SIN-IL2RG-LV

SIN-IL2RG-LV is an ex vivo gene therapy candidate designed to treat X-linked severe combined immunodeficiency (X-SCID), a life-threatening primary immunodeficiency caused by mutations in the *IL2RG* gene. The therapy utilizes a self-inactivating (SIN) lentiviral vector (LV) to deliver a functional copy of the Interleukin 2 receptor subunit gamma (*IL2RG*) gene into a patient's own autologous CD34+ hematopoietic stem cells. The *IL2RG* gene encodes the common gamma (γc) chain, a critical component of receptors for several cytokines (IL-2, IL-4, IL-7, IL-9, IL-15, and IL-21) that are essential for the development and function of T-cells, B-cells, and natural killer (NK) cells. By restoring γc chain expression and subsequent JAK-STAT signaling, the therapy aims to reconstitute a functional immune system in patients who lack suitable matched donors for allogeneic hematopoietic stem-cell transplantation. Preclinical studies have demonstrated its safety and efficacy in restoring immune function in patient-derived cells and animal models.

Other names

IL2RG-SIN-LVIL-2RG-SIN-LVIL 2RG-SIN-LVSelf-inactivating lentiviral vector carrying IL2RGpCCL-EF1α-hIL2RG