KR102551664B1 - 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법 - Google Patents

알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법 Download PDF

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KR102551664B1
KR102551664B1 KR1020197020995A KR20197020995A KR102551664B1 KR 102551664 B1 KR102551664 B1 KR 102551664B1 KR 1020197020995 A KR1020197020995 A KR 1020197020995A KR 20197020995 A KR20197020995 A KR 20197020995A KR 102551664 B1 KR102551664 B1 KR 102551664B1
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쇼부 오다테
왈터 스트랩스
레이날드 미카엘 레스카뷰
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인텔리아 테라퓨틱스, 인크.
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KR1020197020995A 2016-12-22 2017-12-21 알파-1 항트립신 결핍을 치료하기 위한 조성물 및 방법 Active KR102551664B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662438219P 2016-12-22 2016-12-22
US62/438,219 2016-12-22
PCT/US2017/067800 WO2018119182A1 (en) 2016-12-22 2017-12-21 Compositions and methods for treating alpha-1 antitrypsin deficiency

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KR20190100943A KR20190100943A (ko) 2019-08-29
KR102551664B1 true KR102551664B1 (ko) 2023-07-05

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US (3) US11549107B2 (enExample)
EP (1) EP3559232A1 (enExample)
JP (2) JP7383478B2 (enExample)
KR (1) KR102551664B1 (enExample)
CN (2) CN117737062A (enExample)
AU (2) AU2017379073B2 (enExample)
BR (1) BR112019012825A2 (enExample)
CA (1) CA3047415A1 (enExample)
CO (1) CO2019007776A2 (enExample)
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SA (1) SA519402192B1 (enExample)
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TW (2) TW202509233A (enExample)
WO (1) WO2018119182A1 (enExample)
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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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BR112021007343A2 (pt) * 2018-10-18 2021-08-03 Intellia Therapeutics, Inc. composições e métodos para expressão de transgene a partir de um lócus de albumina
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
WO2020168135A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
US12594301B2 (en) 2019-09-27 2026-04-07 Beam Therapeutics Inc. Compositions and methods for treatment of liquid cancers
WO2022060707A1 (en) * 2020-09-15 2022-03-24 Rutgers, The State University Of New Jersey Systems for gene editing and methods of use thereof
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20220160847A1 (en) * 2020-11-20 2022-05-26 Mark Egly Method of preventing and/or treating a plurality of diseases
EP4259792A1 (en) 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination
AU2022216614B2 (en) 2021-02-05 2026-03-05 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
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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