CN113438954A - 可用于治疗gm1神经节苷脂病的组合物 - Google Patents

可用于治疗gm1神经节苷脂病的组合物 Download PDF

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Publication number
CN113438954A
CN113438954A CN201980079543.8A CN201980079543A CN113438954A CN 113438954 A CN113438954 A CN 113438954A CN 201980079543 A CN201980079543 A CN 201980079543A CN 113438954 A CN113438954 A CN 113438954A
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aav
seq
glb1
gangliosidosis
certain embodiments
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CN201980079543.8A
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Chinese (zh)
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J·M·威尔逊
C·欣德勒
N·卡茨
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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CN201980079543.8A 2018-10-01 2019-09-30 可用于治疗gm1神经节苷脂病的组合物 Pending CN113438954A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862739811P 2018-10-01 2018-10-01
US62/739811 2018-10-01
US201962835178P 2019-04-17 2019-04-17
US62/835178 2019-04-17
PCT/US2019/053797 WO2020072354A1 (en) 2018-10-01 2019-09-30 Compositions useful for treating gm1 gangliosidosis

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CN113438954A true CN113438954A (zh) 2021-09-24

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US (1) US12594327B2 (https=)
EP (1) EP3860659A1 (https=)
JP (2) JP7534290B2 (https=)
KR (1) KR20210071017A (https=)
CN (1) CN113438954A (https=)
AU (1) AU2019351815A1 (https=)
BR (1) BR112021006060A2 (https=)
CA (1) CA3114175A1 (https=)
CL (1) CL2021000798A1 (https=)
CO (1) CO2021005443A2 (https=)
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MX (1) MX2021003869A (https=)
PE (1) PE20211596A1 (https=)
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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
WO2023184688A1 (zh) * 2022-03-31 2023-10-05 北京锦篮基因科技有限公司 功能性β-半乳糖苷酶变体、AAV介导的人β-半乳糖苷酶表达载体及其用途

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CN113438954A (zh) 2018-10-01 2021-09-24 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂病的组合物
MX2022009462A (es) * 2020-02-02 2022-11-10 Univ Pennsylvania Composiciones útiles para tratar la gangliosidosis gm1.
WO2024040258A2 (en) * 2022-08-19 2024-02-22 Codexis, Inc. Engineered beta- galactosidase polypeptides
CN120035662A (zh) * 2022-10-12 2025-05-23 新型医药公司 用于治疗、预防或改善GM1神经节苷脂沉积症或Morquio综合征B的药物组合物及其施用方法

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2023184688A1 (zh) * 2022-03-31 2023-10-05 北京锦篮基因科技有限公司 功能性β-半乳糖苷酶变体、AAV介导的人β-半乳糖苷酶表达载体及其用途

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SA521421639B1 (ar) 2025-04-21
JP2022512589A (ja) 2022-02-07
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