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US 10,925,902 B2Granted
Genetically engineered hematopoietic stem cells and uses thereof
AssigneeVor Biopharma Inc.
InventorsJoseph Bolen, Aleksandar Filip Radovic-Moreno, John Lydeard
Life of this patent
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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.
Claims19
A method of treating a hematopoietic malignancy, 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 CLL1 that is engineered to have reduced or eliminated expression of CLL1;
- and
- (b) a therapeutically effective amount of at least one agent comprising an anti-CD33 binding domain or an anti-CLL1 binding domain;
- wherein the hematopoietic malignancy is acute myeloid leukemia (AML).
3 more elements
A pharmaceutical composition comprising:
- (a) a plurality of hematopoietic cells (HCs), wherein the plurality of HCs comprises
- (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 CLL1 that is engineered to have reduced or eliminated expression of CLL1;
- and
- (b) a therapeutically effective amount of at least one agent comprising an anti-CD33 binding domain or an anti-CLL1 binding domain.
2 more elements
A method of preparing a population of genetically engineered hematopoietic cells (HCs), the method comprising:
- (a) providing a population of HCs obtained from a human subject, wherein the population of HCs or descendants thereof express CD33 and CLL1;
- and
- (b) genetically modifying the population of HCs such that
- (i) a gene encoding CD33 is engineered to have reduced or eliminated expression of CD33;
- and
- (ii) a gene encoding CLL1 is engineered to have reduced or eliminated expression of CLL1.
3 more elements
Dependencies are inferred from the claim text.