JP2021520203A5 - - Google Patents
Info
- Publication number
- JP2021520203A5 JP2021520203A5 JP2020554181A JP2020554181A JP2021520203A5 JP 2021520203 A5 JP2021520203 A5 JP 2021520203A5 JP 2020554181 A JP2020554181 A JP 2020554181A JP 2020554181 A JP2020554181 A JP 2020554181A JP 2021520203 A5 JP2021520203 A5 JP 2021520203A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- sequence
- capsid
- aav9
- aavrh74
- 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 |
|---|---|---|---|
| EP18305399.0 | 2018-04-05 | ||
| EP18305399 | 2018-04-05 | ||
| PCT/EP2019/058560 WO2019193119A1 (en) | 2018-04-05 | 2019-04-04 | HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021520203A JP2021520203A (ja) | 2021-08-19 |
| JP2021520203A5 true JP2021520203A5 (https=) | 2022-04-25 |
| JP7374119B2 JP7374119B2 (ja) | 2023-11-06 |
Family
ID=62063468
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020554181A Active JP7374119B2 (ja) | 2018-04-05 | 2019-04-04 | 減少した肝臓向性を有するＡＡＶ９とＡＡＶｒｈ７４とのハイブリッド組換えアデノ随伴ウイルス血清型 |
| JP2021558887A Withdrawn JP2022526405A (ja) | 2018-04-05 | 2019-10-04 | 肝向性の低減及び筋形質導入の増大を有するＡＡＶ９とＡＡＶｒｈ７４とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021558887A Withdrawn JP2022526405A (ja) | 2018-04-05 | 2019-10-04 | 肝向性の低減及び筋形質導入の増大を有するＡＡＶ９とＡＡＶｒｈ７４とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型 |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US12037362B2 (https=) |
| EP (2) | EP3775173A1 (https=) |
| JP (2) | JP7374119B2 (https=) |
| KR (2) | KR20210015770A (https=) |
| CN (2) | CN112004925B (https=) |
| AU (2) | AU2019249890B2 (https=) |
| BR (2) | BR112020020223A2 (https=) |
| CA (2) | CA3093347A1 (https=) |
| IL (2) | IL277740A (https=) |
| MA (1) | MA51353B1 (https=) |
| TN (1) | TN2020000187A1 (https=) |
| WO (2) | WO2019193119A1 (https=) |
| ZA (1) | ZA202005422B (https=) |
Families Citing this family (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20210363193A1 (en) * | 2018-04-27 | 2021-11-25 | Universität Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
| US12466864B2 (en) | 2019-03-25 | 2025-11-11 | Genethon | Production of large-sized quasidystrophins using overlapping AAV vectors |
| CN114127088B (zh) * | 2019-04-23 | 2024-05-14 | 国家医疗保健研究所 | 变体衣壳蛋白和腺相关病毒aav变体及其药物组合物 |
| 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 |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| US12188039B2 (en) | 2019-11-08 | 2025-01-07 | The Board Of Regents Of The University Of Texas System | Recombinant adeno-associated viral vector for gene delivery |
| EP4073257A1 (en) * | 2019-12-09 | 2022-10-19 | UCL Business Ltd | Gene therapy composition and treatment for myh7-linked cardiomyopathy |
| EP4077684A1 (en) | 2019-12-18 | 2022-10-26 | Genethon | Production of recombinant viral vectors from plant hairy roots |
| WO2021229255A1 (en) | 2020-05-14 | 2021-11-18 | Genethon | Tools and method for preventing a a¥ neutralization by antibodies |
| EP3913060A1 (en) | 2020-05-22 | 2021-11-24 | Genethon | Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy |
| CN116322790A (zh) | 2020-06-09 | 2023-06-23 | 吉尼松公司 | 用于治疗扩张型心肌病的nrf2激活剂 |
| US20230220391A1 (en) | 2020-06-09 | 2023-07-13 | Genethon | CILP-1 Inhibitors for Use in the Treatment of Dilated Cardiomyopathies |
| WO2021250076A1 (en) | 2020-06-09 | 2021-12-16 | Genethon | Treatment of genetic dilated cardiomyopathies |
| US20230256117A1 (en) | 2020-06-19 | 2023-08-17 | Genethon | Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg |
| CA3187635A1 (en) | 2020-07-03 | 2022-01-06 | Genethon | Method for engineering novel hybrid aav capsids through hypervariable regions swapping |
| EP4179096A1 (en) | 2020-07-10 | 2023-05-17 | Genethon | A novel muscle-specific promoter |
