UCSF Reprograms Immune Cells In Vivo to Fight Cancer — SkimNews
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- UCSF researchers used a two-particle CRISPR-Cas9 system to insert cancer-targeting DNA into T cells inside the body — the first time a large stretch of DNA has been placed at a precise genomic location in human T cells without first extracting them.
- One particle carries CRISPR-Cas9 tools coated with antibodies targeting CD3 (a T-cell-exclusive protein); the second delivers CAR instructions designed to activate only at a T-cell-specific genomic "on switch," avoiding off-target edits.
- In mouse experiments published in Nature, a single injection eliminated detectable leukemia within two weeks, with engineered CAR-T cells comprising up to 40% of the immune cell population in some organs; the same approach also cleared multiple myeloma and a solid sarcoma tumor.
- Senior author Justin Eyquem said the in vivo-generated cells actually outperformed laboratory-engineered CAR-T cells because lab-grown cells lose "stemness" and proliferative capacity outside the body.
- Current CAR-T therapy costs $400,000–$500,000 and requires weeks of external cell manufacturing; the in vivo approach could eliminate that process, the need for preparatory chemotherapy, and extend access beyond major cancer centers.
- Eyquem and collaborators from Gladstone Institutes, Duke University, and the Innovative Genomics Institute have founded Azalea Therapeutics to move the platform toward clinical trials, though human safety and efficacy data remain ahead.
Why it matters: Today, the seven FDA-approved CAR-T therapies cost $400,000–$500,000 and require specialized manufacturing facilities, restricting access to major cancer centers. UCSF's single-injection in vivo approach worked in mice against leukemia, multiple myeloma, and even a solid sarcoma tumor — historically CAR-T's hardest target — and produced cells that outperformed their lab-grown counterparts. Human trials remain the next gate.
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