CN114008194A - 用于基因疗法的经修饰的腺相关病毒（aav）颗粒 - Google Patents

用于基因疗法的经修饰的腺相关病毒（aav）颗粒 Download PDF

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Publication number
CN114008194A
CN114008194A CN202080044805.XA CN202080044805A CN114008194A CN 114008194 A CN114008194 A CN 114008194A CN 202080044805 A CN202080044805 A CN 202080044805A CN 114008194 A CN114008194 A CN 114008194A
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aav
binding site
capsid
disease
ligand
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Chinese (zh)
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P·赫彭斯托尔
M·马费伊
F·德卡斯特罗·里斯
K·M·珀夫
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Europaisches Laboratorium fuer Molekularbiologie EMBL
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Europaisches Laboratorium fuer Molekularbiologie EMBL
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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CN202080044805.XA 2019-05-08 2020-05-07 用于基因疗法的经修饰的腺相关病毒（aav）颗粒 Pending CN114008194A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP19173365.8 2019-05-08
EP19173365.8A EP3736330A1 (en) 2019-05-08 2019-05-08 Modified adeno-associated virus (aav) particles for gene therapy
PCT/EP2020/062713 WO2020225363A2 (en) 2019-05-08 2020-05-07 Modified adeno-associated virus (aav) particles for gene therapy

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US (1) US20220288234A1 (https=)
EP (2) EP3736330A1 (https=)
JP (2) JP7737312B2 (https=)
KR (1) KR20220008863A (https=)
CN (1) CN114008194A (https=)
AU (1) AU2020268759A1 (https=)
BR (1) BR112021022385A2 (https=)
CA (1) CA3139183A1 (https=)
IL (1) IL287614A (https=)
MX (1) MX2021013619A (https=)
SG (1) SG11202112029XA (https=)
WO (1) WO2020225363A2 (https=)

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CA3201533A1 (en) 2020-11-11 2022-05-19 European Molecular Biology Laboratory Modified viral particles for gene therapy
CN118109515A (zh) * 2022-11-29 2024-05-31 中国科学院深圳先进技术研究院 一种基于生物正交技术的病毒载体及其制备方法和应用
CN121889172A (zh) 2023-07-21 2026-04-17 意大利商博雷亚医疗有限责任公司 制备表面修饰病毒衣壳的方法

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105408486A (zh) * 2013-05-21 2016-03-16 佛罗里达大学研究基金会有限公司 衣壳修饰的raav3载体组合物以及在人肝癌基因治疗中的用途
WO2017189963A1 (en) * 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2019006043A1 (en) * 2017-06-27 2019-01-03 Regeneron Pharmaceuticals, Inc. RECOMBINANT VIRAL VECTORS WITH MODIFIED TROPISM AND USES THEREOF FOR TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS
CN109476707A (zh) * 2016-05-13 2019-03-15 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
EP3461836A1 (en) * 2017-09-28 2019-04-03 Universität zu Köln Mutated adeno-associated viral capsid proteins for chemical coupling of ligands, nanoparticles or drugs via thioether binding and production method thereof

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
KR102437522B1 (ko) 2012-05-25 2022-08-26 셀렉티스 면역요법을 위한 동종이형 및 면역억제제 저항성인 ｔ 세포의 조작 방법
WO2014191128A1 (en) 2013-05-29 2014-12-04 Cellectis Methods for engineering t cells for immunotherapy by using rna-guided cas nuclease system
CN104592364B (zh) * 2013-10-30 2018-05-01 北京大学 定点突变和定点修饰的腺相关病毒、其制备方法及应用
US10179176B2 (en) 2016-02-16 2019-01-15 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
WO2018035503A1 (en) * 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105408486A (zh) * 2013-05-21 2016-03-16 佛罗里达大学研究基金会有限公司 衣壳修饰的raav3载体组合物以及在人肝癌基因治疗中的用途
WO2017189963A1 (en) * 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN109476707A (zh) * 2016-05-13 2019-03-15 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
WO2019006043A1 (en) * 2017-06-27 2019-01-03 Regeneron Pharmaceuticals, Inc. RECOMBINANT VIRAL VECTORS WITH MODIFIED TROPISM AND USES THEREOF FOR TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS
EP3461836A1 (en) * 2017-09-28 2019-04-03 Universität zu Köln Mutated adeno-associated viral capsid proteins for chemical coupling of ligands, nanoparticles or drugs via thioether binding and production method thereof

Non-Patent Citations (9)

* Cited by examiner, † Cited by third party
Title
DHRUVA KATREKAR等: "Oligonucleotide conjugated multi-functional adenoassociated viruses", SCIENTIFIC REPORTS, vol. 8, no. 1, pages 3589 *
MATTHEW D STACHLER等: "Site-specific Modification of AAV Vector Particles With Biophysical Probes and Targeting Ligands Using Biotin Ligase", MOLECULAR THERAPY, vol. 16, no. 8, pages 1467 - 1473, XP055187899, DOI: 10.1038/mt.2008.129 *
MATTSON G等: "A practical approach to crosslinking", MOLECULAR BIOLOGY REPORTS, vol. 17, 1 April 1993 (1993-04-01), pages 167 - 183, XP009033258, DOI: 10.1007/BF00986726 *
PEI WU等: "Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism", JOURNAL OF VIROLOGY, vol. 74, no. 18, pages 8635 - 8647 *
RACHEL E KELEMEN等: "A Precise Chemical Strategy To Alter the Receptor Specificity of the Adeno-Associated Virus", ANGEWANDTE CHEMIE INTERNATIONAL EDITION, vol. 55, no. 36, pages 10645 - 10649, XP055623118 *
XIANGQUN SHI等: "Insertional Mutagenesis at Positions 520 and 584 of AdenoAssociated Virus Type 2 (AAV2) Capsid Gene and Generation of AAV2 Vectors with Eliminated Heparin- Binding Ability and Introduced Novel Tropism", HUMAN GENE THERAPY, vol. 17, no. 3, pages 353 - 361, XP055507574 *
YAO T等: "Site-Specific PEGylated Adeno-Associated Viruses with Increased Serum Stability and Reduced ImmunogenicityYao T, Zhou X, Zhang C, et al. Site-specific PEGylated adeno-associated viruses with increased serum stability and reduced immunogenicity[J]. Molecules, 2017, 22(7): 1155.", MOLECULES, vol. 22, no. 7, pages 1155 *
YARONG LIU等: "Site-Specific Modification of Adeno-Associated Viruses via a Genetically Engineered Aldehyde Tag", SMALL, vol. 9, no. 3, pages 421 - 429, XP055421753, DOI: 10.1002/smll.201201661 *
ZHANG C等: "Development of next generation adeno-associated viral vectors capable of selective tropism and efficient gene delivery", BIOMATERIALS, vol. 80, pages 134 - 145, XP055727141, DOI: 10.1016/j.biomaterials.2015.11.066 *

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WO2020225363A2 (en) 2020-11-12
AU2020268759A1 (en) 2021-12-16
IL287614A (en) 2021-12-01
SG11202112029XA (en) 2021-11-29
WO2020225363A3 (en) 2020-12-17
MX2021013619A (es) 2022-02-22
EP3966319A2 (en) 2022-03-16
CA3139183A1 (en) 2020-11-12
JP7737312B2 (ja) 2025-09-10
JP2022531892A (ja) 2022-07-12
JP2025121993A (ja) 2025-08-20
US20220288234A1 (en) 2022-09-15
KR20220008863A (ko) 2022-01-21
EP3736330A1 (en) 2020-11-11

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