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**LNP-168-ABE8e-HBG** is an investigational, antibody-free bone-marrow-targeted lipid nanoparticle gene-editing therapy developed by YolTech. The LNP uses the ionizable lipid Lipid-168 to intravenously deliver mRNA encoding the adenine base editor ABE8e together with sgRNA-25 to hematopoietic stem and progenitor cells. The guide directs base editing of the HBG1 and HBG2 promoter region, increasing gamma-globin and fetal hemoglobin expression to restore globin-chain balance. It is being developed as a potential one-time in vivo treatment for transfusion-dependent beta-thalassemia and sickle cell disease without ex vivo cell collection, myeloablative conditioning, or hematopoietic stem-cell transplantation.
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