KR20230124553A - 글리코겐축적병 1a형을 치료하기 위한 조성물 및 방법 - Google Patents

글리코겐축적병 1a형을 치료하기 위한 조성물 및 방법 Download PDF

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KR20230124553A
KR20230124553A KR1020237016287A KR20237016287A KR20230124553A KR 20230124553 A KR20230124553 A KR 20230124553A KR 1020237016287 A KR1020237016287 A KR 1020237016287A KR 20237016287 A KR20237016287 A KR 20237016287A KR 20230124553 A KR20230124553 A KR 20230124553A
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seq
cas9
amino acid
polynucleotide
modified
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Korean (ko)
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브라이언 캐퍼티
탕기스 보누드
로-아이 쳉
마이클 페커
이본느 아라틴-샤우스
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빔 테라퓨틱스, 인크.
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    • C12Y305/04004—Adenosine deaminase (3.5.4.4)

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KR1020237016287A 2020-10-14 2021-10-14 글리코겐축적병 1a형을 치료하기 위한 조성물 및 방법 Pending KR20230124553A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063091891P 2020-10-14 2020-10-14
US63/091,891 2020-10-14
US202163248081P 2021-09-24 2021-09-24
US63/248,081 2021-09-24
PCT/US2021/055057 WO2022081890A1 (en) 2020-10-14 2021-10-14 Compositions and methods for treating glycogen storage disease type 1a

Publications (1)

Publication Number Publication Date
KR20230124553A true KR20230124553A (ko) 2023-08-25

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KR1020237016287A Pending KR20230124553A (ko) 2020-10-14 2021-10-14 글리코겐축적병 1a형을 치료하기 위한 조성물 및 방법

Country Status (7)

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US (1) US20230383277A1 (https=)
EP (1) EP4229195A4 (https=)
JP (1) JP2023545497A (https=)
KR (1) KR20230124553A (https=)
AU (1) AU2021360919A1 (https=)
CA (1) CA3198671A1 (https=)
WO (1) WO2022081890A1 (https=)

Families Citing this family (8)

* 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
CA3128755C (en) 2019-02-13 2024-06-04 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
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
CA3196831A1 (en) 2020-09-25 2022-03-31 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
WO2025194124A1 (en) * 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. Modified st1cas9 guide nucleic acids

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
US11946040B2 (en) * 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
WO2020168088A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
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
KR20240037299A (ko) * 2021-07-23 2024-03-21 빔 테라퓨틱스, 인크. Crispr/cas 편집 시스템용 가이드 rna

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AU2021360919A1 (en) 2023-06-01
WO2022081890A1 (en) 2022-04-21
EP4229195A1 (en) 2023-08-23
JP2023545497A (ja) 2023-10-30
EP4229195A4 (en) 2025-08-13
US20230383277A1 (en) 2023-11-30
CA3198671A1 (en) 2022-04-21

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