Prime Medicine, Inc. is an American biotechnology company developing gene therapies based on prime editing. The company is headquartered in Cambridge, Massachusetts.[1]
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
- World first: ultra-powerful CRISPR treatment trialled in a person Nature^
- Karen O'Hanlon Cohrt. First-Ever Prime-Editing Therapy Shows Safety and Efficacy in Patient With Chronic Granulomatous Disease CRISPR Medicine News^
- A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD ) ClinicalTrials.gov^
- Prime Editing Shows Promise in Chronic Disease Treatment Clinical Trial Vanguard^
- Prime Editing Clinical Results Published for the First Time Ever Inside Precision Medicine^
- Prime Editing for p47phox-Deficient Chronic Granulomatous Disease New England Journal of Medicine^
- 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^
- Cystic Fibrosis Foundation Invests up to $15 Million in Prime Medicine Global Genes^
- Cystic Fibrosis Foundation grants new funding to advance development of Prime Medicine’s prime editors BioWorld^
- BMS pays $110M to form T-cell therapy pact, helping Prime buy time to advance prioritized pipeline Fierce Biotech^