Editas Medicine(EDIT)2024-11-04 12:33Clinical Development - Achieved in vivo preclinical proof of concept for hematopoietic stem and progenitor cell editing, demonstrating a 29% editing level in HSPCs after a single dose[3] - Induced fetal hemoglobin (HbF) expression in human red blood cells, with an average of 20% HbF expressing cells present one month post-treatment[3] - Reni-cel (renizgamglogene autogedtemcel) is on track to present clinical data at the ASH Annual Meeting on December 7-10, 2024, following the completion of enrollment for adolescent and adult cohorts in the RUBY trial[4] - Completed enrollment of the adult cohort in the EdiTHAL trial for transfusion-dependent beta thalassemia, with additional clinical data expected by year-end 2024[5] Financial Performance - Sold future license fees under the Cas9 license agreement to DRI Healthcare Trust for an upfront cash payment of 265 million in cash, cash equivalents, and marketable securities, increasing to approximately $320 million post-receipt of DRI payment[6] Strategic Partnerships - Engaged Moelis & Company LLC to lead the global process for partnering or out-licensing reni-cel, aiming to reduce spending in 2025[6] - Focused on optimal capital use while advancing the in vivo pipeline and evaluating opportunities for reni-cel commercialization[2] Technology and Innovation - The proprietary targeted lipid nanoparticle (tLNP) formulation is key for extrahepatic tissue delivery, positioning the company for leadership in in vivo programmable gene editing[2] - The company aims to deliver transformative genomic medicines for serious diseases, leveraging CRISPR/Cas12a and Cas9 genome editing systems[8]