Prime Medicine
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| Type | Public |
|---|---|
| ISIN | US74168J1016 |
| Industry | Biotechnology |
| Founded | 2019 |
| Founders |
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| Headquarters | Cambridge, Massachusetts, U.S. |
Key people |
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| Website | primemedicine |
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][3] 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.[4] Following an announcement of preliminary results in May 2025,[5] Prime Medicine and its clinical collaborators reported in December 2025 that two CGD patients treated with PM359 had been "effectively cured" of the disease.[6][7]
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.[8]
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.[9] An additional $24 million commitment from the foundation was announced in July 2025.[10]
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.[11]
See also
References
- ↑ Ledford, Heidi (19 May 2025). "World first: ultra-powerful CRISPR treatment trialled in a person". Nature. doi:10.1038/d41586-025-01593-z.
- ↑ Karen O'Hanlon Cohrt. "First-Ever Prime-Editing Therapy Shows Safety and Efficacy in Patient With Chronic Granulomatous Disease". CRISPR Medicine News.
- ↑ "News: The FDA Has Cleared the First Clinical Trial Application for a Prime Editor". CRISPR Medicine. Retrieved 2026-05-05.
- ↑ "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 for p47phox-Deficient Chronic Granulomatous Disease". New England Journal of Medicine.
- ↑ "Prime Editing Clinical Results Published for the First Time Ever". Inside Precision 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.
- ↑ Taylor, Nick Paul (30 Sep 2024). "BMS pays $110M to form T-cell therapy pact, helping Prime buy time to advance prioritized pipeline". Fierce Biotech.