Focus on in vivo CRISPR-edited medicines based on Editas researchers’ recent scientific progress in multiple tissues: Achieved pre-clinical in vivo proof of concept of high level HBG1/2 promoter editing and HbF induction in a humanized mouse model for treatment of sickle cell disease and beta thalassemia with a single dose of an HSC-targeted lipid nanoparticle (tLNP) formulation Achieved in vivo proof of concept of high... Read More