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

Brief summary

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).

Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.

Trial details

status

NOT_YET_RECRUITING

phase

PHASE1, PHASE2

study type

INTERVENTIONAL

enrollment

56

sponsor

Editas Medicine, Inc.

conditions

Heterozygous Familial Hypercholesterolemia (HeFH)

interventions

DRUG: Drug: EDIT-401; OTHER: Placebo

start date

2026-11-01

start date type

ESTIMATED

primary completion date

2029-06-01

has results

false

data source

ClinicalTrials.gov

data processed

2026-10-29T09:00:05

modifications

selected fields only; whitespace normalized; brief summary truncated to 2,000 characters