Rapid CAR Screening and circRNA-driven CAR-NK Cells for Persistent Shed-resistant Immunotherapy
免疫疗法
医学
免疫学
计算机科学
免疫系统
作者
Joo-Yoon Chung,J.H. Hong,Su‐Min Yee,Hye Won Lim,Donghyeon Lee,Sang-Won Yoon,Seung-Hwan Lee,Yu-Jin Jung,Sue Shin,Song Cheol Kim,Chang‐Han Lee,Mihue Jang
标识
DOI:10.1101/2025.08.19.671003
摘要
Chimeric antigen receptor (CAR)-based therapeutics offer great potential against cancer and autoimmune diseases. This study presents a high-throughput mRNA-based platform to rapidly screen and optimize CAR-NK cells targeting shed-resistant membrane-proximal epitopes of mesothelin (MSLN)-an antigen frequently overexpressed in solid tumors. Using a yeast surface-display library, we identified single-chain variable fragments that selectively bind membrane-immobilized mature MSLN, minimizing inhibition by soluble MSLN (solMSLN) and enhancing cell-cell avidity. Linear mRNA (linRNA) encoding CAR was electroporated into human primary NK cells for live-cell phenotyping screening of antigen-specific cytotoxicity. Final optimization included co-stimulatory domain (OX40ζ) and cytokine (IL21) co-transfection. The finalized circular mRNA (circRNA)-based CAR-MS10-NK cells exhibited prolonged CAR and superior anti-tumor activities in MSLN-positive models, including solMSLN-secreting pancreatic tumors. In vivo, circCAR-MS10-NK cells significantly reduced tumor burden, outperforming lentivirus-based CAR-NK counterparts in both potency and manufacturability. This platform enables rapid non-viral CAR design and functional screening, for the scalable cost-effective development of shed-resistant antigen-targeting immunotherapies.