RiboCAR-T / MeiraGTx - LARVOL DELTA

Riboswitch-regulated gene and cell therapy (ESGCT 2024) - "RiboCAR-T cells with riboswitch-controlled chimeric antigen receptors (CARs) had more stem/memory-like phenotypes and exhibited superior anti-tumor activities against lymphoma when compared with conventional CAR-T cells that expressed constitutive CAR. Thus, we have developed the first synthetic aptamer riboswitch that is capable of controlling therapeutic gene expression through orally available small-molecule inducers. This robust gene regulation system enables both temporal and spatial control of gene expression, providing not only improved efficacy but also a safety mechanism for both gene and cell therapies."

IO biomarker • Hematological Malignancies • Lymphoma

Riboswitch‐regulated chimeric antigen receptor (RiboCAR) enhances CAR‐T cell anti‐cancer efficacy (ESGCT 2023) - "More importantly, T cells with RiboCAR showed delayed exhaustion during expansion in the absence of small molecule inducer and enhanced target cell-stimulated T cell activation and anti-cancer cytotoxicity in the presence of small molecule inducer, when compared with T cells constitutively expressing CAR. With a bioavailable small molecule inducer, the RiboCAR-T cell activity can be precisely tuned and "remotely" controlled in vivo, thus improving the efficacy and safety of CAR-T cell therapy."

CAR T-Cell Therapy • Clinical • Oncology