SGX-001

SGX-001 is a lentiviral gene therapy being developed by Somagenetix AG for the treatment of p47 chronic granulomatous disease (CGD), a rare inherited immunodeficiency disorder. The therapy aims to correct genetic defects in phagocytes—white blood cells responsible for killing pathogens—by delivering a functional copy of the neutrophil cytosolic factor 1 (NCF1) gene using a lentiviral vector. This approach seeks to restore normal immune function in patients with p47-CGD, who otherwise suffer from recurrent life-threatening infections due to dysfunctional phagocytes. The current standard of care is stem cell transplantation, which requires an immunologically compatible donor and is not always available. SGX-001 has received orphan drug designation and is currently in preclinical development, with clinical trials planned for 2026[1][2][3].