) | CA3123003A1 (https=) |
| SG (1) | SG11202105873SA (https=) |
| WO (1) | WO2020123645A1 (https=) |
Families Citing this family (22)
| 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 |
| EP3596112A2 (en) | 2017-03-17 | 2020-01-22 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| 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 |
| MY208145A (en) | 2018-06-18 | 2025-04-18 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| KR20210028162A (ko) | 2018-06-29 | 2021-03-11 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 |
| AU2020229340A1 (en) | 2019-02-26 | 2021-09-16 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy |
| RS65421B1 (sr) | 2019-08-21 | 2024-05-31 | Res Inst Nationwide Childrens Hospital | Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije |
| CA3164335A1 (en) * | 2020-01-10 | 2021-07-15 | Fatih Ozsolak | Viral vector for combination therapy |
| IL299094A (en) | 2020-06-15 | 2023-02-01 | Res Inst Nationwide Childrens Hospital | Administration of an adeno-associated virus vector for muscular dystrophies |
| EP4086276A1 (en) | 2021-05-03 | 2022-11-09 | Université d'Aix-Marseille | Composition for treating dysferlinopathy |
| 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 |
| US20240390522A1 (en) * | 2021-09-28 | 2024-11-28 | University Of Florida Research Foundation, Incorporated | Gene therapy for duchenne muscular dystrophy |
| EP4219726A1 (en) | 2021-10-15 | 2023-08-02 | Research Institute at Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| WO2023196853A1 (en) * | 2022-04-05 | 2023-10-12 | Astellas Gene Therapies, Inc. | Compositions and methods for the treatment of muscular dystrophies |
| GB2633537A (en) * | 2022-05-18 | 2025-03-12 | Inadcure Found Inc | Gene therapies for treatment of infantile neuroaxonal dystrophy |
| JP2025535062A (ja) * | 2022-10-06 | 2025-10-22 | ソリッド・バイオサイエンシーズ・インコーポレーテッド | 二重トランスフェクションベクター |
| CN117959464A (zh) * | 2022-10-27 | 2024-05-03 | 苏州新芽基因生物技术有限公司 | 用于疾病治疗的基因编辑器和抗纤维化抑制剂的核酸药物组合物 |
| CN116218849B (zh) * | 2023-01-18 | 2024-01-23 | 昆明理工大学 | 针对utrophin激活治疗杜氏肌营养不良的sgRNA和应用 |
| EP4698558A2 (en) * | 2023-04-18 | 2026-02-25 | Research Institute at Nationwide Children's Hospital, Inc. | Gene therapy for treating limb girdle muscular dystrophy r9 and congenital muscular dystrophy 1c |
| WO2024229164A2 (en) * | 2023-05-03 | 2024-11-07 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase |
| CN116926125B (zh) * | 2023-09-07 | 2024-06-11 | 昆明理工大学 | 一种抑制炎症与基因编辑同时进行的基因载体 |
| WO2025085731A1 (en) * | 2023-10-18 | 2025-04-24 | Children's National Medical Center | TRANSFORMING GROWTH FACTOR ß (TGF-ß) OLIGONUCLEOTIDE COMBINATION THERAPY FOR TREATING MUSCULOSKELETAL DISEASES |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8314290B2 (en) * | 2004-12-21 | 2012-11-20 | Monsanto Technology Llc | Temporal regulation of gene expression by MicroRNAs |
| CN102311973A (zh) * | 2010-07-05 | 2012-01-11 | 北京五加和分子医学研究所有限公司 | 一种新型miRNA和编码蛋白基因共表达载体 |
| JP2014103905A (ja) * | 2012-11-28 | 2014-06-09 | Tokai Univ | 標的遺伝子のノックダウン方法およびそのためのＲＮＡｉ用発現ベクター |
| PL3097197T3 (pl) * | 2014-01-21 | 2021-06-28 | Vrije Universiteit Brussel | Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie |
| KR102244434B1 (ko) * | 2014-08-11 | 2021-04-23 | 삼성전자주식회사 | 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법 |
| 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 |
| US10543260B2 (en) * | 2015-07-22 | 2020-01-28 | The Board Of Trustees Of The Leland Stanford Junior University | Replacement gene tolerizing vectors and methods of use thereof |
| KR102787119B1 (ko) * | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| 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 |
| EP3570896B1 (en) * | 2017-01-23 | 2023-07-26 | Rutgers, The State University of New Jersey | Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use |
| WO2018200419A1 (en) * | 2017-04-23 | 2018-11-01 | The Trustees Of The University Of Pennsylvania | Viral vectors comprising engineered aav capsids and compositions containing the same |
| CA3078096A1 (en) * | 2017-10-20 | 2018-10-19 | Genethon | Use of syncytin for targeting drug and gene delivery to regenerating muscle tissue |
-
2019
- 2019-12-11 WO PCT/US2019/065718 patent/WO2020123645A1/en not_active Ceased
- 2019-12-11 JP JP2021533367A patent/JP7788855B2/ja active Active
- 2019-12-11 KR KR1020217021723A patent/KR20210124969A/ko not_active Ceased
- 2019-12-11 US US17/312,259 patent/US20220031865A1/en active Pending
- 2019-12-11 SG SG11202105873SA patent/SG11202105873SA/en unknown
- 2019-12-11 CA CA3123003A patent/CA3123003A1/en active Pending
- 2019-12-11 AU AU2019395388A patent/AU2019395388A1/en active Pending
- 2019-12-11 CN CN201980091591.9A patent/CN113646004A/zh active Pending
- 2019-12-11 EP EP19895501.5A patent/EP3893940A4/en not_active Withdrawn
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