EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)

Authors
Category Primary study
Registry of TrialsClinicalTrials.gov
Year 2026
A phase 1/2, two-part trial consisting of an open label dose-escalation part and a randomized, double blind, placebo-controlled part to investigate safety, tolerability, pharmacokinetics, and efficacy with EDIT-401 gene editing in adult male and female participants 18 to 65 years of age who require additional lowering of low-density lipoprotein cholesterol (LDL-C) such as those with clinical diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH).
Epistemonikos ID: 93a76181eca6ebdad5c08e569cfcdaa13dddd049
First added on: Oct 27, 2026