KR20210126680A - 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법 - Google Patents

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

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KR20210126680A
KR20210126680A KR1020217029279A KR20217029279A KR20210126680A KR 20210126680 A KR20210126680 A KR 20210126680A KR 1020217029279 A KR1020217029279 A KR 1020217029279A KR 20217029279 A KR20217029279 A KR 20217029279A KR 20210126680 A KR20210126680 A KR 20210126680A
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cas9
adenosine deaminase
domain
amino acid
variant
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니콜 가우델리
마이클 페커
베른트 제츠체
이안 슬레이메이커
이 유
데이비드 에이. 본
승주 이
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빔 테라퓨틱스, 인크.
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KR1020217029279A 2019-02-13 2020-02-13 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법 Pending KR20210126680A (ko)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US201962805238P 2019-02-13 2019-02-13
US62/805,238 2019-02-13
US62/805,271 2019-02-13
US201962852224P 2019-05-23 2019-05-23
US201962852228P 2019-05-23 2019-05-23
US62/852,224 2019-05-23
US62/852,228 2019-05-23
US201962931722P 2019-11-06 2019-11-06
US62/931,722 2019-11-06
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
US202062966526P 2020-01-27 2020-01-27
US62/966,526 2020-01-27
PCT/US2020/018195 WO2020168135A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating alpha-1 antitrypsin deficiency

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KR20210126680A true KR20210126680A (ko) 2021-10-20

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US (1) US20230101597A1 (https=)
EP (1) EP3923994A4 (https=)
JP (2) JP7646554B2 (https=)
KR (1) KR20210126680A (https=)
CN (2) CN120138046A (https=)
AU (1) AU2020223314A1 (https=)
CA (1) CA3128878A1 (https=)
WO (1) WO2020168135A1 (https=)

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AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
AU2020215730A1 (en) 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors
JP7693552B2 (ja) * 2019-02-13 2025-06-17 ビーム セラピューティクス インク． アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法
CN120174005A (zh) 2019-02-13 2025-06-20 比姆医疗股份有限公司 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
WO2021189110A1 (en) * 2020-03-25 2021-09-30 University Of Tasmania Dna altering proteins and uses therefor
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CA3198671A1 (en) * 2020-10-14 2022-04-21 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
CN112553246A (zh) * 2020-12-08 2021-03-26 安徽省农业科学院水稻研究所 一种基于CRISPR-SaCas9系统的高效基因组编辑载体及其应用
WO2022150372A1 (en) * 2021-01-05 2022-07-14 Horizon Discovery Ltd. Guide rna designs and complexes for type v cas systems
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