TW202332767A - 用於治療龐貝氏症之抗ＴｆＲ：ＧＡＡ及抗ＣＤ６３：ＧＡＡ插入 - Google Patents

用於治療龐貝氏症之抗ＴｆＲ：ＧＡＡ及抗ＣＤ６３：ＧＡＡ插入 Download PDF

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TW202332767A
TW202332767A TW112103659A TW112103659A TW202332767A TW 202332767 A TW202332767 A TW 202332767A TW 112103659 A TW112103659 A TW 112103659A TW 112103659 A TW112103659 A TW 112103659A TW 202332767 A TW202332767 A TW 202332767A
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seq
set forth
sequence set
amino acid
composition
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TW112103659A
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安德魯 拜克
瑪莉亞 佩格提斯
凱薩琳 希格納
利亞 沙賓
寶拉米 沙邁
伊凡吉羅 佩發尼斯
菲利普 卡拉法提
妮可 基汀
帕斯卡林 艾米威森
約翰 杜根
敏 高
羅伯特 鮑伯
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美商雷傑納榮製藥公司
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Publication of TW202332767A publication Critical patent/TW202332767A/zh

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TW112103659A 2022-02-02 2023-02-02 用於治療龐貝氏症之抗ＴｆＲ：ＧＡＡ及抗ＣＤ６３：ＧＡＡ插入 TW202332767A (zh)

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US202263306040P 2022-02-02 2022-02-02
US63/306,040 2022-02-02
US202263369902P 2022-07-29 2022-07-29
US63/369,902 2022-07-29

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CN120659627A (zh) * 2022-07-29 2025-09-16 瑞泽恩制药公司 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法
IL326018A (en) * 2023-07-28 2026-03-01 Regeneron Pharma Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease
WO2025064507A1 (en) * 2023-09-19 2025-03-27 Poseida Therapeutics, Inc. Compositions and methods for integration of viral vectors
WO2025160324A2 (en) * 2024-01-26 2025-07-31 Regeneron Pharmaceuticals, Inc. Methods and compositions for using plasma cell depleting agents and/or b cell depleting agents to suppress host anti-aav antibody response and enable aav transduction and re-dosing
US20250276092A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025265017A1 (en) * 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i
WO2026006390A2 (en) * 2024-06-26 2026-01-02 Intellia Therapeutics, Inc. Modified guide rnas for genome editing

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