MB-117

MB-117 is an investigational **ex vivo autologous hematopoietic stem and progenitor cell gene therapy** for X-linked severe combined immunodeficiency. The product uses a self-inactivating lentiviral vector to introduce a functional copy of the *IL2RG* gene into a patient's CD34-positive hematopoietic cells before intravenous reinfusion. Restored expression of the common gamma-chain cytokine-receptor subunit is intended to re-establish signaling required for T-cell, B-cell, and natural-killer-cell development and function. MB-117 has been developed through a Mustang Bio and St. Jude Children's Research Hospital collaboration as a potentially curative treatment for newly diagnosed X-linked severe combined immunodeficiency.

Other names

CD34+ HSCs transduced with the lentivirus vector VSV-G pseudotyped CL20-4i-EF1α-hγc-OPTVSV-G pseudotyped CL20-4i-EF1α-hγc-OPT transduced CD34+ HSCs