SGX-002

SGX-002 is an ex vivo lentiviral gene therapy being developed by Somagenetix for the treatment of X-linked Chronic Granulomatous Disease (X-CGD). The therapy utilizes a self-inactivating (SIN) lentiviral vector to deliver a functional copy of the CYBB gene, which encodes the gp91phox protein, into the patient's own hematopoietic stem cells. X-CGD is a rare primary immunodeficiency caused by mutations in the CYBB gene, leading to a lack of superoxide production in phagocytes and increased susceptibility to severe infections. By restoring gp91phox expression, SGX-002 aims to provide a durable cure for patients with this life-threatening condition. It is currently in the preclinical stage of development.