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 has presented promising preclinical proof-of-concept data for EDIT-401, a potential one-time therapy aimed at significantly reducing LDL-cholesterol levels, at the ESGCT Congress in Seville, Spain [1][3] Group 1: Efficacy Data - In preclinical non-human primate studies, EDIT-401 achieved a mean LDL-C reduction of ≥90% [3][7] - The therapy demonstrated robust efficacy within 48 hours of a single dose, with similar results observed in mice with high baseline LDL-C and reduced LDLR function [7] - A ≥6-fold mean increase in LDLR protein was noted in the liver of non-human primates, indicating effective upregulation [7] Group 2: Therapeutic Strategy - The therapeutic strategy utilizes CRISPR/Cas9 nuclease and dual gRNAs with LNP delivery to disrupt negative regulatory elements, enhancing mRNA stability and enabling potent LDLR upregulation [7] - The approach requires only a moderate level of functional editing of LDLR alleles to achieve significant outcomes [7] Group 3: Durability of Effect - The LDL-C reduction effect was maintained in mouse models over a three-month study period, suggesting durability of the treatment [7] Group 4: Company Overview - Editas Medicine focuses on developing transformative in vivo gene editing medicines using CRISPR technology for serious diseases [6] - The company holds exclusive licenses for the Cas12a and Cas9 patent estates from the Broad Institute and Harvard University for human medicines [6]