Prime Medicine presents prime editing data in primates for GSD1b

Prime Medicine reported new preclinical data demonstrating the ability of liver-targeted Prime Editors to efficiently and precisely correct one of the most prevalent disease-causing mutations of glycogen storage disease 1b in non-human primates – NHP – and mouse models. GSD1b is a rare, serious progressive disease that causes impaired glycogen metabolism and affects approximately 1,500 patients. To address the underlying genetic cause of GSD1b, Prime Medicine is advancing Prime Editors that are delivered to the liver by single intravenous infusion and designed to enable a precise correction of the disease-causing mutations. Prime researchers identified pegRNAs that precisely corrected the p.L348fs and p.G339C mutations in liver cells, which were then evaluated in vitro, demonstrating average editing of 77% and 37%, respectively.These findings provide important proof-of-concept for Prime Medicine’s LNP liver-targeted delivery approach, and support the further advancement of the company’s Prime Editors targeting the p.L348fs and p.G339C mutations in GSD1b.