FDA Grants RMAT Designation To Prime's PM359 For Treating CGD Based On Phase 1/2 Data

India, June 22 -- Prime Medicines Inc. (PRME), a biotechnology company, on Monday announced the receipt of a Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for PM359, a hematopoietic stem cell therapy for p47phox deficiency chronic granulomatous disease.

Chronic granulomatous disease (CGD) is a rare inherited hematologic disorder characterized by susceptibility to severe infections alongside inflammatory and autoimmune complications. The disease affects the production of functional neutrophils and is diagnosed with a frequency of about one in every 100,000-200,000 individuals in the U.S. alone with the p47phox accounting for 25% of all cases.

PM359 is an investigational autologous hematopoietic stem cell thera...