JP2025087691A5 - - Google Patents

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Publication number
JP2025087691A5
JP2025087691A5 JP2025017262A JP2025017262A JP2025087691A5 JP 2025087691 A5 JP2025087691 A5 JP 2025087691A5 JP 2025017262 A JP2025017262 A JP 2025017262A JP 2025017262 A JP2025017262 A JP 2025017262A JP 2025087691 A5 JP2025087691 A5 JP 2025087691A5
Authority
JP
Japan
Prior art keywords
tada
amino acid
adenosine deaminase
gsd1a
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
JP2025017262A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025087691A (ja
Filing date
Publication date
Priority claimed from PCT/US2020/018124 external-priority patent/WO2020168088A1/en
Application filed filed Critical
Publication of JP2025087691A publication Critical patent/JP2025087691A/ja
Publication of JP2025087691A5 publication Critical patent/JP2025087691A5/ja
Pending legal-status Critical Current

Links

JP2025017262A 2019-02-13 2025-02-05 糖原病1a型を治療するための組成物および方法 Pending JP2025087691A (ja)

Applications Claiming Priority (18)

Application Number Priority Date Filing Date Title
US201962805271P 2019-02-13 2019-02-13
US62/805,271 2019-02-13
US201962852228P 2019-05-23 2019-05-23
US201962852224P 2019-05-23 2019-05-23
US62/852,224 2019-05-23
US62/852,228 2019-05-23
US201962876354P 2019-07-19 2019-07-19
US62/876,354 2019-07-19
US201962912992P 2019-10-09 2019-10-09
US62/912,992 2019-10-09
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/US2020/018124 WO2020168088A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating glycogen storage disease type 1a
JP2021546892A JP7631209B2 (ja) 2019-02-13 2020-02-13 糖原病1a型を治療するための組成物および方法

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2021546892A Division JP7631209B2 (ja) 2019-02-13 2020-02-13 糖原病1a型を治療するための組成物および方法

Publications (2)

Publication Number Publication Date
JP2025087691A JP2025087691A (ja) 2025-06-10
JP2025087691A5 true JP2025087691A5 (https=) 2025-11-26

Family

ID=72045644

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021546892A Active JP7631209B2 (ja) 2019-02-13 2020-02-13 糖原病1a型を治療するための組成物および方法
JP2025017262A Pending JP2025087691A (ja) 2019-02-13 2025-02-05 糖原病1a型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
JP2021546892A Active JP7631209B2 (ja) 2019-02-13 2020-02-13 糖原病1a型を治療するための組成物および方法

Country Status (8)

Country Link
US (1) US20220127594A1 (https=)
EP (1) EP3924478A4 (https=)
JP (2) JP7631209B2 (https=)
KR (1) KR20210129108A (https=)
CN (2) CN120138047A (https=)
AU (1) AU2020221355A1 (https=)
CA (1) CA3128886A1 (https=)
WO (1) WO2020168088A1 (https=)

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WO2019178427A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2019222555A1 (en) 2018-05-16 2019-11-21 Arbor Biotechnologies, Inc. Novel crispr-associated systems and components
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
WO2020168088A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
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
JP2023505234A (ja) * 2019-12-04 2023-02-08 アーバー バイオテクノロジーズ, インコーポレイテッド ヌクレアーゼを含む組成物及びその使用
US12416001B2 (en) * 2020-02-14 2025-09-16 Ohio State Innovation Foundation Nucleobase editors and methods of use thereof
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
EP4504947A4 (en) * 2022-04-07 2026-03-25 Univ Duke COMPOSITIONS AND METHODS TO PROMOTE LIVER REGENERATION BY GENE EDITING IN METABOLIC LIVER DISEASE
CN117568313B (zh) * 2024-01-15 2024-04-26 上海贝斯昂科生物科技有限公司 基因编辑组合物及其用途

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL310721B2 (en) * 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
BR112018008971A2 (pt) * 2015-11-06 2018-11-27 Crispr Therapeutics Ag materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
LT3436077T (lt) * 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
WO2018020323A2 (en) * 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
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
RU2019130504A (ru) * 2017-02-28 2021-03-30 Вор Байофарма, Инк. Композиции и способы ингибирования линиеспецифических белков
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
US10392616B2 (en) * 2017-06-30 2019-08-27 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
CN111801345A (zh) * 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
KR20250133482A (ko) * 2018-05-11 2025-09-05 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법
WO2020168088A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
EP4100519A2 (en) * 2020-02-05 2022-12-14 The Broad Institute, Inc. Adenine base editors and uses thereof

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