JPWO2019193119A5 - - Google Patents
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- Publication number
- JPWO2019193119A5 JPWO2019193119A5 JP2020554181A JP2020554181A JPWO2019193119A5 JP WO2019193119 A5 JPWO2019193119 A5 JP WO2019193119A5 JP 2020554181 A JP2020554181 A JP 2020554181A JP 2020554181 A JP2020554181 A JP 2020554181A JP WO2019193119 A5 JPWO2019193119 A5 JP WO2019193119A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- sequence
- recombinant
- 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 | 2018-04-05 | ||
EP18305399.0 | 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 |
JPWO2019193119A5 true JPWO2019193119A5 (hr) | 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JP2021558887A Withdrawn JP2022526405A (ja) | 2018-04-05 | 2019-10-04 | 肝向性の低減及び筋形質導入の増大を有するAAV9とAAVrh74とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型 |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JP2021558887A Withdrawn JP2022526405A (ja) | 2018-04-05 | 2019-10-04 | 肝向性の低減及び筋形質導入の増大を有するAAV9とAAVrh74とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型 |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 (2) | US20210107948A1 (hr) |
EP (2) | EP3775173A1 (hr) |
JP (2) | JP7374119B2 (hr) |
KR (2) | KR20210015770A (hr) |
CN (2) | CN112004925A (hr) |
AU (2) | AU2019249890A1 (hr) |
BR (2) | BR112020020223A2 (hr) |
CA (2) | CA3093347A1 (hr) |
IL (2) | IL277740A (hr) |
MA (1) | MA51353B1 (hr) |
TN (1) | TN2020000187A1 (hr) |
WO (2) | WO2019193119A1 (hr) |
ZA (1) | ZA202005422B (hr) |
Families Citing this family (28)
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 |
CN114521143A (zh) | 2019-09-19 | 2022-05-20 | 吉尼松公司 | 减轻fkrp心脏毒性的基因治疗表达系统 |
EP4073087A4 (en) * | 2019-11-08 | 2023-11-29 | The Board Of Regents Of The University Of Texas System | RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR FOR GENE DELIVERY |
US20230047424A1 (en) * | 2019-12-09 | 2023-02-16 | Ucl Business Ltd | Gene therapy composition and treatment for myh7-linked cardiomyopathy |
JP2023507460A (ja) | 2019-12-18 | 2023-02-22 | ジェネトン | 植物毛状根からの組換えウイルスベクターの生産 |
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 |
JP2023528661A (ja) | 2020-06-09 | 2023-07-05 | ジェネトン | 拡張型心筋症の処置における使用のためのnrf2活性化剤 |
EP4162049A1 (en) | 2020-06-09 | 2023-04-12 | Genethon | Cilp-1 inhibitors for use in the treatment of dilated cardiomyopathies |
JP2023528663A (ja) | 2020-06-09 | 2023-07-05 | ジェネトン | 遺伝性拡張型心筋症の処置 |
CA3182313A1 (en) | 2020-06-19 | 2021-12-23 | Isabelle Richard | Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg |
WO2022003211A1 (en) | 2020-07-03 | 2022-01-06 | Genethon | Method for engineering novel hybrid aav capsids through hypervariable regions swapping |
BR112023000305A2 (pt) | 2020-07-10 | 2023-03-21 | Genethon | Promotor, sistema de expressão e composição farmacêutica |
BR112023004176A2 (pt) | 2020-09-10 | 2023-04-11 | Genethon | Capsídeo de aav modificado por peptídeo |
CA3200563A1 (en) | 2020-09-29 | 2022-04-07 | Genethon | Enhancing utrophin expression in cell by inducing mutations within utrophin regulatory elements and therapeutic use thereof |
EP4273236A1 (en) * | 2020-12-29 | 2023-11-08 | Jiangsu Genscript Probio Biotech Co., Ltd. | Hek293t cell strain having high dispersibility and screening method therefor |
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 |
CA3226119A1 (en) | 2021-08-04 | 2023-02-09 | Giuseppe RONZITTI | Hybrid promoters for gene expression in muscles and in the cns |
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 |
WO2023147304A1 (en) | 2022-01-25 | 2023-08-03 | The Trustees Of The University Of Pennsylvania | Aav capsids for improved heart transduction and detargeting of liver |
