Researchers at University of California San Francisco have developed a new approach that could transform CAR-T cell therapy by turning it into a simple injection instead of a complex lab procedure.
Traditional CAR-T therapy requires removing a patient’s immune cells, modifying them in a lab, and reinfusing them weeks later. This process is slow, expensive, and limited to specialized centers, often costing up to $500,000.
The new method uses a dual-particle system to reprogram T cells directly inside the body. One particle targets T cells in the bloodstream, while the other delivers CRISPR-Cas9 to precisely edit their DNA. This adds a gene that enables the cells to recognize and attack cancer. Built-in safeguards ensure the gene only activates in the correct location.
In mouse studies, a single injection rapidly eliminated aggressive leukemia within two weeks. The approach also showed effectiveness in multiple myeloma and some solid tumors. Notably, the engineered cells were stronger and longer-lasting than lab-made versions, and the method may remove the need for pre-treatment chemotherapy.
Researchers have launched Azalea Therapeutics to advance this technology into human trials, aiming to make CAR-T therapy faster, safer, and widely accessible.