LNP-028-ABE8e-HBG

**LNP-028-ABE8e-HBG** is a preclinical, non-viral in vivo hematopoietic stem-cell gene-editing therapy developed using an antibody-free lipid nanoparticle platform. The LNP-028 formulation co-delivers mRNA encoding the adenine base editor ABE8e and a single-guide RNA targeting the **HBG1/2 gamma-globin promoter** to human bone-marrow hematopoietic stem cells after intravenous administration. Base editing of the HBG promoter reactivates fetal hemoglobin expression in erythroid progeny, with the goal of correcting globin-chain imbalance in transfusion-dependent beta-thalassemia and increasing fetal hemoglobin in sickle cell disease. The program has been demonstrated in humanized immunodeficient mouse models using human hematopoietic stem cells and remains preclinical. ([nature.com](https://www.nature.com/articles/s41551-025-01480-y))