Chronic Granulomatous Disease research hub

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

This study is testing a new gene editing treatment called PM359 for Chronic Granulomatous Disease (CGD) caused by a specific genetic change. It aims to see if the treatment is safe and effective by correcting the faulty gene in a person's own cells before returning them to the body.