Editas Medicine Announces Strategic Transition to in vivo Gene Editing Company with Intent to Achieve Human Proof of Concept in Approximately Two Years

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) formulationAchieved in vivo proof of concept of high efficiency editing in the liver in non-human primates Ending development of reni-cel afte ...