Nature Medicine, Published online: 03 March 2026; doi:10.1038/s41591-026-04254-4 In a phase 1 trial enrolling six patients with heterozygous familial hypercholesterolemia, treatment with lipid nanoparticles designed for base editing and inactivation of PCSK9 in hepatocytes showed preliminary evidence of reduced low-density lipoprotein levels without the occurrence of serious adverse events or evidence of off-target editing.
Nature Medicine, Published online: 03 March 2026;
doi:10.1038/s41591-026-04254-4
In a phase 1 trial enrolling six patients with heterozygous familial hypercholesterolemia, treatment with lipid nanoparticles designed for base editing and inactivation of PCSK9 in hepatocytes showed preliminary evidence of reduced low-density lipoprotein levels without the occurrence of serious adverse events or evidence of off-target editing.
— Source: Nature Medicine (https://www.nature.com/articles/s41591-026-04254-4)