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
Prior art date
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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/fr
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
EP18305399.0 2018-04-05
EP18305399 2018-04-05
PCT/EP2019/058560 WO2019193119A1 (fr) 2018-04-05 2019-04-04 Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit

Publications (3)

Publication Number Publication Date
JP2021520203A JP2021520203A (ja) 2021-08-19
JPWO2019193119A5 true JPWO2019193119A5 (fr) 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JP2021558887A Withdrawn JP2022526405A (ja) 2018-04-05 2019-10-04 肝向性の低減及び筋形質導入の増大を有するAAV9とAAVrh74とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型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JP2021558887A Withdrawn JP2022526405A (ja) 2018-04-05 2019-10-04 肝向性の低減及び筋形質導入の増大を有するAAV9とAAVrh74とのペプチド改変ハイブリッド組換えアデノ随伴ウイルス血清型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) US12037362B2 (fr)
EP (2) EP3775173A1 (fr)
JP (2) JP7374119B2 (fr)
KR (2) KR20210015770A (fr)
CN (1) CN113795574A (fr)
AU (2) AU2019249890A1 (fr)
BR (2) BR112020020223A2 (fr)
CA (2) CA3093347A1 (fr)
IL (2) IL277740A (fr)
MA (1) MA51353B1 (fr)
TN (1) TN2020000187A1 (fr)
WO (2) WO2019193119A1 (fr)
ZA (1) ZA202005422B (fr)

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* Cited by examiner, † Cited by third party
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CN112040988A (zh) * 2018-04-27 2020-12-04 海德堡大学 修饰的aav衣壳多肽用于治疗肌肉疾病
WO2021053124A1 (fr) 2019-09-19 2021-03-25 Genethon Système d'expression de thérapie génique atténuant la toxicité cardiaque de fkrp
EP4073087A4 (fr) * 2019-11-08 2023-11-29 The Board Of Regents Of The University Of Texas System Vecteur viral adéno-associé recombiné pour la délivrance de gènes
TW202129002A (zh) * 2019-12-09 2021-08-01 英商Ucl商業有限責任公司 用於myh7關聯之心肌病之基因療法組合物及治療
US20230032826A1 (en) 2019-12-18 2023-02-02 Genethon Production of recombinant viral vectors from plant hairy roots
WO2021229255A1 (fr) 2020-05-14 2021-11-18 Genethon Outils et procédé de prévention d'une neutralisation des virus vaa par des anticorps
EP3913060A1 (fr) 2020-05-22 2021-11-24 Genethon Vecteurs codant la glucose-6-phosphatase (g6pase-a) pour la thérapie génique
EP4162049A1 (fr) 2020-06-09 2023-04-12 Genethon Inhibiteurs de cilp-1 destinés à être utilisés dans le traitement de cardiomyopathies dilatées
EP4161559A1 (fr) 2020-06-09 2023-04-12 Genethon Activateur de nrf2 destiné à être utilisé dans le traitement de cardiomyopathies dilatées
CA3185497A1 (fr) 2020-06-09 2021-12-16 Genethon Traitement de cardiomyopathies dilatees genetiques
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
US20230242905A1 (en) 2020-07-03 2023-08-03 Genethon Method for Engineering Novel Hybrid AAV Capsids Through Hyper Variable Regions Swapping
CA3184923A1 (fr) 2020-07-10 2022-01-13 William LOSTAL Nouveau promoteur specifique des muscles
CN116096431A (zh) 2020-09-10 2023-05-09 吉尼松公司 肽修饰的aav衣壳
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
WO2022204476A1 (fr) 2021-03-26 2022-09-29 The Board Of Regents Of The University Of Texas System Édition de nucléotides pour remettre en phase des transcrits de la dmd par édition de base et édition génomique prémium (« prime editing »)
WO2022226301A1 (fr) * 2021-04-23 2022-10-27 The University Of North Carolina At Chapel Hill Capsides de vaa tropiques chimériques cardiaques
JP2024529540A (ja) 2021-08-04 2024-08-06 ジェネトン 筋肉及びcnsにおける遺伝子発現のためのハイブリッドプロモーター
AU2023211652A1 (en) 2022-01-25 2024-08-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023237731A1 (fr) 2022-06-09 2023-12-14 Genethon Gde tronquée au niveau du terminal n pour le traitement de la maladie de stockage du glycogène de type iii
WO2023237748A1 (fr) 2022-06-10 2023-12-14 Genethon Capside d'aav modifiée par un peptide présentant une efficacité de transduction musculaire améliorée
WO2024074727A1 (fr) 2022-10-07 2024-04-11 Genethon Immunothérapie cellulaire par car-t anti-fap pour traiter des myopathies squelettiques
WO2024079249A1 (fr) 2022-10-12 2024-04-18 Genethon Vecteur aav hybride améliorant l'expression d'un transgène dans le foie
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
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

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DK2292780T3 (en) 2003-09-30 2017-12-04 Univ Pennsylvania Clades and sequences of adeno-associated virus (AAV), vectors containing them, and uses thereof
WO2006110689A2 (fr) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Procede d'augmentation de la fonction d'un vecteur aav
ES2724800T3 (es) 2011-02-17 2019-09-16 Univ Pennsylvania Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
DK2839014T3 (da) 2012-04-18 2021-03-08 Childrens Hospital Philadelphia Sammensætning og fremgangsmåder til højeffektiv genoverførsel ved anvendelse af aav-capsid-varianter
US20140162319A2 (en) 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
CN116004721A (zh) 2013-07-12 2023-04-25 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
HUE047996T2 (hu) 2013-07-22 2020-05-28 Childrens Hospital Philadelphia AAV variáns és készítmények, eljárások és alkalmazások sejtekbe, szervekbe és szövetekbe történõ géntranszferhez
WO2015191508A1 (fr) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Capsides chimériques
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
CA3018076A1 (fr) 2016-03-18 2017-09-21 The Children's Hospital Of Philadelphia Agent therapeutique pour le traitement de maladies, notamment les maladies touchant le systeme nerveux central
SG11201811603WA (en) * 2016-07-26 2019-02-27 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
CN112533644A (zh) * 2018-04-03 2021-03-19 斯特里迪比奥公司 靶向眼组织的病毒载体

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