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

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).

Key inclusion Criteria: 1. Clinical diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH) 2. Low-density lipoprotein cholesterol (LDL-C) ≥ 70 and ≤ 300 mg/dL (≥ 1.8 mmol/L and ≤ 7.8 mmol/L) 3. Treatment including two (2) or more approved Lipid Lowering Therapies 4. Participant weight \> 40 kg and ≤ 110 kg Key Exclusion Criteria: 1. Any known history of Homozygous Familial Hypercholesterolemia (HoFH) or lipid apheresis 2. Evidence of an unstable or acute cardiovascular disorder 3. Any history of Diabetes Mellitus (DM): 1. Part 1: Type 1 and Type 2 DM 2. Part 2: Type 1 DM 4. Active or chronic history of liver disease 5. Reduced renal function