JPWO2019193119A5 - - Google Patents

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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
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JP
Japan
Prior art keywords
aav
sequence
recombinant
aav9
aavrh74
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JP2020554181A
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English (en)
Japanese (ja)
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JP2021520203A (ja
JP7374119B2 (ja
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Priority claimed from PCT/EP2019/058560 external-priority patent/WO2019193119A1/en
Publication of JP2021520203A publication Critical patent/JP2021520203A/ja
Publication of JPWO2019193119A5 publication Critical patent/JPWO2019193119A5/ja
Application granted granted Critical
Publication of JP7374119B2 publication Critical patent/JP7374119B2/ja
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JP2020554181A 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 Active JP7374119B2 (ja)

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

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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JP2021558887A Withdrawn JP2022526405A (ja) 2018-04-05 2019-10-04 肝向性の低減及び筋形質導入の増大を有するAAV9とAAVrh74とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型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JP2021558887A Withdrawn JP2022526405A (ja) 2018-04-05 2019-10-04 肝向性の低減及び筋形質導入の増大を有するAAV9とAAVrh74とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型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)

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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

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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
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