JP2025170240A5 - - Google Patents

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Publication number
JP2025170240A5
JP2025170240A5 JP2025122950A JP2025122950A JP2025170240A5 JP 2025170240 A5 JP2025170240 A5 JP 2025170240A5 JP 2025122950 A JP2025122950 A JP 2025122950A JP 2025122950 A JP2025122950 A JP 2025122950A JP 2025170240 A5 JP2025170240 A5 JP 2025170240A5
Authority
JP
Japan
Prior art keywords
adenosine deaminase
fusion protein
base editor
domain
seq
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
JP2025122950A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025170240A (ja
Filing date
Publication date
Priority claimed from PCT/US2020/018195 external-priority patent/WO2020168135A1/en
Priority claimed from JP2022514994A external-priority patent/JP7717684B2/ja
Application filed filed Critical
Publication of JP2025170240A publication Critical patent/JP2025170240A/ja
Publication of JP2025170240A5 publication Critical patent/JP2025170240A5/ja
Pending legal-status Critical Current

Links

JP2025122950A 2019-09-09 2025-07-23 新規核酸塩基エディター及びその使用方法 Pending JP2025170240A (ja)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201962897777P 2019-09-09 2019-09-09
US62/897,777 2019-09-09
USPCT/US2020/018195 2020-02-13
PCT/US2020/018195 WO2020168135A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating alpha-1 antitrypsin deficiency
JP2022514994A JP7717684B2 (ja) 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法
PCT/US2020/049975 WO2021050571A1 (en) 2019-09-09 2020-09-09 Novel nucleobase editors and methods of using same

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2022514994A Division JP7717684B2 (ja) 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法

Publications (2)

Publication Number Publication Date
JP2025170240A JP2025170240A (ja) 2025-11-18
JP2025170240A5 true JP2025170240A5 (cg-RX-API-DMAC7.html) 2026-03-09

Family

ID=74867196

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022514994A Active JP7717684B2 (ja) 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法
JP2025122950A Pending JP2025170240A (ja) 2019-09-09 2025-07-23 新規核酸塩基エディター及びその使用方法

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2022514994A Active JP7717684B2 (ja) 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法

Country Status (8)

Country Link
US (1) US20230075877A1 (cg-RX-API-DMAC7.html)
EP (1) EP4028026A4 (cg-RX-API-DMAC7.html)
JP (2) JP7717684B2 (cg-RX-API-DMAC7.html)
KR (1) KR20220076467A (cg-RX-API-DMAC7.html)
CN (1) CN114667149A (cg-RX-API-DMAC7.html)
AU (1) AU2020344547A1 (cg-RX-API-DMAC7.html)
CA (1) CA3153624A1 (cg-RX-API-DMAC7.html)
WO (1) WO2021050571A1 (cg-RX-API-DMAC7.html)

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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
KR20210126680A (ko) * 2019-02-13 2021-10-20 빔 테라퓨틱스, 인크. 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법
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
CA3128755C (en) 2019-02-13 2024-06-04 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
EP4313118A4 (en) * 2021-03-26 2025-06-18 Beam Therapeutics Inc. ADENOSINE DEAMINASE VARIANTS AND THEIR USES
WO2022240858A1 (en) 2021-05-10 2022-11-17 Mammoth Biosciences, Inc. Effector proteins and methods of use
WO2023275892A1 (en) * 2021-06-29 2023-01-05 Council Of Scientific & Industrial Research Engineered fncas9 and uses thereof
US20240352439A1 (en) * 2021-09-03 2024-10-24 The University Of Chicago Polypeptides and methods for modifying nucleic acids
CA3231679A1 (en) * 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Hbb-modulating compositions and methods
EP4409000A1 (en) 2021-09-28 2024-08-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of beta-hemoglobinopathies
WO2023099591A1 (en) 2021-12-01 2023-06-08 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for increasing fetal hemoglobin content by editing the +55-kb region of the erythroid-specific bcl11a enhancer
WO2023141590A2 (en) 2022-01-21 2023-07-27 Mammoth Biosciences, Inc. Effector proteins and methods of use
WO2023144104A1 (en) 2022-01-25 2023-08-03 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of βeta-thalassemia
WO2023196802A1 (en) 2022-04-04 2023-10-12 The Broad Institute, Inc. Cas9 variants having non-canonical pam specificities and uses thereof
JP2025515503A (ja) 2022-04-28 2025-05-15 ザ ブロード インスティテュート，インコーポレーテッド 塩基エディタをコードするａａｖベクターおよびその使用
US20250223326A1 (en) 2022-05-10 2025-07-10 Institut National de la Santé et de la Recherche Médicale Syncitin-1 fusion proteins and uses thereof for cargo delivery into target cells
US20250241956A1 (en) 2022-05-10 2025-07-31 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the cd39 (cag>tag) mutation in patients suffering from beta-thalassemia
WO2024018056A1 (en) 2022-07-22 2024-01-25 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from βeta-thalassemia
CN115820691B (zh) * 2022-07-25 2023-08-22 安徽农业大学 一种基于LbCpf1变体的水稻碱基编辑系统和应用
WO2024040083A1 (en) 2022-08-16 2024-02-22 The Broad Institute, Inc. Evolved cytosine deaminases and methods of editing dna using same
WO2024040874A1 (zh) * 2022-08-22 2024-02-29 山东舜丰生物科技有限公司 突变的Cas12j蛋白及其应用
KR20250075667A (ko) * 2022-09-23 2025-05-28 기초과학연구원 신규한 아데닌 탈아미노효소 변이체 및 이를 사용한 염기 교정 방법
JP2026506554A (ja) 2023-02-06 2026-02-25 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 多重塩基編集による遺伝子改変造血幹細胞の濃縮
WO2024230760A1 (zh) * 2023-05-09 2024-11-14 北京齐禾生科生物科技有限公司 一种可作用于dna的腺苷脱氨酶及其应用
CN117965505A (zh) * 2023-06-28 2024-05-03 微光基因(苏州)有限公司 工程化的腺苷脱氨酶及碱基编辑器
KR20260044217A (ko) 2023-07-25 2026-04-01 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 Cas 엔도뉴클레아제 및 관련 방법
WO2025059520A1 (en) * 2023-09-15 2025-03-20 Beam Therapeutics Inc. Compositions and methods for base editing a phenylalanine hydroxylase polynucleotide
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
CN120536418A (zh) * 2024-02-26 2025-08-26 尧唐(上海)生物科技有限公司 一种脱氨酶变体、包含其的碱基编辑器及其应用

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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
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KR102622411B1 (ko) * 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
EP3538561A4 (en) * 2016-11-11 2020-10-21 The Regents of The University of California RNA-GUIDED VARIANT POLYPEPTIDES AND THEIR METHODS OF USE
KR20250107288A (ko) * 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
CN109957569B (zh) * 2017-12-22 2022-10-25 苏州齐禾生科生物科技有限公司 基于cpf1蛋白的碱基编辑系统和方法
CN109295186B (zh) * 2018-09-30 2023-10-03 中山大学 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用

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