Superior In Vivo Efficacy for T Cell Engineering via Citronellol‐Tailored mRNA‐tLNPs
Efficient in vivo generation of CAR T cells via mRNA-LNP delivery necessitates high-efficiency T cell engineering, yet current systems remain suboptimal. While lymphoid tropism is essential, it is demonstrated that high tropism alone is insufficient for effective in vivo engineering-some highly tropic LNPs frequently exhibit suboptimal T cell transfection. Crucially, intrinsic T cell transfection potency emerges as an orthogonal and equally critical determinant, consistent with the need for both T cell specificity and transfection efficiency. Through screening a tail-varied ionizable lipid library, H<sub>3</sub>T<sub>4</sub> is identified, a citronellol-tailed LNP with enhanced lymphoid tropism and superior intrinsic potency. The CD3-targeted H<sub>3</sub>T<sub>4</sub> LNP (αH<sub>3</sub>T<sub>4</sub>) achieves ~85% in vivo efficacy in T cell engineering, outperforming benchmark LNPs by ∼50% (40% vs. 85%). This proposes a new optimization strategy: effective engineering requires balancing potency and tropism, not maximizing tropism alone. In murine CD19 solid tumor models, αH<sub>3</sub>T<sub>4</sub>-generated CAR T cells achieved 83% tumor clearance, demonstrating potency-driven efficacy. This work establishes intrinsic transfection potency as a primary tunable parameter for next-generation CAR T platforms.
Authors
- Zhe Hu (ORCID: https://orcid.org/0000-0001-7652-6939)
- Li Wang (ORCID: https://orcid.org/0000-0001-8527-6345)
- Y. H. Zhan (ORCID: https://orcid.org/0009-0009-9399-5548)
- Di Yu
- Y. Xu
- Jun Deng
- Jun Guo
- Yanfang Cui
- Baoqian Zhang
- Shuaijie Ji
- Chunrong Liu
- Hong Yuan
- Junlin Zhu
Institutions
- Translational Research Institute (AU)
- The University of Queensland (AU)
- Huazhong Agricultural University (CN)
- Renji Hospital (CN)
- Central China Normal University (CN)
- Shanghai Cancer Institute (CN)
- State Key Laboratory of Oncogene and Related Genes (CN)
Publication Details
- Journal
- Small
- Published
- 2026-03-12
- DOI
- https://doi.org/10.1002/smll.202509742
- Primary Topic
- CAR-T cell therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00