JP2023545497A - グリコーゲン貯蔵症１ａ型を治療するための組成物及び方法 - Google Patents

グリコーゲン貯蔵症１ａ型を治療するための組成物及び方法 Download PDF

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JP2023545497A
JP2023545497A JP2023522972A JP2023522972A JP2023545497A JP 2023545497 A JP2023545497 A JP 2023545497A JP 2023522972 A JP2023522972 A JP 2023522972A JP 2023522972 A JP2023522972 A JP 2023522972A JP 2023545497 A JP2023545497 A JP 2023545497A
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seq
modified
cas9
polynucleotide
adenosine deaminase
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ブライアン キャファティ―、
タンギス ボーヌード、
ロ―アイ チェン、
マイケル パッカー、
イボンヌ アラティン―シャウス、
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ビーム セラピューティクス インク．
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JP2023522972A 2020-10-14 2021-10-14 グリコーゲン貯蔵症１ａ型を治療するための組成物及び方法 Pending JP2023545497A (ja)

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

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JP2023545497A true JP2023545497A (ja) 2023-10-30
JP2023545497A5 JP2023545497A5 (https=) 2024-10-23

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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

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020028823A1 (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
WO2020168135A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2020168088A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
WO2020168122A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
KR20240037299A (ko) * 2021-07-23 2024-03-21 빔 테라퓨틱스, 인크. Crispr/cas 편집 시스템용 가이드 rna

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020028823A1 (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
WO2020168135A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
WO2020168088A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
WO2020168122A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence

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AU2021360919A1 (en) 2023-06-01
WO2022081890A1 (en) 2022-04-21
EP4229195A1 (en) 2023-08-23
EP4229195A4 (en) 2025-08-13
KR20230124553A (ko) 2023-08-25
US20230383277A1 (en) 2023-11-30
CA3198671A1 (en) 2022-04-21

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