Ribo-CAR-T

Ribo-CAR-T is a proprietary riboswitch-regulated CAR-T cell therapy platform developed by MeiraGTx. The technology incorporates a synthetic mammalian ON riboswitch into the coding sequence of the chimeric antigen receptor (CAR) transgene, allowing for precise, dose-dependent control of CAR expression via an orally available small molecule inducer, such as MXU-001. Unlike traditional CAR-T therapies that express the CAR constitutively, Ribo-CAR-T enables CAR expression to be tuned from undetectable levels in the absence of the inducer to levels that can exceed standard constitutive expression when the inducer is present. This regulated approach is designed to enhance T cell activity, reduce exhaustion markers, and improve the safety profile by allowing clinicians to modulate or pause therapy if adverse effects occur. Preclinical studies have demonstrated significant improvements in cytotoxicity and in-vivo potency compared to conventional CD19 CAR-T therapies. The platform is currently in preclinical development for applications in oncology and autoimmune diseases.

Other names

riboswitch regulated CAR-T cell therapy