edit-401

**EDIT-401** is an experimental CRISPR-based gene therapy developed by Editas Medicine for the treatment of hyperlipidemia (elevated LDL cholesterol, LDL-C). EDIT-401 utilizes CRISPR/Cas9 and dual guide RNAs delivered via lipid nanoparticles to precisely disrupt negative regulatory elements in the 3' UTR of the LDL receptor (LDLR) gene, thereby increasing LDLR mRNA stability and upregulating LDLR protein expression in hepatocytes. In preclinical studies, a single dose of EDIT-401 demonstrated highly potent (≥90% mean reduction) and durable LDL-C lowering in both non-human primates and mice within 48 hours, with effects maintained over at least three months in mice. This approach is designed to be a one-time, potentially life-long therapy for refractory hypercholesterolemia, significantly outperforming current standard-of-care lipid-lowering drugs[1][2][3].