Editas Medicine to Present Preclinical Data Demonstrating Progress in the Development of an in vivo Gene Editing Pipeline at the American Society of Gene and Cell Therapy Annual Meeting

Core Insights - Editas Medicine announced the acceptance of five abstracts for presentation at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), highlighting its advancements in in vivo gene editing medicines [1][2] Company Developments - The company is making significant progress in preclinical development of in vivo medicines aimed at serious diseases, with a focus on gene upregulation editing strategies [2] - Editas Medicine will present preclinical data that demonstrates its ability to increase protein levels to address diseases caused by genetic mutations [2][4] - The presentations will include proof of concept data from studies using targeted lipid nanoparticles (tLNPs) for delivering gene editing cargo to hematopoietic stem cells [4][5] Presentation Details - An oral presentation titled "In Vivo Delivery of HBG1/2 Promoter Editing Cargo to HSC of Humanized Mouse and Non-Human Primate with Lipid Nanoparticles" is scheduled for May 14, 2025 [3] - Multiple poster presentations will also take place, covering topics such as improved LNP targeting ligands and the design of modified guide RNAs for enhanced gene editing potency [3][5] Research Focus - The research emphasizes in vivo CRISPR editing capabilities, aiming to upregulate target protein expression and reduce disease-associated biomarkers in relevant animal models [4][5] - Editas Medicine is leveraging its exclusive licenses for CRISPR/Cas12a and Cas9 technologies to develop a robust pipeline of transformative gene editing medicines [6]