CRISPR technique programs T cells inside patients to target cancer

Scientists at the University of California - San Francisco, together with collaborators from the Gladstone Institutes, Duke University and the Innovative Genomics Institute, have devised a dual-particle delivery platform that inserts a large DNA segment into a precise genomic site of T cells inside living organisms. The CRISPR-Cas9-loaded particle targets CD3 on T cells, while a second particle supplies the CAR gene, which integrates at a T-cell-specific “on switch.”

In humanized mouse models, a single dose cleared detectable leukemia within two weeks and also eradicated multiple myeloma and a solid sarcoma, with engineered cells comprising up to 40% of immune cells in some tissues. The in-vivo engineered T cells showed superior function compared with conventional lab-produced CAR-T cells. Although still pre-clinical, the researchers have founded Azalea Therapeutics to advance the approach toward human trials, aiming to cut costs, reduce waiting times and broaden access to CAR-T therapy.