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US 2021/0228641 A1Published application
Genetically engineered hematopoietic stem cells and uses thereof
AssigneeVor Biopharma Inc.
InventorsJoseph Bolen, Aleksandar Filip Radovic-Moreno, John Lydeard
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Abstract
Genetically engineered hematopoietic cells such as hematopoietic stem cells having one or more genetically edited genes of lineage-specific cell-surface proteins and therapeutic uses thereof, either alone or in combination with immune therapy that targets the lineage-specific cell-surface proteins.
Claims28
A method, comprising administering to a subject in need thereof:
- (a) a plurality of genetically engineered hematopoietic cells (HCs) comprising:
- (i) a genetically engineered gene encoding CD33 that is engineered to have reduced or eliminated expression of CD33, and
- (ii) a genetically engineered gene encoding CD123 that is engineered to have reduced or eliminated expression of CD123;
- and
- (b) a therapeutically effective amount of at least one agent comprising an anti-CD33 binding domain, an anti-CD123 binding domain, or an anti-CD33 binding domain and an anti-CD123 binding domain.
2 more elements
Dependencies are inferred from the claim text.