MB-107 (St. Jude Children's Research Hospital)

MB-107 (and its related product MB-207) is an experimental ex vivo autologous lentiviral gene therapy developed by St. Jude Children's Research Hospital in collaboration with the NIH and UCSF, and licensed to Mustang Bio. The therapy targets X-linked severe combined immunodeficiency (X-SCID or SCID-X1), a life-threatening genetic disorder caused by mutations in the *IL2RG* gene, which encodes the common gamma chain (γc) essential for cytokine signaling in immune cells. The treatment involves harvesting a patient's CD34+ hematopoietic stem cells and transducing them with a self-inactivating (SIN) lentiviral vector carrying a codon-optimized human *IL2RG* complementary DNA (cDNA) under the control of an EF1α promoter. Following low-dose busulfan conditioning, the gene-corrected cells are reinfused into the patient to restore the development and function of T, B, and natural killer (NK) cells. Clinical data have demonstrated broad immune reconstitution in both newly diagnosed infants and older patients who failed prior hematopoietic stem cell transplantation.

Other names

Lentiviral gene therapy for X-SCIDSt. Jude SCID-X1 gene therapyIL2RG gene therapyIL-2RG gene therapyIL 2RG gene therapyLentiviral gene therapy for SCID-X1