HGI-003

HGI-003 is an investigational gene therapy being developed by Shenzhen Hemogen for the treatment of X-linked Severe Combined Immunodeficiency (X-SCID). X-SCID is a rare, life-threatening primary immunodeficiency caused by mutations in the *IL2RG* gene, which encodes the common gamma chain (γc) shared by multiple interleukin receptors. This genetic defect leads to a profound deficiency of functional T cells and natural killer (NK) cells, leaving patients highly susceptible to severe infections. HGI-003 is designed to deliver a functional copy of the *IL2RG* gene into the patient's hematopoietic stem cells, aiming to restore immune system development and function. As of early 2024, the program is in the preclinical stage of development.