Prime Medicine has been granted approval by the US Food and Drug Association (FDA) to initiate trials of the first-ever prime editing drug.

The FDA cleared Prime Medicine’s investigational new drug (IND) application for PM359 for the treatment of chronic granulomatous disease (CGD), allowing the company to initiate a Phase I/II trial in the US.

The company hopes to run sites globally but will need to receive appropriate clearance. Initial data is expected from the trial next year.

Prime Medicine CEO Keith Gottesdiener said: “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.

“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.”

The Phase I/II trial is a global, first-in-human trial designed to assess the safety, biological activity, and early signs of efficacy of PM359 in adult and paediatric patients with CGD.

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Initially, Prime Medicine will enrol adult patients with stable disease. If safety and biological activity are demonstrated in the first cohort, adult patients with active infection or severe inflammation as well as adolescent and paediatric participants will be enrolled.

Patients will be monitored for early biological markers of restored immune function and long-term resolution and prevention of infectious and inflammatory complications associated with CGD.

PM359 is the first-ever prime editing drug to receive IND clearance from the FDA. The drug comprises autologous blood stem cells modified ex vivo using prime editing to correct a high percentage of cells containing the disease-causing mutation.

PM359 also received rare paediatric drug designation and orphan drug designation from the FDA.

CGD landscape

CGD is a rare inherited haematologic disorder caused by mutations in the NADPH oxidase complex, which is required for phagocytic cells, in particular neutrophils, to destroy many invasive microorganisms.

The disease begins in childhood, with most children diagnosed within the first three years of life.

Patients with CGD develop infections from both typical and unusual bacteria, fungi, and mycobacteria. These infections may present in various organs and can lead to long-term organ damage and failure.

Patients can also develop non-infectious inflammatory disease, the most common being inflammatory bowel disease, soft tissue granulomas, and strictures of the urinary or digestive tract.

The disease can result in patients taking lifelong regimens of antibiotics and antifungals to prevent infections.

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