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

Core Insights - Editas Medicine is transitioning to focus exclusively on in vivo CRISPR-edited medicines, driven by recent scientific advancements that have accelerated timelines for viability in this area [2][3] - The company has achieved significant pre-clinical proof of concept in multiple tissues, including a ~40% editing of the HBG1/2 promoter site in hematopoietic stem cells (HSCs) and high efficiency editing in the liver of non-human primates [3][4] - Editas plans to extend its cash runway into Q2 2027 through cost-saving measures, including a reduction of approximately 65% of its workforce [6][10] Scientific Progress - The company has successfully induced HbF expression in human red blood cells, with an average of 20% HbF expressing cells observed one month post-treatment in mice [3] - The proprietary targeted lipid nanoparticle (tLNP) formulation was utilized for effective delivery of gene editing machinery to HSCs [3] - High efficiency editing in the liver was achieved in collaboration with Genevant, with further data expected to be shared in Q1 2025 [4] Strategic Changes - Editas is discontinuing the development of reni-cel due to the inability to secure a commercial partner, and will focus on the RUBY and EdiTHAL trials for sickle cell disease and beta thalassemia [1][8] - The company is implementing a significant workforce reduction, which will include departures from the management team [6][7] - The transition to a fully in vivo company is aimed at expanding therapeutic possibilities and positioning Editas as a leader in the CRISPR gene editing field [2][5]