CN114026115A - 用于溶酶体病症的基因疗法 - Google Patents

用于溶酶体病症的基因疗法 Download PDF

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Publication number
CN114026115A
CN114026115A CN202080041473.XA CN202080041473A CN114026115A CN 114026115 A CN114026115 A CN 114026115A CN 202080041473 A CN202080041473 A CN 202080041473A CN 114026115 A CN114026115 A CN 114026115A
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raav
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nucleic acid
gcase
seq
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Chinese (zh)
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赫维·莱茵
阿萨·阿贝利奥维奇
劳拉·赫克曼
弗兰兹·赫夫蒂
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Privelle Therapeutics
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Privelle Therapeutics
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CN202080041473.XA 2019-04-10 2020-04-10 用于溶酶体病症的基因疗法 Pending CN114026115A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962831840P 2019-04-10 2019-04-10
US62/831,840 2019-04-10
US202062990246P 2020-03-16 2020-03-16
US62/990,246 2020-03-16
PCT/US2020/027788 WO2020210713A1 (en) 2019-04-10 2020-04-10 Gene therapies for lysosomal disorders

Publications (1)

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CN114026115A true CN114026115A (zh) 2022-02-08

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US (2) US11903985B2 (https=)
EP (1) EP3953378A1 (https=)
JP (1) JP7664848B2 (https=)
KR (1) KR20220006527A (https=)
CN (1) CN114026115A (https=)
AU (1) AU2020270984B2 (https=)
BR (1) BR112021019880A2 (https=)
CA (1) CA3136117A1 (https=)
IL (1) IL286901A (https=)
MX (1) MX2021012184A (https=)
WO (1) WO2020210713A1 (https=)

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CN117018231A (zh) * 2023-08-16 2023-11-10 科辉智药(深圳)新药研究中心有限公司 用于治疗神经病变的基因疗法及其应用
WO2025039622A1 (zh) * 2023-08-23 2025-02-27 上海金珂博生物技术有限公司 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途

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PL3953377T3 (pl) 2019-04-10 2026-02-16 Prevail Therapeutics, Inc. Terapie genowe zaburzeń lizosomalnych
IL300407A (en) 2020-08-10 2023-04-01 Prevail Therapeutics Inc Gene therapies for lysosomal disorders
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MX2024009003A (es) * 2022-01-21 2024-09-17 Astrazeneca Ireland Ltd Terapia genica para la enfermedad de gaucher.
WO2023202637A1 (en) 2022-04-19 2023-10-26 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating neurodegenerative disorders
WO2025166048A1 (en) * 2024-01-31 2025-08-07 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic gba1 genes
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