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Cancer BiologybioRxiv
Preprint

BCMA/CD19 dual-targeting with FasTCAR: AZD0120 demonstrates potent preclinical efficacy in multiple myeloma

Huan Shi, Wenjie YiniD, Hua Zhang, Xiaoyan Jiang, Jiaping HeiD, Guangyao Zhu, Michael Glen OverstreetiD, Mark CobboldiD, Lianjun Shen

Abstract

Chimeric antigen receptor (CAR)-T cell therapy has improved outcomes for patients with multiple myeloma (MM), but its broader use is restricted by manufacturing complexities and treatment-related toxicities. AZD0120 is a dual-targeting B-cell maturation antigen (BCMA)/CD19 CAR-T cell therapy manufactured via the rapid FasTCAR process. We developed a dual-targeting "loop" CAR that incorporates a novel humanized anti-BCMA single-chain variable fragment (scFv), clone SG, and an FMC63-derived anti-CD19 scFv. This AZD0120 CAR preserved functional binding to both antigens and conferred robust in vitro and in vivo cytotoxicity while maintaining single-antigen reactivity. Conventional manufacture of CAR-T cells with the AZD0120 CAR (AZD0120C) yielded cells with minimal tonic signaling, limited responsiveness to soluble BCMA, and preservation of naive/stem cell memory-enriched phenotypes, yet robust cytokine production upon BCMA+ target engagement. AZD0120C demonstrated cytotoxicity comparable to benchmark BCMA CAR-Ts across MM lines in vitro and showed strong in vivo expansion and tumor control in xenograft models. FasTCAR manufacturing - designed to shorten vein-to-vein timelines and enrich less-differentiated phenotypes - further enhanced in vivo performance: AZD0120 consistently achieved superior tumor control and greater CAR-T expansion vs AZD0120C across disseminated MM.1S, NALM-6, and JeKo-1 models, with superior efficacy observed at lower cell doses. Collectively, these data support clinical evaluation of AZD0120 as a differentiated BCMA/CD19 CAR-T cell therapy with the potential to improve disease control and patient access in MM. Key PointsO_LIAZD0120 is a dual-targeting CAR-T that displays a favorable anti-myeloma functional profile and co-targets a source of potential relapse C_LIO_LIThe FasTCAR process yields TN/SCM-rich CAR-T populations, promotes in vivo expansion and achieves potent tumor control in xenograft models C_LI

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