Prime Medicine, a biotechnology company committed to delivering a new class of differentiated, one-time curative genetic therapies, today 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.
“We are thrilled to achieve this important milestone for our first product candidate, PM359, which represents the first-ever IND clearance for a Prime Editor product candidate and a significant advancement in the field of next-generation gene editing,” said Keith Gottesdiener, M.D., President and Chief Executive Officer of Prime Medicine. “Based on data from our preclinical studies, we believe PM359 has the potential to sufficiently correct a prevalent disease-causing mutation of CGD, leading to amelioration of disease for these patients. We look forward to the planned initiation of our Phase 1/2 trial and further determining the potential therapeutic impact of PM359 in patients with this devastating disease.”
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.
👉 Prime Medicine Announces FDA Clearance of Investigational New Drug (IND) Application for PM359 for the Treatment of Chronic Granulomatous Disease (CGD) – Prime Medicine, Inc.
