RPCAR01

RPCAR01 is an investigational autologous chimeric antigen receptor (CAR) T-cell therapy developed by Roswell Park Comprehensive Cancer Center for the treatment of advanced or metastatic hepatocellular carcinoma (HCC). The therapy is engineered to target glypican-3 (GPC3), a cell-surface protein frequently overexpressed in HCC but rarely found in healthy adult tissues. To enhance the persistence and efficacy of the T cells within the immunosuppressive tumor microenvironment, RPCAR01 utilizes CRISPR/Cas9 genome-editing technology to disrupt the transforming growth factor-beta receptor 2 (TGFβR2) gene. This modification is designed to prevent TGFβ-mediated signaling from inhibiting the T cells' cytotoxic activity, potentially allowing for more robust and sustained anti-tumor responses. RPCAR01 is currently being evaluated in a Phase I clinical trial to assess its safety, tolerability, and preliminary efficacy in patients with GPC3-positive relapsed or refractory HCC.

Other names

Anti-GPC3-CAR Autologous T LymphocytesAnti-GPC-3-CAR Autologous T LymphocytesAnti-GPC 3-CAR Autologous T LymphocytesGPC3-targeted CAR-T cells with TGFβR2 disruption