CN112040988A - 修饰的aav衣壳多肽用于治疗肌肉疾病 - Google Patents

修饰的aav衣壳多肽用于治疗肌肉疾病 Download PDF

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Publication number
CN112040988A
CN112040988A CN201980028398.0A CN201980028398A CN112040988A CN 112040988 A CN112040988 A CN 112040988A CN 201980028398 A CN201980028398 A CN 201980028398A CN 112040988 A CN112040988 A CN 112040988A
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muscle
aav capsid
aav
capsid polypeptide
specifically defined
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D·格里姆
J·温曼
J·埃尔安达里
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Fraunhofer-Gesellschaft zur Förderung der Angewandten Forschung eV
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Fraunhofer-Gesellschaft zur Förderung der Angewandten Forschung eV
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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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    • C07K2319/035—Fusion polypeptide containing a localisation/targetting motif containing a signal for targeting to the external surface of a cell, e.g. to the outer membrane of Gram negative bacteria, GPI- anchored eukaryote proteins
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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CN201980028398.0A 2018-04-27 2019-04-26 修饰的aav衣壳多肽用于治疗肌肉疾病 Pending CN112040988A (zh)

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EP18169822.6 2018-04-27
EP18169822 2018-04-27
PCT/EP2019/060790 WO2019207132A1 (en) 2018-04-27 2019-04-26 Modified aav capsid polypeptides for treatment of muscular diseases

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US (1) US20210363193A1 (enExample)
EP (2) EP3784288B1 (enExample)
JP (2) JP2021521833A (enExample)
CN (1) CN112040988A (enExample)
AU (2) AU2019258830C1 (enExample)
CA (1) CA3097375A1 (enExample)
WO (1) WO2019207132A1 (enExample)

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CN113480615A (zh) * 2021-07-30 2021-10-08 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113563430A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN116096431A (zh) * 2020-09-10 2023-05-09 吉尼松公司 肽修饰的aav衣壳
WO2023169115A1 (zh) * 2022-03-11 2023-09-14 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
CN118005749A (zh) * 2023-07-21 2024-05-10 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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Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116096431A (zh) * 2020-09-10 2023-05-09 吉尼松公司 肽修饰的aav衣壳
CN113480615A (zh) * 2021-07-30 2021-10-08 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113563430A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
WO2023169115A1 (zh) * 2022-03-11 2023-09-14 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
CN118005749A (zh) * 2023-07-21 2024-05-10 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN118005749B (zh) * 2023-07-21 2024-07-16 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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EP3784288B1 (en) 2026-05-27
JP7707261B2 (ja) 2025-07-14
WO2019207132A1 (en) 2019-10-31
AU2019258830B2 (en) 2025-12-18
CA3097375A1 (en) 2019-10-31
AU2026202070A1 (en) 2026-04-09
EP3784288C0 (en) 2026-05-27
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JP2024020346A (ja) 2024-02-14
US20210363193A1 (en) 2021-11-25
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