Editas Medicine (EDIT) announced its achievement of in vivo preclinical proof of concept of hematopoietic stem and progenitor cell, HSPC, editing and fetal hemoglobin induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells. The Company observed high levels of editing of the HBG1/2 promoter, leveraging our clinically validated upregulation strategy, utilizing a novel and Editas-proprietary targeted lipid nanoparticle, tLNP, formulation for extrahepatic tissue delivery. The Company is also providing business development and financial updates, including that it initiated a process to partner or out-license reni-cel. Ex Vivo Hemoglobinopathies: Reni-cel for Severe Sickle Cell Disease – On-track to present a substantive clinical data set of sickle cell patients with considerable clinical follow-up in the RUBY trial at the American Society of Hematology Annual Meeting and Exposition, December 7-10, 2024. Reni-cel for Transfusion-dependent Beta Thalassemia: On-track to present additional clinical data from the EdiTHAL trial by year-end 2024.
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