AU2019249890B2 - Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism - Google Patents

Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism Download PDF

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AU2019249890B2
AU2019249890B2 AU2019249890A AU2019249890A AU2019249890B2 AU 2019249890 B2 AU2019249890 B2 AU 2019249890B2 AU 2019249890 A AU2019249890 A AU 2019249890A AU 2019249890 A AU2019249890 A AU 2019249890A AU 2019249890 B2 AU2019249890 B2 AU 2019249890B2
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AU2019249890A1 (en
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Evelyne GICQUEL
Federico Mingozzi
Isabelle Richard
Giuseppe RONZITTI
Patrice VIDAL
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Institut National de la Sante et de la Recherche Medicale INSERM
Genethon
Universite D'Evry Val D'Essonne
Association Institut de Myologie
Sorbonne Universite
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Institut National de la Sante et de la Recherche Medicale INSERM
Genethon
Universite D'Evry Val D'Essonne
Association Institut de Myologie
Sorbonne Universite
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    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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AU2019249890A 2018-04-05 2019-04-04 Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism Active AU2019249890B2 (en)

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

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AU2019249890A1 AU2019249890A1 (en) 2020-09-24
AU2019249890B2 true AU2019249890B2 (en) 2024-10-10

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AU2019249890A Active AU2019249890B2 (en) 2018-04-05 2019-04-04 Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism
AU2019439673A Withdrawn AU2019439673A1 (en) 2018-04-05 2019-10-04 Peptide-modified hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism and increased muscle transduction

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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)

* Cited by examiner, † Cited by third party
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015006743A1 (en) * 2013-07-12 2015-01-15 The Children's Hospital Of Philadelphia Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies

Family Cites Families (15)

* Cited by examiner, † Cited by third party
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.
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.

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015006743A1 (en) * 2013-07-12 2015-01-15 The Children's Hospital Of Philadelphia Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies

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