JPWO2020123645A5 - - Google Patents
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- Publication number
- JPWO2020123645A5 JPWO2020123645A5 JP2021533367A JP2021533367A JPWO2020123645A5 JP WO2020123645 A5 JPWO2020123645 A5 JP WO2020123645A5 JP 2021533367 A JP2021533367 A JP 2021533367A JP 2021533367 A JP2021533367 A JP 2021533367A JP WO2020123645 A5 JPWO2020123645 A5 JP WO2020123645A5
- Authority
- JP
- Japan
- Prior art keywords
- recombinant viral
- viral vector
- sequence
- gene
- 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.)
- Granted
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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 (4)
| Publication Number | Publication Date |
|---|---|
| JP2022513456A JP2022513456A (ja) | 2022-02-08 |
| JPWO2020123645A5 true JPWO2020123645A5 (https=) | 2022-12-20 |
| JP2022513456A5 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 |
|---|---|---|---|---|
| CA3005633C (en) | 2015-11-16 | 2023-11-21 | Research Institute Of Nationwide Children's Hospital | Materials and methods for treatment of titin-based myopathies and other titinopathies |
| WO2019012336A2 (en) | 2017-03-17 | 2019-01-17 | Newcastle University | ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY |
| MX2020007876A (es) | 2018-01-31 | 2020-12-03 | Res Inst Nationwide Childrens Hospital | Terapia genica para la distrofia muscular de cinturas tipo 2c. |
| CN120393053A (zh) | 2018-06-18 | 2025-08-01 | 全国儿童医院研究所 | 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送 |
| MX2020014119A (es) | 2018-06-29 | 2021-06-18 | Res Inst Nationwide Childrens Hospital | Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a. |
| MX2021010356A (es) | 2019-02-26 | 2022-03-04 | Res Inst Nationwide Childrens Hospital | SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR. |
| PT4017871T (pt) | 2019-08-21 | 2024-04-22 | Res Inst Nationwide Childrens Hospital | Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular |
| EP4087617A4 (en) * | 2020-01-10 | 2024-05-01 | Solid Biosciences Inc. | VIRAL VECTOR FOR COMBINATION THERAPY |
| EP4164668A1 (en) | 2020-06-15 | 2023-04-19 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery 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 |
| JP2023059858A (ja) | 2021-10-15 | 2023-04-27 | 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 | 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用 |
| AR129014A1 (es) * | 2022-04-05 | 2024-07-03 | Astellas Gene Therapies Inc | Composiciones y métodos para el tratamiento de distrofias musculares |
| WO2023225592A2 (en) * | 2022-05-18 | 2023-11-23 | Inadcure Foundation Inc. | Gene therapies for treatment of infantile neuroaxonal dystrophy |
| IL320023A (en) * | 2022-10-06 | 2025-06-01 | Solid Biosciences Inc | Dual cell-to-cell gene transfer vector |
| 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 |
| WO2024220592A2 (en) * | 2023-04-18 | 2024-10-24 | 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 |
| EP3097197B1 (en) * | 2014-01-21 | 2020-12-16 | Vrije Universiteit Brussel | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| KR102244434B1 (ko) * | 2014-08-11 | 2021-04-23 | 삼성전자주식회사 | 재조합 벡터 및 이를 이용한 목적 폴리펩타이드의 생산 방법 |
| IL286316B2 (en) * | 2015-01-16 | 2023-03-01 | Univ Washington | New micro-dystrophins and related method of use |
| 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 |
| EA201891317A3 (ru) * | 2015-11-30 | 2019-04-30 | Дьюк Юниверсити | Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения |
| AU2017250791B2 (en) * | 2016-04-15 | 2022-11-24 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
| WO2018136880A1 (en) * | 2017-01-23 | 2018-07-26 | 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 |
| WO2019077149A1 (en) * | 2017-10-20 | 2019-04-25 | Genethon | USE OF SYNCYTINE FOR TARGETING DRUG AND GENE ADMINISTRATION TO REGENERATE MUSCLE TISSUE |
-
2019
- 2019-12-11 WO PCT/US2019/065718 patent/WO2020123645A1/en not_active Ceased
- 2019-12-11 AU AU2019395388A patent/AU2019395388A1/en active Pending
- 2019-12-11 JP JP2021533367A patent/JP7788855B2/ja active Active
- 2019-12-11 US US17/312,259 patent/US20220031865A1/en active Pending
- 2019-12-11 KR KR1020217021723A patent/KR20210124969A/ko not_active Ceased
- 2019-12-11 SG SG11202105873SA patent/SG11202105873SA/en unknown
- 2019-12-11 EP EP19895501.5A patent/EP3893940A4/en not_active Withdrawn
- 2019-12-11 CA CA3123003A patent/CA3123003A1/en active Pending
- 2019-12-11 CN CN201980091591.9A patent/CN113646004A/zh active Pending
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