JP2021521833A5 - - Google Patents
Info
- Publication number
- JP2021521833A5 JP2021521833A5 JP2020560127A JP2020560127A JP2021521833A5 JP 2021521833 A5 JP2021521833 A5 JP 2021521833A5 JP 2020560127 A JP2020560127 A JP 2020560127A JP 2020560127 A JP2020560127 A JP 2020560127A JP 2021521833 A5 JP2021521833 A5 JP 2021521833A5
- Authority
- JP
- Japan
- Prior art keywords
- aav capsid
- capsid polypeptide
- muscle
- amino acid
- treating
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023191983A JP7707261B2 (ja) | 2018-04-27 | 2023-11-10 | 筋疾患の治療のための改変ａａｖカプシドポリペプチド |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18169822.6 | 2018-04-27 | ||
| EP18169822 | 2018-04-27 | ||
| PCT/EP2019/060790 WO2019207132A1 (en) | 2018-04-27 | 2019-04-26 | Modified aav capsid polypeptides for treatment of muscular diseases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023191983A Division JP7707261B2 (ja) | 2018-04-27 | 2023-11-10 | 筋疾患の治療のための改変ａａｖカプシドポリペプチド |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021521833A JP2021521833A (ja) | 2021-08-30 |
| JP2021521833A5 true JP2021521833A5 (https=) | 2022-05-09 |
Family
ID=62167107
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020560127A Pending JP2021521833A (ja) | 2018-04-27 | 2019-04-26 | 筋疾患の治療のための改変ａａｖカプシドポリペプチド |
| JP2023191983A Active JP7707261B2 (ja) | 2018-04-27 | 2023-11-10 | 筋疾患の治療のための改変ａａｖカプシドポリペプチド |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023191983A Active JP7707261B2 (ja) | 2018-04-27 | 2023-11-10 | 筋疾患の治療のための改変ａａｖカプシドポリペプチド |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210363193A1 (https=) |
| EP (1) | EP3784288A1 (https=) |
| JP (2) | JP2021521833A (https=) |
| CN (1) | CN112040988A (https=) |
| AU (2) | AU2019258830B2 (https=) |
| CA (1) | CA3097375A1 (https=) |
| WO (1) | WO2019207132A1 (https=) |
Families Citing this family (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2020011386A (es) | 2018-04-27 | 2021-01-29 | Spacecraft Seven Llc | Terapia genica para la degeneracion del snc. |
| CN113966399A (zh) | 2018-09-26 | 2022-01-21 | 加州理工学院 | 用于靶向基因疗法的腺相关病毒组合物 |
| WO2020101042A1 (en) | 2018-11-16 | 2020-05-22 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| EP3947700A4 (en) | 2019-04-01 | 2023-01-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| EP4028064A1 (en) * | 2019-09-12 | 2022-07-20 | The Broad Institute, Inc. | Engineered adeno-associated virus capsids |
| JP7651562B2 (ja) * | 2019-09-19 | 2025-03-26 | ジェネトン | Ｆｋｒｐの心毒性を軽減する遺伝子治療発現系 |
| TWI897882B (zh) * | 2019-09-20 | 2025-09-21 | 英商Ｕｃｌ商業有限責任公司 | 右心室心律失常性心肌病之基因療法組合物及治療 |
| EP4041755A4 (en) * | 2019-10-10 | 2023-10-25 | Solid Biosciences Inc. | Modified aav capsids and uses thereof |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| JP2023523573A (ja) | 2020-04-14 | 2023-06-06 | ジェネトン | 酸性セラミダーゼ欠乏症を処置するためのベクター |
| US12552838B2 (en) | 2020-04-20 | 2026-02-17 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| US20230173102A1 (en) * | 2020-04-28 | 2023-06-08 | Genethon | Use of a Synthetic AAV Capsid for Gene Therapy of Muscle and Central Nervous System Disorders |
| EP4126912A4 (en) * | 2020-05-05 | 2024-04-24 | Duke University | COMPOSITIONS AND METHODS FOR FORMING AND SECRETING EXTRACELLULAR VESILES AND AAV PARTICLES |
| WO2021230385A1 (en) | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| US20230159949A1 (en) * | 2020-07-22 | 2023-05-25 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
| KR20230053591A (ko) * | 2020-07-22 | 2023-04-21 | 더 브로드 인스티튜트, 인코퍼레이티드 | 조작된 근육 표적화 조성물 |
| KR20230042468A (ko) * | 2020-08-05 | 2023-03-28 | 스페이스크래프트 세븐, 엘엘씨 | Csrp3 (시스테인 및 글리신 풍부 단백질 3) 유전자 요법 |
| KR20230043869A (ko) | 2020-08-07 | 2023-03-31 | 스페이스크래프트 세븐, 엘엘씨 | Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법 |
| EP4421179A3 (en) | 2020-09-10 | 2024-12-18 | Genethon | Peptide-modified aav capsid |
| CN113754727B (zh) * | 2020-09-30 | 2022-07-12 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| BR112023006305A2 (pt) * | 2020-10-09 | 2023-05-09 | Tenaya Therapeutics Inc | Métodos e composições de terapia gênica com placofilina-2 |
| US11781156B2 (en) | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
| AU2021378075B2 (en) | 2020-11-11 | 2025-03-27 | Borea Therapeutics S.R.L. | Modified viral particles for gene therapy |
| WO2022114243A1 (en) * | 2020-11-25 | 2022-06-02 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting dmpk gene |
| US20240115734A1 (en) * | 2021-02-09 | 2024-04-11 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| CN113480615B (zh) * | 2021-07-30 | 2022-05-31 | 上海信致医药科技有限公司 | 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| CN113563430B (zh) * | 2021-07-30 | 2022-05-31 | 上海信致医药科技有限公司 | 用于治疗眼部疾病的基因递送系统及其应用 |
| WO2023060113A1 (en) | 2021-10-05 | 2023-04-13 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
