autologous CD34+ cells transduced with a lentiviral vector encoding the human NCF1 gene clinical trials

An open-label, single-arm, phase 1/2 first-in-human study to assess the safety and efficacy of autologous CD34+ cells transduced with a lentiviral vector encoding the human NCF1 gene (SGX-001) in paediatric and adult patients with chronic granulomatous disease caused by p47phox deficiency

This study is testing a new gene therapy called SGX-001 for children and adults with a specific type of chronic granulomatous disease (CGD). It aims to see if the treatment is safe and if it helps the body fight infections better.