Prime Medicine, Inc.

We are a biotechnology company focused on developing genetic medicines for diseases caused by genetic alterations, cellular dysfunction, or dysregulated gene expression. Our approach uses Prime Editing, a gene editing technology that modifies genomic DNA without creating double-stranded breaks. We believe Prime Editing is a versatile and precise method for rewriting, replacing, or repairing DNA sequences, potentially addressing a broad range of diseases with improved safety and specificity compared to older editing technologies. We are developing wholly owned in vivo programs for liver diseases and Cystic Fibrosis (CF), with support from the Cystic Fibrosis Foundation (CFF). We also have partnered ex vivo programs with Bristol-Myers Squibb (BMS), supported by our modular delivery and Prime Editor platform. Early clinical data from our ex vivo program, PM359 for CGD, showed restored functional protein activity in treated patients, providing clinical validation of Prime Editing and a potentially curative treatment for CGD patients. Our initial development priorities include genetic diseases affecting the liver and lung, and hematologic disorders addressed through ex vivo editing. Our lead program, PM577 for Wilson Disease, aims to correct the H1069Q mutation in the ATP7B gene, supported by preclinical data showing targeted correction and restored copper homeostasis in animal models. We are also developing follow-on Prime Editors for additional ATP7B mutations. Additionally, PM647 is being advanced for alpha-1 antitrypsin deficiency (AATD), another liver-targeted disease.