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 |
WO2023237731A1 (en) | 2022-06-09 | 2023-12-14 | Genethon | N-terminal truncated gde for the treatment of glycogen storage disease iii |
WO2023237748A1 (en) | 2022-06-10 | 2023-12-14 | Genethon | Peptide-modified aav capsid with enhanced muscle transduction efficiency |
WO2024074727A1 (en) | 2022-10-07 | 2024-04-11 | Genethon | Immunotherapy of skeletal myopathies using anti-fap car-t cells |
WO2024079249A1 (en) | 2022-10-12 | 2024-04-18 | 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 |
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 |
Family Cites Families (15)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ES2411479T3 (es) | 2003-09-30 | 2013-07-05 | The Trustees Of The University Of Pennsylvania | Clados de virus adeno-asociados (AAV), secuencias, vectores que los contienen, y usos de los mismos |
EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
EP2675902B1 (en) | 2011-02-17 | 2019-03-27 | The Trustees Of The University Of Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
WO2013123503A1 (en) | 2012-02-17 | 2013-08-22 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
EP2839014B1 (en) | 2012-04-18 | 2020-12-16 | 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 |
CN105612253A (zh) * | 2013-03-15 | 2016-05-25 | 费城儿童医院 | 含有填充者/填充物多核苷酸序列的载体及其制备方法 |
KR102268473B1 (ko) * | 2013-04-20 | 2021-06-25 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달 |
CN105518145A (zh) * | 2013-07-12 | 2016-04-20 | 费城儿童医院 | Aav载体和用于抗aav(腺相关病毒)中和抗体的检测 |
IL297919A (en) | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Modified Aav and preparations, methods and uses for gene transfer to cells, organs and tissues |
WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
BR112017005892A2 (pt) | 2014-09-24 | 2017-12-12 | Hope City | variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma |
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 |
CN109715650B (zh) * | 2016-07-26 | 2024-03-08 | 生物马林药物股份有限公司 | 新颖腺相关病毒衣壳蛋白 |
EA202092362A1 (ru) * | 2018-04-03 | 2021-01-11 | Страйдбайо, Инк. | Вирусные векторы для нацеливания на ткани глаза |
-
2019
- 2019-04-04 KR KR1020207032024A patent/KR20210015770A/ko unknown
- 2019-04-04 WO PCT/EP2019/058560 patent/WO2019193119A1/en active Application Filing
- 2019-04-04 JP JP2020554181A patent/JP7374119B2/ja active Active
- 2019-04-04 US US16/981,086 patent/US20210107948A1/en active Pending
- 2019-04-04 CN CN201980023352.XA patent/CN112004925A/zh active Pending
- 2019-04-04 EP EP19714230.0A patent/EP3775173A1/en active Pending
- 2019-04-04 MA MA51353A patent/MA51353B1/fr unknown
- 2019-04-04 CA CA3093347A patent/CA3093347A1/en active Pending
- 2019-04-04 TN TNP/2020/000187A patent/TN2020000187A1/en unknown
- 2019-04-04 AU AU2019249890A patent/AU2019249890A1/en active Pending
- 2019-04-04 BR BR112020020223-1A patent/BR112020020223A2/pt unknown
- 2019-10-04 EP EP19782603.5A patent/EP3947653A1/en not_active Withdrawn
- 2019-10-04 KR KR1020217031502A patent/KR20210153047A/ko not_active Application Discontinuation
- 2019-10-04 CA CA3134183A patent/CA3134183A1/en not_active Withdrawn
- 2019-10-04 US US17/600,715 patent/US20220228167A1/en not_active Abandoned
- 2019-10-04 WO PCT/EP2019/076958 patent/WO2020200499A1/en unknown
- 2019-10-04 BR BR112021019821A patent/BR112021019821A2/pt not_active Application Discontinuation
- 2019-10-04 AU AU2019439673A patent/AU2019439673A1/en not_active Withdrawn
- 2019-10-04 JP JP2021558887A patent/JP2022526405A/ja not_active Withdrawn
- 2019-10-04 CN CN201980095204.9A patent/CN113795574A/zh active Pending
-
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
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