Editas Medicine Reports New In Vivo Proof of Concept Data in an Undisclosed Liver Target at the American Society of Gene and Cell Therapy Annual Meeting

Core Insights - Editas Medicine has presented in vivo proof of concept data for a potential first-in-class treatment targeting an undisclosed liver condition, showcasing significant advancements in gene editing technology [1][4]. Group 1: In Vivo Editing Strategy - The company utilized lipid nanoparticles (LNPs) to deliver CRISPR/Cas RNA cargo for editing a liver target gene, resulting in the upregulation of the target gene and a meaningful reduction in disease-specific biomarkers in mice [2][7]. - A dose-response study demonstrated approximately 70% editing of the target gene in a disease-specific mouse model, leading to over 80% reduction in disease biomarkers [7]. Group 2: Presentation Details - Editas Medicine will present its findings in a poster session at the 28th Annual Meeting of the American Society of Gene and Cell Therapy on May 14, 2025, with additional data to be shared at the TIDES USA 2025 conference on May 21 [5][4]. Group 3: Future Developments - The company is making significant progress towards clinical applications and plans to disclose the specific disease target and development candidate later this year [4].