JP7066861B2 - Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 - Google Patents

Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 Download PDF

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JP7066861B2
JP7066861B2 JP2020541862A JP2020541862A JP7066861B2 JP 7066861 B2 JP7066861 B2 JP 7066861B2 JP 2020541862 A JP2020541862 A JP 2020541862A JP 2020541862 A JP2020541862 A JP 2020541862A JP 7066861 B2 JP7066861 B2 JP 7066861B2
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amino acid
capsid protein
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sequence
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JP2021512601A5 (enExample
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アルバート・バーンズ・シーモア
シーミン・セヘル・アーメド
ジェーソン・ボーク・ライト
セリーナ・ニコル・ドリヴ
ヒラード・ルビン
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ホモロジー・メディシンズ・インコーポレイテッド
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JP2020541862A 2018-02-01 2019-02-01 Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 Expired - Fee Related JP7066861B2 (ja)

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US201862625150P 2018-02-01 2018-02-01
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PCT/US2019/016351 WO2019152841A1 (en) 2018-02-01 2019-02-01 Adeno-associated virus compositions for pah gene transfer and methods of use thereof

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JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
WO2019152841A1 (en) * 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof
KR20210008501A (ko) 2018-05-09 2021-01-22 바이오마린 파머수티컬 인크. 페닐케톤뇨증을 치료하는 방법
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
PE20220233A1 (es) * 2019-06-10 2022-02-07 Homology Medicines Inc Composiciones de virus adenoasociados para la transferencia del gen de arsa y metodos de uso de las mismas
TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
WO2021144649A2 (en) * 2020-01-16 2021-07-22 Takeda Pharmaceutical Company Limited Adeno associated virus based gene therapy for phenylketonuria
CN115461066B (zh) * 2020-04-28 2026-01-06 美国辉瑞有限公司 用于基因疗法的编码天冬氨酸酰化酶(aspa)的经修饰的核酸和载体
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
EP4211252A1 (en) * 2020-09-09 2023-07-19 Homology Medicines, Inc. Vectorized antibodies and uses thereof
KR20230079172A (ko) 2020-10-01 2023-06-05 젠자임 코포레이션 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트
CN115197967B (zh) * 2021-04-08 2023-09-15 广州派真生物技术有限公司 制备重组腺相关病毒的辅助质粒及其应用
US20230055381A1 (en) * 2021-06-25 2023-02-23 Oxford Biomedica Solutions Llc Adeno-associated virus packaging systems
US20230090654A1 (en) * 2021-08-24 2023-03-23 Homology Medicines, Inc. Adeno-associated virus formulations
WO2023077085A2 (en) * 2021-10-29 2023-05-04 Oxford Biomedica Solutions Llc Methods and compositions for the purification of adeno-associated virus
CN116656740A (zh) * 2023-05-30 2023-08-29 浙江大学 一种腺相关病毒基因治疗载体及其在制备治疗苯丙酮尿症的药物中的应用
CN119876192B (zh) * 2024-07-02 2025-11-07 浙江大学滨江研究院 用于治疗苯丙酮尿症的多核苷酸

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