- 专利标题: Compositions and methods for treating hemoglobinopathies
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申请号: US17720186申请日: 2022-04-13
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公开(公告)号: US11752202B2公开(公告)日: 2023-09-12
- 发明人: Ian Slaymaker , Nicole Gaudelli , Yi Yu , Bernd Zetsche , David A. Born , Seung-Joo Lee , Michael Packer
- 申请人: Beam Therapeutics Inc.
- 申请人地址: US MA Cambridge
- 专利权人: Beam Therapeutics Inc.
- 当前专利权人: Beam Therapeutics Inc.
- 当前专利权人地址: US MA Cambridge
- 代理机构: Greenberg Traurig, LLP
- 代理商 Melissa Hunter-Ensor; Nicholas R. Ballor
- 分案原申请号: US17214643 2021.03.26
- 主分类号: A61K38/50
- IPC分类号: A61K38/50 ; A61P7/06 ; A61K35/15 ; A61K35/18 ; A61K35/28 ; A61K38/46 ; C12N5/078 ; C12N9/22 ; C12N9/78 ; C12N15/11 ; C12N15/90 ; C07K14/47 ; A61P7/00 ; A61K31/7088
摘要:
The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.
公开/授权文献
- US20220370575A1 COMPOSITIONS AND METHODS FOR TREATING HEMOGLOBINOPATHIES 公开/授权日:2022-11-24
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