MUNC-T3

MUNC-T3 is an investigational ex vivo gene therapy consisting of autologous T-cells transduced with a lentiviral vector expressing the human UNC13D cDNA. Developed by Assistance Publique - Hôpitaux de Paris, it is designed to treat familial hemophagocytic lymphohistiocytosis type 3 (FHL3), a rare and life-threatening autosomal recessive immunodeficiency. FHL3 is caused by mutations in the UNC13D gene, which encodes the Munc13-4 protein essential for the fusion of cytolytic granules in cytotoxic T-lymphocytes and natural killer cells. By restoring functional Munc13-4 expression, MUNC-T3 aims to re-establish normal immune effector function and control viral triggers. In clinical trials, MUNC-T3 is administered as a component of a dual gene therapy approach alongside MUNC-CD34 (transduced autologous hematopoietic stem cells) to provide immediate therapeutic benefit while awaiting full hematopoietic reconstitution.

Other names

transduced autologous T-cellsUNC13D-transduced T-cellsUNC-13D-transduced T-cellsUNC 13D-transduced T-cells