| EP4421179A3 (en) | 2020-09-10 | 2024-12-18 | Genethon | Peptide-modified aav capsid |
| US20230272433A1 (en) | 2020-09-29 | 2023-08-31 | Genethon | Enhancing Utrophin Expression in Cell by Inducing Mutations Within Utrophin Regulatory Elements and Therapeutic Use Thereof |
| KR20230117177A (ko) * | 2020-12-03 | 2023-08-07 | 유니버시티 오브 매사추세츠 | 진행성 골화성 섬유이형성증을 위한 신규 유전자 치료제의개발 |
| EP4273236A4 (en) * | 2020-12-29 | 2025-01-08 | Jiangsu Genscript Probio Biotech Co., Ltd. | HEK293T CELL STRAIN WITH HIGH DISPERSIBILITY AND SCREENING METHODS THEREFOR |
| US20240115734A1 (en) * | 2021-02-09 | 2024-04-11 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| US20240165271A1 (en) | 2021-03-26 | 2024-05-23 | The Board Of Regents Of The University Of Texas System | Nucleotide editing to reframe dmd transcripts by base editing and prime editing |
| WO2022226301A1 (en) * | 2021-04-23 | 2022-10-27 | The University Of North Carolina At Chapel Hill | Chimeric heart tropic aav capsids |
| EP4381077A1 (en) | 2021-08-04 | 2024-06-12 | Genethon | Hybrid promoters for gene expression in muscles and in the cns |
| CN115232818B (zh) * | 2021-09-18 | 2025-10-21 | 南京启真基因工程有限公司 | 构建dok7基因突变的先天性肌无力模型猪核移植供体细胞的基因编辑系统及其应用 |
| US20230211014A1 (en) * | 2021-12-29 | 2023-07-06 | Genetic Cures for Kids Inc. | AAV Gene Therapy for Spastic Paraplegia |
| CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| CN119317718A (zh) | 2022-01-25 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于改善心脏转导和肝脏去靶向的aav衣壳 |
| CN114657152A (zh) * | 2022-03-31 | 2022-06-24 | 苏州博腾生物制药有限公司 | 一种新型aav血清型及其制备方法 |
| CN116925192B (zh) * | 2022-04-12 | 2025-04-29 | 合肥星眸生物科技有限公司 | 一种融合型腺相关病毒及其应用 |
| WO2023237731A1 (en) | 2022-06-09 | 2023-12-14 | Genethon | N-terminal truncated gde for the treatment of glycogen storage disease iii |
| ES3057171T3 (en) | 2022-06-10 | 2026-02-26 | Genethon | Peptide-modified aav capsid with enhanced muscle transduction efficiency |
| CN120112308A (zh) | 2022-10-07 | 2025-06-06 | 吉尼松公司 | 使用抗fap car-t细胞免疫治疗骨骼肌病 |
| EP4602055A1 (en) | 2022-10-12 | 2025-08-20 | Genethon | Hybrid aav vector enhancing transgene expression in the liver |
| CN116041443B (zh) * | 2022-12-30 | 2023-09-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2024173802A2 (en) * | 2023-02-17 | 2024-08-22 | Biogen Ma Inc. | Rgd-containing peptides for delivering payloads |
| CN116693633B (zh) * | 2023-02-21 | 2023-12-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| 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 |
| CN121420060A (zh) | 2023-06-29 | 2026-01-27 | 吉尼松公司 | 用于治疗糖原贮积病iii的n-端截短的糖原脱支酶 |
| KR20260035966A (ko) * | 2023-07-10 | 2026-03-13 | 케이트 테라퓨틱스, 인크. | Lgmd2b/디스펄린병증을 치료하기 위한 aav 유전자 치료 접근법 |
| WO2025017168A1 (en) | 2023-07-19 | 2025-01-23 | Genethon | Novel optimized utrophin micro-genes |
| CN121605119A (zh) | 2023-07-20 | 2026-03-03 | 吉尼松公司 | 新的中型肌营养不良蛋白 |
| 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 |
| CN117031014A (zh) * | 2023-08-14 | 2023-11-10 | 陕西脉元生物科技有限公司 | 抗matr3自身抗体的试剂在制备检测神经系统自身免疫疾病的产品中的应用及试剂盒 |
| WO2025072698A2 (en) * | 2023-09-27 | 2025-04-03 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Aav9 vectors and capsid polypeptides for use in treating cns disorders |
| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2025137378A1 (en) * | 2023-12-20 | 2025-06-26 | Aavantibio, Inc. | Aav capsid engineering for cardiac and/or musculoskeletal gene therapy |
| WO2025133247A1 (en) | 2023-12-21 | 2025-06-26 | Genethon | Methods for aav vector re-administration |
| WO2025232917A1 (en) * | 2024-05-10 | 2025-11-13 | Hangzhou Jiayin Biotech Ltd. | Novel liver detargeted aav variants and uses thereof |
