CN113195721A - 治疗α-1抗胰蛋白酶缺乏症的组合物和方法 - Google Patents

治疗α-1抗胰蛋白酶缺乏症的组合物和方法 Download PDF

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CN113195721A
CN113195721A CN201980082770.6A CN201980082770A CN113195721A CN 113195721 A CN113195721 A CN 113195721A CN 201980082770 A CN201980082770 A CN 201980082770A CN 113195721 A CN113195721 A CN 113195721A
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sequence
aat
rna
nucleic acid
seq
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J·D·芬恩
H-R·黄
A·福尔热
X·解
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Intelia Therapeutics Co ltd
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Intelia Therapeutics Co ltd
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CN201980082770.6A 2018-10-18 2019-10-18 治疗α-1抗胰蛋白酶缺乏症的组合物和方法 Pending CN113195721A (zh)

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US201862747522P 2018-10-18 2018-10-18
US62/747,522 2018-10-18
PCT/US2019/057092 WO2020082047A1 (en) 2018-10-18 2019-10-18 Compositions and methods for treating alpha-1 antitrypsin deficiencey

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CN116438306A (zh) * 2020-10-20 2023-07-14 St制药株式会社 用于5'-加帽的rna合成的寡核苷酸

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JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス,インコーポレーテッド 第ix因子を発現するための組成物及び方法
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WO2023064918A1 (en) * 2021-10-15 2023-04-20 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
EP4419111A1 (en) * 2021-10-19 2024-08-28 Precision Biosciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
WO2024254519A2 (en) * 2023-06-08 2024-12-12 Mammoth Biosciences, Inc. Compositions and methods for the modification of human genes expressed by liver cells
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

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Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110382697A (zh) * 2016-12-22 2019-10-25 因特利亚治疗公司 用于治疗α-1抗胰蛋白酶缺乏症的组合物及方法
CN110382697B (zh) * 2016-12-22 2023-12-29 因特利亚治疗公司 用于治疗α-1抗胰蛋白酶缺乏症的组合物及方法
CN116438306A (zh) * 2020-10-20 2023-07-14 St制药株式会社 用于5'-加帽的rna合成的寡核苷酸

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EP3867378A1 (en) 2021-08-25
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TW202027797A (zh) 2020-08-01
BR112021007289A2 (pt) 2021-07-27
MX2021004276A (es) 2021-09-08
US20200270618A1 (en) 2020-08-27
JP2022505381A (ja) 2022-01-14
IL282237A (en) 2021-05-31
CO2021006367A2 (es) 2021-08-19
PH12021550842A1 (en) 2021-12-06
WO2020082047A1 (en) 2020-04-23
AU2019361204A1 (en) 2021-05-27
WO2020082047A8 (en) 2024-11-07
EA202191067A1 (ru) 2021-11-17

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