MuSK-CAART + cyclophosphamide

MuSK-CAART + cyclophosphamide is an investigational combination therapy for the treatment of muscle-specific kinase antibody-positive myasthenia gravis (MuSK-MG). MuSK-CAART is an autologous chimeric autoantibody receptor T-cell (CAAR-T) therapy that uses genetically modified T-cells, engineered to express a chimeric receptor containing a segment of the MuSK protein, enabling them to specifically target and eliminate MuSK-reactive B-cells responsible for producing pathogenic autoantibodies. The aim is to reduce autoantibody-mediated disease activity without causing broad immunosuppression. Cyclophosphamide is used as a preconditioning chemotherapy agent to deplete endogenous lymphocytes, enhancing the expansion and engraftment of infused MuSK-CAART cells. The combination is currently being evaluated in a Phase 1 clinical trial for safety and optimal dosing in patients with MuSK-MG[1][2][5].