Prime Medicine
From Wikipedia, the free encyclopedia
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Company type | Public |
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| 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]
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]