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Editas Medicine Reports Promising in Vivo Gene Editing Data for Sickle Cell Disease and Beta Thalassemia Treatment
ByAinvest
Thursday, Jun 12, 2025 11:05 am ET1min read
Editas Medicine reported new in vivo data showing therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells in non-human primates using a single dose of its proprietary targeted lipid nanoparticle. The data demonstrated a mean on-target editing level of 58% in stem cells, exceeding the predicted editing threshold for therapeutic benefit. This approach is being developed to treat sickle cell disease and beta thalassemia.
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