JP2025037975A5 - - Google Patents

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Publication number
JP2025037975A5
JP2025037975A5 JP2024208066A JP2024208066A JP2025037975A5 JP 2025037975 A5 JP2025037975 A5 JP 2025037975A5 JP 2024208066 A JP2024208066 A JP 2024208066A JP 2024208066 A JP2024208066 A JP 2024208066A JP 2025037975 A5 JP2025037975 A5 JP 2025037975A5
Authority
JP
Japan
Prior art keywords
amino acid
acid sequence
adenosine deaminase
alpha
base editor
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024208066A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025037975A (ja
Filing date
Publication date
Priority claimed from JP2021546890A external-priority patent/JP7646554B2/ja
Application filed filed Critical
Publication of JP2025037975A publication Critical patent/JP2025037975A/ja
Publication of JP2025037975A5 publication Critical patent/JP2025037975A5/ja
Pending legal-status Critical Current

Links

JP2024208066A 2019-02-13 2024-11-29 アルファ-1アンチトリプシン不全を治療するための組成物および方法 Pending JP2025037975A (ja)

Applications Claiming Priority (16)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US201962805238P 2019-02-13 2019-02-13
US62/805,271 2019-02-13
US62/805,238 2019-02-13
US201962852224P 2019-05-23 2019-05-23
US201962852228P 2019-05-23 2019-05-23
US62/852,228 2019-05-23
US62/852,224 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
JP2021546890A JP7646554B2 (ja) 2019-02-13 2020-02-13 アルファ-1アンチトリプシン不全を治療するための組成物および方法
PCT/US2020/018195 WO2020168135A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating alpha-1 antitrypsin deficiency

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2021546890A Division JP7646554B2 (ja) 2019-02-13 2020-02-13 アルファ-1アンチトリプシン不全を治療するための組成物および方法

Publications (2)

Publication Number Publication Date
JP2025037975A JP2025037975A (ja) 2025-03-18
JP2025037975A5 true JP2025037975A5 (https=) 2025-10-14

Family

ID=72044596

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021546890A Active JP7646554B2 (ja) 2019-02-13 2020-02-13 アルファ-1アンチトリプシン不全を治療するための組成物および方法
JP2024208066A Pending JP2025037975A (ja) 2019-02-13 2024-11-29 アルファ-1アンチトリプシン不全を治療するための組成物および方法

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2021546890A Active JP7646554B2 (ja) 2019-02-13 2020-02-13 アルファ-1アンチトリプシン不全を治療するための組成物および方法

Country Status (8)

Country Link
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=)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP4396354A4 (en) * 2021-09-26 2025-12-03 Wave Life Sciences Ltd OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES
CN114634923B (zh) * 2022-04-07 2024-02-23 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
KR20250126155A (ko) * 2022-11-04 2025-08-22 라이프 에디트 테라퓨틱스, 인크. 내부 삽입 부위를 갖는 진화된 아데닌 데아미나제 및 rna-가이드 뉴클레아제 융합 단백질 및 사용 방법
WO2025202473A1 (en) * 2024-03-28 2025-10-02 Revvity Discovery Limited A nucleic acid deaminase, a base editor and uses thereof
CN119162157B (zh) * 2024-11-14 2025-06-03 锐正基因(苏州)有限公司 用于碱基编辑的脱氨酶及其变体

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3019595A4 (en) * 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
BR112017007765B1 (pt) * 2014-10-14 2023-10-03 Halozyme, Inc Composições de adenosina deaminase-2 (ada2), variantes do mesmo e métodos de usar o mesmo
IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
US11512311B2 (en) * 2016-03-25 2022-11-29 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (A1AT) deficiency
CN110214183A (zh) * 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
KR102622411B1 (ko) * 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
US11866697B2 (en) * 2017-05-18 2024-01-09 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
KR102761791B1 (ko) * 2017-06-26 2025-02-05 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법
CA3108281A1 (en) * 2018-08-03 2020-02-06 Beam Therapeutics Inc. Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence
AU2020344547A1 (en) * 2019-09-09 2022-03-24 Beam Therapeutics Inc. Novel nucleobase editors and methods of using same

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