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US 2026/0256948 A1Published application
Systems and methods for the treatment of hemoglobinopathies
AssigneeEditas Medicine, Inc.
InventorKaiHsin CHANG
Life of this patent
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Abstract
Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci in cells and increasing expression of fetal hemoglobin.
Claims67
A method of inducing expression of fetal hemoglobin (HbF) in a population of cells (e.g., CD34+ hematopoietic stem and progenitor cells (HSPCs) and/or red blood cells (RBCs), in a subject suffering from a β-hemoglobinopathy, the method comprising:
- administering to the subject a population of modified cells comprising a plurality of modified CD34+ or hematopoietic stem cells comprising an indel in an HBG promoter,
- thereby inducing expression of HbF in the population of cells (e.g., HSPCs and/or RBCs), wherein HbF as a percentage of total hemoglobin (% HbF) in the subject is about 5%, 10%, 15%, 20%, 25%, 30%, 35%, 40%, 45%, 50%, 55%, 60%, 65%, 70%, 75%, or 80% HbF.
A method of producing a modified population of peripheral blood nucleated cells in a subject, the method comprising:
- administering to the subject a population of modified cells comprising a plurality of modified CD34+ or hematopoictic stem cells comprising an indel in an HBG promoter,
- thereby producing the modified population of peripheral blood nucleated cells, wherein the modified population of peripheral blood nucleated cells as a percentage of the total population of peripheral blood nucleated cells is at least 50%, 55%, 60%, 65%, 70%, 75%, 80%, 85%, 90% or 95%.
A method of generating a population of F-cells in a subject, the method comprising:
- administering to the subject a population of modified cells comprising a plurality of modified CD34+ or hematopoietic stem cells comprising an indel in an HBG promoter,
- wherein the percentage of F-cells among circulating red blood cells six months after said administering is at least 90%.
Dependencies are inferred from the claim text.