TW202020156A - 用於治療原發性高草酸尿症第1型(ph1)之羥酸氧化酶1(hao1)基因編輯之組合物及方法 - Google Patents

用於治療原發性高草酸尿症第1型(ph1)之羥酸氧化酶1(hao1)基因編輯之組合物及方法 Download PDF

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TW202020156A
TW202020156A TW108127073A TW108127073A TW202020156A TW 202020156 A TW202020156 A TW 202020156A TW 108127073 A TW108127073 A TW 108127073A TW 108127073 A TW108127073 A TW 108127073A TW 202020156 A TW202020156 A TW 202020156A
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composition
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sequence selected
sequence
guide
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TW108127073A
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柴克瑞 威廉 岱美克
書布 歐德
布萊德利 安德魯 莫雷
雷納德 麥克 雷斯卡里
安妮特 胡伯納
瓦特 史瑞普斯
莎拉 貝絲 海斯
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美商英特利亞醫療公司
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TW108127073A 2018-07-31 2019-07-30 用於治療原發性高草酸尿症第1型(ph1)之羥酸氧化酶1(hao1)基因編輯之組合物及方法 TW202020156A (zh)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201862712904P 2018-07-31 2018-07-31
US62/712,904 2018-07-31
US201862738936P 2018-09-28 2018-09-28
US62/738,936 2018-09-28
US201962834328P 2019-04-15 2019-04-15
US62/834,328 2019-04-15
US201962841734P 2019-05-01 2019-05-01
US62/841,734 2019-05-01

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TW202020156A true TW202020156A (zh) 2020-06-01

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US (2) US20210163943A1 (enExample)
EP (1) EP3830267A1 (enExample)
JP (1) JP2021531804A (enExample)
KR (1) KR20210053888A (enExample)
CN (1) CN112867795A (enExample)
AU (1) AU2019313348A1 (enExample)
BR (1) BR112021001546A2 (enExample)
CA (1) CA3113190A1 (enExample)
CO (1) CO2021002691A2 (enExample)
MX (1) MX2021001070A (enExample)
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WO (1) WO2020028327A1 (enExample)

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IL303506A (en) 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
US20230034581A1 (en) 2021-06-04 2023-02-02 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof
IL308803A (en) * 2021-06-04 2024-01-01 Arbor Biotechnologies Inc Gene editing systems comprising guide RNA targeting hydroxy acid oxidase 1 (HAO1) and uses thereof
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
CN118086297B (zh) * 2024-02-28 2025-10-03 复旦大学附属儿科医院 用于人HAO1基因编辑的sgRNA、组合物及其应用
CN117925623B (zh) * 2024-03-20 2024-06-07 北京引正基因科技有限公司 用于hao1基因编辑和治疗ph1的组合物

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CA3113190A1 (en) 2020-02-06
CO2021002691A2 (es) 2021-04-08
EP3830267A1 (en) 2021-06-09
JP2021531804A (ja) 2021-11-25
KR20210053888A (ko) 2021-05-12
BR112021001546A2 (pt) 2021-05-04
US20260043026A1 (en) 2026-02-12
CN112867795A (zh) 2021-05-28
AU2019313348A1 (en) 2021-03-04
US20210163943A1 (en) 2021-06-03
WO2020028327A1 (en) 2020-02-06
MX2021001070A (es) 2021-05-27

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