Editas Medicine Inc., a pioneering gene editing company, has announced new _in vivo_ data showcasing the potential of their gene upregulation strategy in hematopoietic stem cells (HSCs). The data, presented at the American Society of Gene and Cell Therapy Annual Meeting, revealed therapeutically relevant levels of _HBG1/2_ promoter editing achieved with a single dose of proprietary targeted lipid nanoparticles in humanized mice and non-human primates. This clinically validated approach aims to upregulate fetal hemoglobin (HbF) and is in pre-clinical development as a novel treatment for sickle cell disease and beta thalassemia. The results highlight the potential of Editas' strategy to address significant unmet medical needs in these diseases.
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