MuSK-CAART

MuSK-CAART is an investigational autologous cell therapy designed to treat patients with myasthenia gravis who are positive for antibodies against muscle-specific kinase (MuSK). The therapy consists of patient-derived T cells engineered to express a chimeric autoantibody receptor (CAAR) that incorporates the extracellular domain of the MuSK protein. This design enables the modified T cells to specifically recognize and eliminate B-cells producing pathogenic anti-MuSK antibodies, while sparing other healthy B-cells. The CAAR construct includes a 4-1BB costimulatory domain and a CD3 zeta signaling domain to enhance T-cell activation and persistence. By selectively depleting autoreactive B-cells, MuSK-CAART aims to reduce disease-driving autoantibodies without causing generalized immunosuppression. Developed by Cabaletta Bio, it is currently being evaluated in Phase 1 clinical trials as a one-time intravenous infusion for patients with anti-MuSK antibody-positive myasthenia gravis[1][2][3][4][5][7][8].

Other names

muscle-specific kinase chimeric autoantibody receptor T cell therapyMuSK CAART