CN118581121A - 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体 - Google Patents
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| US201762509616P | 2017-05-22 | 2017-05-22 | |
| US62/509,616 | 2017-05-22 | ||
| CN201880042428.9A CN110945127B (zh) | 2017-05-22 | 2018-05-22 | 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体 |
| PCT/US2018/033866 WO2018217731A1 (en) | 2017-05-22 | 2018-05-22 | Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia b |
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| CN201880042428.9A Active CN110945127B (zh) | 2017-05-22 | 2018-05-22 | 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体 |
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| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| 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 |
| EP3867380A2 (en) | 2018-10-18 | 2021-08-25 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor ix |
| TWI851647B (zh) * | 2019-01-16 | 2024-08-11 | 日商武田藥品工業股份有限公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體 |
| US11596671B2 (en) * | 2019-02-01 | 2023-03-07 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia B using viral vectors encoding recombinant fix variants with increased expression |
| KR20210141608A (ko) * | 2019-03-19 | 2021-11-23 | 체에스엘 베링 렝나우 아게 | 치료요법에서 인자 ix 변이체 및 이의 용도 |
| CN114207135A (zh) * | 2019-06-11 | 2022-03-18 | 夏尔人类遗传性治疗公司 | 用于治疗由失调的血浆激肽释放酶介导的疾病的抗体的腺相关病毒载体递送 |
| EP3986481A2 (en) | 2019-06-20 | 2022-04-27 | Takeda Pharmaceutical Company Limited | Method of treatment with viral-based gene therapy |
| CN110684798A (zh) * | 2019-09-03 | 2020-01-14 | 深圳新诺微环生物科技有限公司 | 肌肉靶向的微环dna基因治疗 |
| PE20231100A1 (es) * | 2020-01-16 | 2023-07-18 | Takeda Pharmaceuticals Co | Terapia genica basada en virus adenoasociados para la fenilcetonuria |
| TW202246505A (zh) * | 2021-03-05 | 2022-12-01 | 俄羅斯聯邦商亞那拜恩有限公司 | 編碼凝血因子ｉｘ蛋白的密碼子優化的核酸及其用途 |
| US20240287490A1 (en) * | 2021-06-23 | 2024-08-29 | Inspirar Limited | Composition and method for treating hemophilia b |
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