Prime Medicine

Last updated
Prime Medicine, Inc.
Company type Public
ISIN US74168J1016
Industry Biotechnology
Founded2019;7 years ago (2019)
Founders
Headquarters Cambridge, Massachusetts, U.S.
Key people
  • Allan Reine (CEO)
  • David R. Liu (Co-founder and Scientific Advisory Board member)
Website primemedicine.com

Prime Medicine, Inc. is an American biotechnology company developing gene therapies based on prime editing. The company is headquartered in Cambridge, Massachusetts. [1]

Contents

Pipeline

In May 2024, Prime Medicine announced that the U.S. FDA had accepted its IND application for PM359, a gene therapy involving transplantation of autologous CD34+ stem cells modified by prime editing ex vivo . [2] PM359 also received Rare Pediatric Disease and Orphan Drug designations from the FDA. A Phase 1/2 open-label clinical trial of PM359 in patients with chronic granulomatous disease (CGD) caused by mutations in the neutrophil cytosolic factor 1 (NCF1, also known as p47phox) gene began in October 2024, making PM359 the first prime editor to enter the clinic. [3] Following an announcement of preliminary results in May 2025, [4] Prime Medicine and its clinical collaborators reported in December 2025 that two CGD patients treated with PM359 had been "effectively cured" [5] of the disease. [6]

In May 2025, Prime Medicine announced that it would prioritize clinical development programs for the treatment of Wilson's disease and alpha-1 antitrypsin deficiency (AATD), two of the most common genetic liver diseases. [7]

Partnerships

In January 2024, Prime Medicine announced a partnership with the Cystic Fibrosis Foundation, with Prime Medicine receiving $15 million in funding towards the development of prime editing-based therapies for cystic fibrosis. [8] An additional $24 million commitment from the foundation was announced in July 2025. [9]

In September 2024, Prime Medicine announced a partnership with Bristol Myers Squibb to develop optimized prime editing reagents for ex vivo T cell therapies. Prime Medicine received $110 million upfront and is eligible for up to $3.5 billion in milestone payments. [10]

See also

References

  1. "World first: ultra-powerful CRISPR treatment trialled in a person". Nature.
  2. Karen O'Hanlon Cohrt. "First-Ever Prime-Editing Therapy Shows Safety and Efficacy in Patient With Chronic Granulomatous Disease". CRISPR Medicine News.
  3. "A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )". ClinicalTrials.gov.
  4. "Prime Editing Shows Promise in Chronic Disease Treatment". Clinical Trial Vanguard.
  5. "Prime Editing Clinical Results Published for the First Time Ever". Inside Precision Medicine.
  6. "Prime Editing for p47phox-Deficient Chronic Granulomatous Disease". New England Journal of Medicine.
  7. "Prime Medicine Announces Strategic Restructuring to Focus on Opportunities in Large Genetic Liver Diseases, Cystic Fibrosis, and Partnered Programs Alongside CEO Leadership Transition". Prime Medicine.
  8. "Cystic Fibrosis Foundation Invests up to $15 Million in Prime Medicine". Global Genes.
  9. "Cystic Fibrosis Foundation grants new funding to advance development of Prime Medicine's prime editors". BioWorld.
  10. "BMS pays $110M to form T-cell therapy pact, helping Prime buy time to advance prioritized pipeline". Fierce Biotech.