Nature Medicine, Published online: 03 March 2026; doi:10.1038/s41591-026-04220-0 A phase 1 study demonstrates proof-of-concept of liver-targeted PCSK9 base editing for familial hypercholesterolemia, but turning this into a transformative therapy will require stringent optimization of editing efficiency, safety, patient selection and trial design.
Nature Medicine, Published online: 03 March 2026;
doi:10.1038/s41591-026-04220-0
A phase 1 study demonstrates proof-of-concept of liver-targeted PCSK9 base editing for familial hypercholesterolemia, but turning this into a transformative therapy will require stringent optimization of editing efficiency, safety, patient selection and trial design.
— Source: Nature Medicine (https://www.nature.com/articles/s41591-026-04220-0)