Editas Medicine shared new in vivo data on May 14, 2025, demonstrating therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells (HSCs) at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). This was achieved with a single dose of proprietary targeted lipid nanoparticle (tLNP) in humanized mice and non-human primates (NHPs).
In an ongoing NHP study, a single intravenous dose of Editas Medicine’s proprietary tLNP achieved up to 47% HBG1/2 editing levels in HSCs. A study in humanized mice showed 48% editing of HBG1/2 in long-term HSCs after a single dose.
Both studies exceeded the predicted editing threshold of ≥25% required for therapeutic benefit, supporting the development of this approach as a potential transformative in vivo gene editing medicine for sickle cell disease and beta thalassemia. Preliminary NHP biodistribution data also showed significant de-targeting of the liver compared to standard LNPs.
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