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Prime Medicine Receives U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for PM359 for the Treatment of Chronic Granulomatous Disease (CGD)

PRME
  • Prime Medicine announced that the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to PM359, an investigational autologous Prime Edited hematopoietic stem cell therapy for p47 phox-deficient chronic granulomatous disease (CGD).
  • The RMAT designation was based on Phase 1/2 clinical data, including results previously published in The New England Journal of Medicine, demonstrating the therapy's potential to address unmet medical needs.
  • PM359 now holds RMAT, Fast Track, Orphan Drug, & Rare Pediatric Disease Designations, enabling intensive FDA engagement & eligibility for rolling & priority Biologics License Application review.
  • Clinical trial results reported in December 2025 showed that a single dose of PM359 drove rapid engraftment & durable restoration of immune cell function with a safety profile consistent with busulfan-based conditioning alone.
  • PM359 is designed to correct the delGT mutation in the NCF1 gene, which causes the most prevalent form of p47 phox-deficient CGD, in an ongoing multinational Phase 1/2 first-in-human trial.
  • Current standard of care for CGD relies on lifelong antimicrobial prophylaxis or allogeneic hematopoietic stem cell transplantation, which carries significant morbidity & is not accessible to all patients.
  • Prime Medicine is advancing PM359 toward potential approval while expanding its broader pipeline to include programs for Wilson Disease & Alpha-1 Antitrypsin Deficiency.