Editas Medicine shared new in vivo data on June 12, 2025, demonstrating therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells (HSCs) with a single dose of proprietary targeted lipid nanoparticle (tLNP) in non-human primates (NHPs). This data was presented at the European Hematology Association (EHA) 2025 Congress.
Latest data from an ongoing NHP study showed that at five months post-single intravenous administration, Editas' tLNP resulted in mean on-target editing levels in the HBG1/2 promoter region of 58% in HSCs. This level well exceeds the predicted editing threshold of ≥25% required for therapeutic benefit.
The biodistribution data in NHPs with Editas’ tLNP continued to show significant de-targeting of the liver in contrast to standard LNPs. These findings validate the further development of Editas’ proprietary HSC-tLNP for editing of the HBG1/2 promoters for the treatment of sickle cell disease and beta thalassemia.
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