Drug intelligence / Profile preview

MuSK-CAART + cyclophosphamide + fludarabine

Development stage
Unknown
Lead developer
Cabaletta Bio
Modality
Cell Therapies, Small Molecules
Administration
Intravenous
01

Overview

This is a **combination therapy** comprising MuSK-CAART (Muscle-Specific Kinase Chimeric AutoAntibody Receptor T cell therapy), **cyclophosphamide**, and **fludarabine**. MuSK-CAART is an experimental, autologous T cell therapy in which patient-derived T cells are genetically engineered to express a chimeric autoantibody receptor (CAAR) containing the extracellular domain of MuSK and signaling domains (4-1BB and CD3 zeta), enabling selective targeting and elimination of B cells expressing antibodies against muscle-specific kinase (MuSK). The principal indication is **MuSK antibody-positive myasthenia gravis**. Cyclophosphamide and fludarabine are chemotherapy agents used as a preconditioning lymphodepleting regimen prior to cell therapy infusion, to enhance expansion and persistence of infused CAAR-T cells. Cyclophosphamide is an alkylating agent that suppresses the immune system, while fludarabine is a purine analog with antimetabolite properties, both commonly used in preparative regimens before CAR-T/CAAR-T cell therapies[1][2][4].

02

Targets

BCR (Immunoglobulin G B Cell Receptor)

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