The Promise of CRISPR Gene Editing in AML Treatment

VOR Bio, a biotech company in Cambridge, is developing a unique application of CRISPR gene editing for the treatment of acute myeloid leukemia (AML). By using CRISPR to delete the gene for the CD33 antigen found on both cancer cells and healthy blood-forming stem cells, VOR aims to make AML patients more responsive to targeted therapies like Pfizer's Myletarg. Preliminary clinical trial results have shown successful deletion of CD33 from stem cells, the engraftment of edited cells in the bone marrow, and promising safety profiles. VOR plans to expand its approach by developing a CD33-directed CAR-T cell therapy, leveraging CRISPR to protect bone marrow transplants and enable the T-cell therapy to kill remaining cancer cells. By harnessing the potential of CRISPR and cell therapies, VOR aims to revolutionize AML treatment and potentially achieve long-term remission and cure for patients.