IVLV-X1

IVLV-X1 is an investigational gene therapy designed for the treatment of X-linked severe combined immunodeficiency (SCID-X1), a rare genetic disorder characterized by profound defects in immune system development. The therapy utilizes a self-inactivating lentiviral vector (LV) carrying a functional copy of the IL2RG gene, which encodes the common gamma chain of several interleukin receptors essential for lymphocyte development and function. By direct intravenous injection, IVLV-X1 delivers the therapeutic gene to modify immune cells in vivo, aiming to restore normal immune function and correct the underlying immunodeficiency. The primary objectives in clinical trials are to evaluate safety, efficiency of gene transfer, and long-term correction of immunodeficiency through immune reconstitution[6][1].