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Researchers at UC San Francisco have developed a way to engineer cancer-fighting CAR-T cells directly inside the body using CRISPR — no lab required. The two-particle delivery system targets T cells in vivo, potentially slashing the cost and wait time of a therapy that currently runs $400K–$500K and takes weeks to produce. Early results in mice showed near-complete elimination of leukemia, multiple myeloma, and even solid tumors.
CAR-T cell therapy has been a game-changer for blood cancers, but its sky-high costs and complex manufacturing process put it out of reach for many patients. The standard approach requires extracting a patient's immune cells, shipping them to a specialized facility for genetic modification, and infusing them back — a process that takes weeks and can cost up to half a million dollars. Now, UC San Francisco researchers may have found a way to skip all of that.
The team developed a two-particle delivery system that sends CRISPR gene-editing tools and cancer-targeting DNA directly into T cells inside the body. The new DNA is inserted at a precise genomic location — an approach that outperformed traditional virus-based methods. In mice, a single injection wiped out nearly all detectable leukemia within two weeks, and the strategy also worked against multiple myeloma and a solid sarcoma tumor. Notably, the in vivo-engineered T cells also appeared to retain more "stemness" and proliferative capacity than their lab-made counterparts.
By the Numbers
Why it matters: If this approach translates to humans, it could eliminate the need for specialized manufacturing facilities, reduce costs dramatically, and allow community hospitals — not just major cancer centers — to offer CAR-T therapy. The team has founded Azalea Therapeutics to advance the platform toward clinical trials.