KR20210129108A - 글리코겐 저장 질환 1a형을 치료하기 위한 조성물 및 방법 - Google Patents

글리코겐 저장 질환 1a형을 치료하기 위한 조성물 및 방법 Download PDF

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KR20210129108A
KR20210129108A KR1020217029280A KR20217029280A KR20210129108A KR 20210129108 A KR20210129108 A KR 20210129108A KR 1020217029280 A KR1020217029280 A KR 1020217029280A KR 20217029280 A KR20217029280 A KR 20217029280A KR 20210129108 A KR20210129108 A KR 20210129108A
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tada
adenosine deaminase
cas9
domain
variant
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Korean (ko)
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니콜 가우델리
마이클 페커
이안 슬레이메이커
이 유
베른트 제츠체
이본 아라틴
프랑신 그레고아르
제네시스 룽
데이비드 에이. 본
승주 이
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빔 테라퓨틱스, 인크.
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KR1020217029280A 2019-02-13 2020-02-13 글리코겐 저장 질환 1a형을 치료하기 위한 조성물 및 방법 Pending KR20210129108A (ko)

Applications Claiming Priority (17)

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
US62/852,228 2019-05-23
US62/852,224 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

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KR20210129108A true KR20210129108A (ko) 2021-10-27

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KR1020217029280A Pending KR20210129108A (ko) 2019-02-13 2020-02-13 글리코겐 저장 질환 1a형을 치료하기 위한 조성물 및 방법

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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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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

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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
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CN114026237A (zh) 2022-02-08
JP7631209B2 (ja) 2025-02-18
AU2020221355A1 (en) 2021-08-12
CN120138047A (zh) 2025-06-13
JP2022519882A (ja) 2022-03-25
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US20220127594A1 (en) 2022-04-28
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