AAV gene therapy for homozygous familial hypercholesterolemia: a phase 1 trial

Nature Medicine, Published online: 04 June 2026; doi:10.1038/s41591-026-04441-3

After preclinical testing, a phase 1 trial involving three individuals with familial hypercholesterolemia demonstrated preliminary evidence for the safety and efficacy of AAV8-mediated LDL receptor gene therapy.

Source: www.nature.com