PRME

Prime Medicine, Inc. is a biotechnology company focused on developing genetic medicines designed to provide durable, and potentially curative, treatment options for patients with diseases driven by defined genetic alterations, acquired cellular dysfunction, or dysregulated gene expression. Its gene editing technology enables targeted modifications to genomic deoxyribonucleic acid (DNA) without introducing double-stranded breaks. The Company pipeline includes wholly owned in vivo programs targeting liver genetic diseases Wilson Disease, Alpha-1 Antitrypsin Deficiency (AATD) as well as early-stage discovery efforts in Cystic Fibrosis (CF). The PM359 is an ex vivo prime editing program designed to correct the GT mutation in the NCF1 gene, the most common cause of Chronic Granulomatous Disease (CGD). This program provides validation of prime editing in human cells. The Wilson Disease is a rare autosomal recessive disorder caused by loss-of-function mutations in ATP7B.