CN110650733A - 经过修饰的aav衣壳和其用途 - Google Patents

经过修饰的aav衣壳和其用途 Download PDF

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Publication number
CN110650733A
CN110650733A CN201880025324.7A CN201880025324A CN110650733A CN 110650733 A CN110650733 A CN 110650733A CN 201880025324 A CN201880025324 A CN 201880025324A CN 110650733 A CN110650733 A CN 110650733A
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aav
capsid protein
amino acid
viral vector
cells
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安娜希塔·喀拉瓦拉
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A Dev Ram Biotechnology Ltd By Share Ltd
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C07—ORGANIC CHEMISTRY
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
CN201880025324.7A 2017-02-28 2018-02-28 经过修饰的aav衣壳和其用途 Pending CN110650733A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762464878P 2017-02-28 2017-02-28
US62/464,878 2017-02-28
PCT/US2018/020215 WO2018160686A1 (en) 2017-02-28 2018-02-28 Modified aav capsids and uses thereof

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CN110650733A true CN110650733A (zh) 2020-01-03

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US (1) US20210130413A1 (enExample)
EP (1) EP3589277A4 (enExample)
JP (1) JP2020510424A (enExample)
CN (1) CN110650733A (enExample)
AU (1) AU2018227483A1 (enExample)
CA (1) CA3054687A1 (enExample)
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Cited By (6)

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CN113121652A (zh) * 2021-04-19 2021-07-16 上海信致医药科技有限公司 视网膜和肌肉高亲和性腺相关病毒衣壳蛋白及相关应用
CN113564187A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 基于aav的抗血管生成基因递送系统及其用途
CN114340683A (zh) * 2019-04-15 2022-04-12 衣阿华大学研究基金会 用于转基因表达的方法和组合物
CN116096377A (zh) * 2020-05-05 2023-05-09 杜克大学 跨物种相容的腺相关病毒组合物及其使用方法
CN116121274A (zh) * 2020-07-29 2023-05-16 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1

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CN106414474B (zh) 2014-03-17 2021-01-15 阿德夫拉姆生物技术股份有限公司 用于视锥细胞中增强的基因表达的组合物和方法
CA2978068C (en) 2015-03-02 2024-05-28 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
MX2019011040A (es) 2017-03-17 2020-01-20 Adverum Biotechnologies Inc Composiciones y metodos para potenciar la expresion genica.
EP3697449A1 (en) * 2017-10-18 2020-08-26 REGENXBIO Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified vegf-trap
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CN113966399A (zh) * 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
EP3934698A1 (en) * 2019-03-04 2022-01-12 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
TW202102526A (zh) * 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
CA3137284A1 (en) * 2019-04-24 2020-10-29 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
TW202532428A (zh) * 2019-04-26 2025-08-16 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼
MX2023002695A (es) * 2020-09-03 2023-05-19 Univ Massachusetts Virus adeno-asociado para la administración de kh902 (conbercept) y usos del mismo.
US20230391864A1 (en) * 2020-10-28 2023-12-07 Regenxbio, Inc. Vectorized anti-tnf-alpha antibodies for ocular indications
CN114686448A (zh) * 2022-03-31 2022-07-01 上海勉亦生物科技有限公司 具有肝脏特异靶向性的纯化的腺相关病毒及其应用
CN112961220B (zh) * 2021-04-19 2023-11-17 信念医药科技(上海)有限公司 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体
TW202313096A (zh) 2021-05-28 2023-04-01 大陸商江蘇恆瑞醫藥股份有限公司 衣殼變異的重組腺相關病毒及其應用
CN117230117A (zh) * 2023-08-04 2023-12-15 中国科学院深圳先进技术研究院 重组腺相关病毒包装质粒、突变体及其制备方法和应用
CN117143920A (zh) * 2023-08-30 2023-12-01 呈诺再生医学科技(北京)有限公司 一种治疗新生血管性眼部疾病的基因药物
CN117368466B (zh) * 2023-12-08 2024-03-12 安徽惠邦生物工程有限公司 用生化分析仪测定尿结合珠蛋白的试剂盒及其制备方法
WO2025214359A1 (en) * 2024-04-09 2025-10-16 Hangzhou Jiayin Biotech Ltd. A novel aav variant

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Cited By (7)

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Publication number Priority date Publication date Assignee Title
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
CN114340683A (zh) * 2019-04-15 2022-04-12 衣阿华大学研究基金会 用于转基因表达的方法和组合物
CN116096377A (zh) * 2020-05-05 2023-05-09 杜克大学 跨物种相容的腺相关病毒组合物及其使用方法
CN116121274A (zh) * 2020-07-29 2023-05-16 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
CN113121652A (zh) * 2021-04-19 2021-07-16 上海信致医药科技有限公司 视网膜和肌肉高亲和性腺相关病毒衣壳蛋白及相关应用
CN113121652B (zh) * 2021-04-19 2022-10-11 上海信致医药科技有限公司 视网膜和肌肉高亲和性腺相关病毒衣壳蛋白及相关应用
CN113564187A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 基于aav的抗血管生成基因递送系统及其用途

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CA3054687A1 (en) 2018-09-07
AU2018227483A1 (en) 2019-09-12
US20210130413A1 (en) 2021-05-06
EP3589277A1 (en) 2020-01-08
EP3589277A4 (en) 2020-08-26
WO2018160686A1 (en) 2018-09-07
JP2020510424A (ja) 2020-04-09

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