Next-Gen Immunotherapy: Engineering Cancer-Fighting Cells Inside the Body

In the 1990s, Bruce Levine and his colleagues set out to engineer immune cells to fight cancer—a bold idea few believed would work. “The wider community was very sceptical,” recalls Levine, an immunologist at the University of Pennsylvania’s Perelman School of Medicine. Their research was often relegated to obscure, poorly attended conference sessions.

Today, their innovation—CAR T cells—has become one of the most powerful tools for treating blood cancers. Ongoing studies suggest these engineered immune cells may also help combat brain tumors, other solid cancers, and autoimmune diseases. Analysts estimate the CAR T therapy market, projected to reach $11 billion this year, could grow to nearly $190 billion by 2034.

Yet the treatment is far from simple. CAR T therapy requires extracting a patient’s T cells, sending them to a lab for genetic modification to express a chimeric antigen receptor (CAR), growing them into hundreds of millions, freezing them, and sending them back for infusion. This complex process limits availability to just 200 centers in the U.S. and drives costs to around $500,000 per dose.

To overcome these barriers, some biotech firms are developing in vivo CAR T therapies—treatments that engineer T cells directly inside the body. These therapies could be mass-produced and distributed at significantly lower costs, potentially around one-tenth of current prices.

The idea has attracted major figures. Levine, Carl June, and Nobel laureate Drew Weissman co-founded Capstan Therapeutics. Jennifer Doudna co-founded Azalea Therapeutics. In March, AstraZeneca committed up to $1 billion to acquire in vivo CAR T developer EsoBiotec.

SOURCE: https://www.nature.com/articles/d41586-025-01570-6 

CREDITS: NATURE JOURNALS