Editas Medicine Reports In Vivo Proof-of-Concept Data for EDIT-401 at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress

Core Insights - Editas Medicine presented preclinical proof-of-concept data for EDIT-401, demonstrating a significant potential to reduce LDL-cholesterol (LDL-C) by ≥90% in non-human primates and mouse models [1][3]. Group 1: Efficacy Data - In vivo studies showed robust efficacy with a ≥90% mean LDL-C reduction achieved within 48 hours of a single dose of EDIT-401 in non-human primates [7]. - The therapy also resulted in a ≥90% LDL-C reduction in mice with high baseline LDL-C and reduced LDL receptor (LDLR) function [7]. - A mean increase of ≥6-fold in LDLR protein levels was observed in the liver of non-human primates, indicating effective upregulation [7]. Group 2: Mechanism of Action - The therapeutic strategy utilizes CRISPR/Cas9 nuclease and dual gRNAs with lipid nanoparticle (LNP) delivery to disrupt negative regulatory elements in the 3' UTR, enhancing mRNA stability and promoting LDLR upregulation [7]. Group 3: Durability of Effect - The LDL-C reduction effect was maintained in mouse models over a three-month study period, suggesting the durability of the treatment [7]. Group 4: Company Overview - Editas Medicine is focused on developing transformative in vivo gene editing medicines using CRISPR technology for serious diseases, holding exclusive licenses for key CRISPR patents [6].