Editas Medicine Reports Proprietary Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for Sickle Cell Disease and Beta Thalassemia at the European Hematology Association 2025 Congress in June

Core Insights - Editas Medicine has demonstrated a mean editing level of 58% in hematopoietic stem cells (HSCs) after a single dose of their proprietary targeted lipid nanoparticle (tLNP), indicating a significant advancement in gene editing for sickle cell disease and beta thalassemia [1][3][4] Company Overview - Editas Medicine is a pioneering gene editing company focused on developing in vivo medicines using CRISPR/Cas12a and CRISPR/Cas9 technologies for serious diseases [7] Research and Development - The company’s in vivo HSC program targets HBG1/2 promoters to increase fetal hemoglobin (HbF) levels, utilizing proprietary AsCas12a for high efficiency and reduced off-target effects [5] - The recent study in non-human primates (NHPs) showed that a single intravenous administration of tLNP resulted in on-target editing levels exceeding the therapeutic threshold of 25% [3][4] Presentation and Data Sharing - Editas Medicine will present these findings at the European Hematology Association (EHA) 2025 Congress in Milan, Italy, on June 14, 2025 [2][7]