Prime Medicine, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the company?s Investigational New Drug (IND) application for PM359, submitted on March 29, for the treatment of chronic granulomatous disease (CGD), enabling the Company to initiate its global Phase 1/2 clinical trial in the United States. The Phase 1/2 clinical trial is a multinational, first-in-human trial designed to assess the safety, biological activity and preliminary efficacy of PM359 in adult and pediatric study participants. Initial study participants will be adults with stable disease.

If safety and biological activity are demonstrated in this cohort, the study is designed to enroll participants with active infection or severe inflammation as well as adolescent and pediatric participants. Participants will be followed for safety, including engraftment and reconstitution of the hematopoietic system, early biological markers of restored immune function, and long-term resolution and prevention of infectious and inflammatory complications of CGD. Prime Medicine expects to report initial clinical data from the study in 2025.