Prime Medicine, Inc., a biotechnology company, engages in delivering genetic medicines to address the spectrum of diseases by deploying gene editing technology in the United States. Its lead therapeutic product candidate is PM359 for Chronic Granulomatous Disease, which is in Phase 1/2 clinical trial; PM577 for Wilson Disease, that is in preclinical studies; and PM647 to treat genetic disorder caused by mutations in the SERPINA1 gene, resulting in production of misfolded Z-AAT protein that accumulates in the liver and leads to hepatocellular injury, cirrhosis, and increased risk of hepatocellular carcinoma. The company also provides vivo programs targeting diseases of the liver, and Cystic Fibrosis programs. In addition, it offers Prime Editing technology comprising a programmable DNA binding domains, such as Cas domains, are typically modified such that they do not cause a double-stranded break in the DNA, as well as a RT domain that copies the edited DNA sequence directly into the target genomic site where the edit is made; and pegRNA which contains a search sequence, also known as a spacer, which provides a target genomic address for the Prime Editor. The company has a research collaboration and license agreement with BMS; Cystic Fibrosis Foundation; Broad Institute; and Beam. Prime Medicine, Inc. was incorporated in 2019 and is headquartered in Cambridge, Massachusetts.
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