Editas Medicine reports new in vivo proof of concept data for liver target

Editas Medicine (EDIT) shared in vivo proof of concept data supporting the development of a potentially first-in-class treatment for an undisclosed liver target in a poster presentation at the 28th Annual Meeting of the American Society of Gene and Cell Therapy in New Orleans. Key findings include: An in vivo dose-response study in a disease-specific mouse model utilizing LNPs to deliver CRISPR/Cas-based cargo demonstrated maximal liver editing of the target gene and resulted in robust target protein upregulation with greater than80% disease biomarker reduction. Editing and subsequent upregulated expression of the target gene in cynomolgus monkey hepatocytes treated with CRISPR/Cas-based editing cargo also achieved greater than50% target gene editing and greater than15-fold protein upregulation.