| CN118202060A (zh) | 2021-10-05 | 2024-06-14 | 再生生物股份有限公司 | 用于重组aav生产的组合物和方法 |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| AU2022420596A1 (en) * | 2021-12-21 | 2024-07-11 | Research Institute At Nationwide Children's Hospital | Materials and methods for the treatment of limb girdle muscular dystrophy |
| CN114516901B (zh) * | 2022-03-11 | 2023-01-17 | 上海勉亦生物科技有限公司 | 一种神经系统高亲和性的aav载体及其应用 |
| EP4493702A4 (en) * | 2022-03-18 | 2026-04-01 | Univ Florida | METHODS AND COMPOSITIONS FOR TREATING TNNT2-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR |
| WO2023196862A1 (en) | 2022-04-06 | 2023-10-12 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2023198828A1 (en) | 2022-04-13 | 2023-10-19 | Universitat Autònoma De Barcelona | Treatment of neuromuscular diseases via gene therapy that expresses klotho protein |
| EP4269426A1 (en) * | 2022-04-29 | 2023-11-01 | Ospedale San Raffaele S.r.l. | Gene therapy |
| ES3057171T3 (en) | 2022-06-10 | 2026-02-26 | Genethon | Peptide-modified aav capsid with enhanced muscle transduction efficiency |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| EP4677073A1 (en) | 2023-03-10 | 2026-01-14 | Genethon | Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter |
| EP4680741A2 (en) | 2023-03-15 | 2026-01-21 | RegenxBio Inc. | Exon skipping gene therapy constructs, vectors and uses thereof |
| US20240384271A1 (en) | 2023-04-05 | 2024-11-21 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| WO2024238807A2 (en) | 2023-05-16 | 2024-11-21 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| CN121712809A (zh) | 2023-07-03 | 2026-03-20 | Ucb生物制药有限责任公司 | 工程化的aav衣壳蛋白 |
| CN116970041B (zh) * | 2023-07-21 | 2024-02-09 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| AU2024332181A1 (en) | 2023-08-31 | 2026-02-12 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2025133247A1 (en) | 2023-12-21 | 2025-06-26 | Genethon | Methods for aav vector re-administration |
| TW202545971A (zh) | 2024-02-08 | 2025-12-01 | 美商戴諾治療公司 | 殼體多肽及其使用方法 |
| WO2025232917A1 (en) * | 2024-05-10 | 2025-11-13 | Hangzhou Jiayin Biotech Ltd. | Novel liver detargeted aav variants and uses thereof |
| WO2026006341A1 (en) | 2024-06-24 | 2026-01-02 | Regenxbio Inc. | Microdystrophin gene therapy administration for treatment of dystrophinopathies |
| WO2026017727A1 (en) | 2024-07-16 | 2026-01-22 | Genethon | Alternative barcoding method to assess transduction efficiency of aav vectors |
| WO2026064428A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064444A1 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6833447B1 (en) * | 2000-07-10 | 2004-12-21 | Monsanto Technology, Llc | Myxococcus xanthus genome sequences and uses thereof |
| EP1456383B1 (en) * | 2001-12-21 | 2014-03-12 | Medigene AG | A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism |
| US8927269B2 (en) * | 2003-05-19 | 2015-01-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Avian adenoassociated virus and uses thereof |
| US7467102B2 (en) * | 2003-09-11 | 2008-12-16 | International Business Machines Corporation | Request type grid computing |
| US20130305398A1 (en) * | 2012-02-16 | 2013-11-14 | Marie Coffin | Genes and uses for plant enhacement |
| WO2013170078A1 (en) * | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| BR112015023272A2 (pt) * | 2013-03-14 | 2017-07-18 | Pioneer Hi Bred Int | célula vegetal, planta, explante vegetal, semente transgênica, método para produzir uma célula vegetal tendo um polinucleotídeo heterólogo que codifica um polipeptídeo tendo atividade de dicamba descarboxilase, método para controlar plantas daninhas em um campo contendo uma cultura e método para controlar plantas daninhas em um campo contendo uma cultura |
| EP3384034B1 (en) * | 2015-12-02 | 2020-07-08 | The Board of Trustees of the Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
| US20190224339A1 (en) * | 2016-04-29 | 2019-07-25 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2018129203A2 (en) * | 2017-01-06 | 2018-07-12 | The Regents Of The University Of California | Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination |
| EP3728568A4 (en) * | 2017-12-19 | 2021-10-06 | The University of North Carolina at Chapel Hill | METHODS AND COMPOSITIONS FOR DELIVERING VIRAL VECTORS BY THE BLOOD-BRAIN BARRIER |
| MA51353B1 (fr) * | 2018-04-05 | 2022-09-30 | Univ Sorbonne | Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit |
-
2019
- 2019-04-26 US US17/051,123 patent/US20210363193A1/en active Pending
- 2019-04-26 CA CA3097375A patent/CA3097375A1/en active Pending
- 2019-04-26 JP JP2020560127A patent/JP2021521833A/ja active Pending
- 2019-04-26 WO PCT/EP2019/060790 patent/WO2019207132A1/en not_active Ceased
- 2019-04-26 EP EP19722029.6A patent/EP3784288A1/en active Pending
- 2019-04-26 AU AU2019258830A patent/AU2019258830B2/en active Active
- 2019-04-26 CN CN201980028398.0A patent/CN112040988A/zh active Pending
-
2023
- 2023-11-10 JP JP2023191983A patent/JP7707261B2/ja active Active
-
2026
- 2026-03-18 AU AU2026202070A patent/AU2026202070A1/en active Pending
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