| WO2026017727A1 (en) | 2024-07-16 | 2026-01-22 | Genethon | Alternative barcoding method to assess transduction efficiency of aav vectors |
| WO2026041758A1 (en) | 2024-08-22 | 2026-02-26 | Genethon | Modified aav capsid with enhanced transduction efficiency |
| WO2026064736A1 (en) * | 2024-09-23 | 2026-03-26 | Carbon Biosciences, Inc. | Parvovirus compositions and related methods for gene therapy |
| WO2026064739A1 (en) * | 2024-09-23 | 2026-03-26 | Carbon Biosciences, Inc. | Parvovirus compositions and related methods for gene therapy |
| CN120842333A (zh) * | 2025-06-20 | 2025-10-28 | 上海朗昇生物科技有限公司 | 工程化的腺相关病毒衣壳蛋白和载体及其用途 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| TR201908020T4 (tr) | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
| MX367100B (es) | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. |
| NZ701693A (en) | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| EP2660325A3 (en) | 2012-05-02 | 2014-02-12 | Christian Medical College | AAV vectors and corresponding nucleotide sequences and methods |
| HK1220488A1 (zh) * | 2013-03-15 | 2017-05-05 | The Children's Hospital Of Philadelphia | 含有填充者/填充物多核苷酸序列的载体及其使用方法 |
| MX388221B (es) * | 2013-04-20 | 2025-03-19 | Res Institute At Nationwide Children´S Hospital | ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2. |
| HK1222673A1 (zh) | 2013-07-12 | 2017-07-07 | The Children's Hospital Of Philadelphia | Aav载体和用於抗aav(腺相关病毒)中和抗体的检测 |
| IL297919A (en) | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| 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 |
| WO2017161273A1 (en) | 2016-03-18 | 2017-09-21 | The Children's Hospital Of Philadelphia | Therapeutic for treatment of diseases including the central nervous system |
| IL305149A (en) * | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| MX2020010465A (es) * | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. |
-
2019
- 2019-04-04 MA MA51353A patent/MA51353B1/fr unknown
- 2019-04-04 BR BR112020020223-1A patent/BR112020020223A2/pt unknown
- 2019-04-04 US US16/981,086 patent/US12037362B2/en active Active
- 2019-04-04 KR KR1020207032024A patent/KR20210015770A/ko not_active Ceased
- 2019-04-04 JP JP2020554181A patent/JP7374119B2/ja active Active
- 2019-04-04 CN CN201980023352.XA patent/CN112004925B/zh active Active
- 2019-04-04 WO PCT/EP2019/058560 patent/WO2019193119A1/en not_active Ceased
- 2019-04-04 TN TNP/2020/000187A patent/TN2020000187A1/en unknown
- 2019-04-04 CA CA3093347A patent/CA3093347A1/en active Pending
- 2019-04-04 AU AU2019249890A patent/AU2019249890B2/en active Active
- 2019-04-04 EP EP19714230.0A patent/EP3775173A1/en active Pending
- 2019-10-04 JP JP2021558887A patent/JP2022526405A/ja not_active Withdrawn
- 2019-10-04 EP EP19782603.5A patent/EP3947653A1/en not_active Withdrawn
- 2019-10-04 BR BR112021019821A patent/BR112021019821A2/pt not_active Application Discontinuation
- 2019-10-04 CA CA3134183A patent/CA3134183A1/en not_active Withdrawn
- 2019-10-04 KR KR1020217031502A patent/KR20210153047A/ko not_active Withdrawn
- 2019-10-04 WO PCT/EP2019/076958 patent/WO2020200499A1/en not_active Ceased
- 2019-10-04 CN CN201980095204.9A patent/CN113795574A/zh active Pending
- 2019-10-04 AU AU2019439673A patent/AU2019439673A1/en not_active Withdrawn
- 2019-10-04 US US17/600,715 patent/US20220228167A1/en not_active Abandoned
-
2020
- 2020-08-31 ZA ZA2020/05422A patent/ZA202005422B/en unknown
- 2020-10-01 IL IL277740A patent/IL277740A/en unknown
-
2021
- 2021-10-03 IL IL286926A patent/IL286926A/en unknown
-
2024
- 2024-06-06 US US18/735,636 patent/US20240317814A1/en active Pending
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