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Revisiting the need for mRNA nucleoside modification in CAR T cell engineering

Kahwaji, N.; Kotzian, N.; Prinz, J. M.; Pu, Y.; Kath, J.; Picht, S.; Hiller, A. L.; Dunne, C. M.; Launspach, M.; Kleyer, A.; Simon, D. N.; Wagner, D. L.; Pichon, C.; Kroenke, G.; Schmueck-Henneresse, M.; Volk, H.-D.; Gossen, M.; Drzeniek, N. M.

2025-01-21 bioengineering
10.1101/2025.01.21.630650 bioRxiv
Show abstract

mRNA-based chimeric antigen receptor (CAR)-T cells offer the promise of enhanced safety and simplified manufacturing. However, in vitro-transcribed (IVT) mRNA is known to trigger antiviral immune responses, inflammatory signaling, and apoptosis in transfected cells. To address these challenges and enable efficient IVT-mRNA expression, modified nucleosides, such as N1-methyl-pseudouridine (m1{Psi}), have become the gold standard for CAR-T cell production, albeit at increased cost. In this study, immune responses to IVT-mRNA were evaluated across five primary human cell types, including T-cells. Unexpectedly, T-cells, unlike other immune and non-immune cell types tested, exhibited no immune activation in response to unmodified mRNA. T-cell viability and cytokine secretion patterns remained unaffected, regardless of whether unmodified mRNA was delivered via lipid nanoparticles (LNPs) or electroporation. Furthermore, CAR expression levels in T-cells were not influenced by mRNA modification with m1{Psi} or 5-methoxy-uridine (5moU) nucleosides. The absence of nucleoside modifications did not compromise CAR-T cell cytotoxic potency, demonstrating that such modifications are not required for producing functional CAR-T cells. These findings eliminate the need for nucleoside modification in T-cell mRNA, simplifying and reducing the cost of CAR-T cell manufacturing while positioning IVT-mRNA as a highly efficient and minimally invasive tool for CAR-T cell engineering.

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