CRISPR-edited stem cell transplant targets blood cancer while sparing healthy cells

A WashU Medicine trial of 30 patients with AML and MDS found that donor stem cells edited with CRISPR to remove CD33 engrafted as safely as standard transplants. The approach could let CD33-targeted therapies attack cancer without destroying healthy blood cells.

• 30 adults with AML or MDS received donor stem cells with CD33 removed via CRISPR
• All 30 patients achieved engraftment by day 28; platelets recovered by day 16 on average
• Average survival in the trial was just over 14 months
• One patient stayed cancer-free more than a year after CD33-targeted CAR-T therapy