TWI841908B - 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 - Google Patents
腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 Download PDFInfo
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| RU2020126034A (ru) * | 2015-09-24 | 2020-09-02 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| EP3368664A4 (en) | 2015-10-28 | 2019-08-28 | Sangamo Therapeutics, Inc. | LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF |
| US11584780B2 (en) | 2016-07-26 | 2023-02-21 | Biomarin Pharmaceutical Inc. | Adeno-associated virus capsid proteins |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| PL3652326T3 (pl) | 2017-07-10 | 2025-02-24 | Uniqure Ip B.V. | Środki i sposoby terapii genowej aav u ludzi |
| MY208862A (en) * | 2017-08-01 | 2025-06-04 | Spark Therapeutics Inc | Factor viii (fviii) gene therapy methods |
| US12296026B2 (en) * | 2018-02-05 | 2025-05-13 | Astellas Gene Therapies, Inc. | Transcription regulatory elements and uses thereof |
| EP3810647A4 (en) * | 2018-04-26 | 2022-08-17 | The University of North Carolina at Chapel Hill | METHODS AND COMPOSITIONS FOR THE TREATMENT OF HEMOPHILIA |
| WO2019217513A2 (en) | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| KR20210008491A (ko) * | 2018-05-14 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 청소년 대상체에서 aav 벡터의 안정한 발현 |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| US12091675B2 (en) * | 2018-07-16 | 2024-09-17 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant FVIII variants with increased expression |
| MX2021001375A (es) * | 2018-08-03 | 2021-04-19 | Sangamo Therapeutics Inc | Parametros clinicos mejorados por la expresion del factor viii. |
| WO2020041773A1 (en) * | 2018-08-24 | 2020-02-27 | Spark Therapeutics, Inc. | Optimized promoter sequences, intron-free expression constructs and methods of use |
| AU2019346655B2 (en) | 2018-09-28 | 2025-03-13 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| MA54951A (fr) | 2019-02-15 | 2021-12-22 | Bayer Healthcare Llc | Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée |
| MA55313A (fr) | 2019-03-13 | 2022-01-19 | Generation Bio Co | Vecteurs d'adn non viraux et leurs utilisations pour exprimer des agents thérapeutiques du facteur viii |
| JP2022534798A (ja) * | 2019-06-05 | 2022-08-03 | オーチャード セラピューティクス（ヨーロッパ）リミテッド | 真核細胞を修飾するための組成物及び方法 |
| US20200405883A1 (en) * | 2019-06-20 | 2020-12-31 | Baxalta Incorporated | Method of treatment with viral-based gene therapy |
| WO2021084277A2 (en) | 2019-11-01 | 2021-05-06 | Freeline Therapeutics Limited | Transcription regulatory elements |
| US20220403005A1 (en) * | 2019-12-06 | 2022-12-22 | The Children's Hospital Of Philadelphia | Compositions and methods for modulating factor viii function |
| EP4073106A2 (en) * | 2019-12-12 | 2022-10-19 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| KR102167829B1 (ko) * | 2020-02-10 | 2020-10-20 | 주식회사 이노테라피 | 아데노연관바이러스용 안정화제 및 이를 이용한 아데노연관바이러스의 안정화 방법 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| MX2023005041A (es) | 2020-11-02 | 2023-05-17 | Biomarin Pharm Inc | Proceso para enriquecer virus adenoasociado. |
| CN115554418B (zh) * | 2022-11-22 | 2023-04-14 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| WO2025045082A1 (zh) * | 2023-09-01 | 2025-03-06 | 康霖生物科技（杭州）有限公司 | 一种缓冲系统及其相应液体制剂及其用途 |
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| US9061072B2 (en) * | 2010-07-14 | 2015-06-23 | Hanmi Science Co., Ltd | Liquid formulation of long-acting human growth hormone conjugate |
| US20150071883A1 (en) * | 2013-09-12 | 2015-03-12 | Biomarin Pharmaceutical Inc. | Adeno-Associated Virus Factor VIII Vectors |
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