Editas Medicine reports in vivo preclinical PoC data for EDIT-401

Editas Medicine (EDIT) reported in vivo preclinical proof-of-concept data for EDIT-401, an experimental, one-time therapy to significantly reduce LDL-cholesterol, at the 32nd Annual European Society of Gene and Cell Therapy Congress in Seville, Spain. The Company shared results from preclinical studies demonstrating potent and durable reductions in LDL-C through upregulation of the LDL receptor. Key EDIT-401 Data Presented includes: Robust efficacy data: greater than or equal to90% LDL-C reduction in non-human primates achieved within 48 hours of a single dose of EDIT-401; greater than or equal to90% LDL-C reduction in mice with high baseline LDL-C and reduced LDLR function Optimized therapeutic strategy: CRISPR/Cas9 nuclease and dual gRNAs with LNP delivery disrupt negative regulatory elements in the 3′ UTR, increasing mRNA stability enabling potent LDLR upregulation greater than or equal to6-fold mean increase in LDLR protein in the NHP liver, requiring only a moderate level of functional editing of LDLR alleles Durable effect: LDL-C reduction maintained in mouse models in a three-month study. “The in vivo proof-of-concept data presented today reinforce the potential impact of our differentiated upregulation strategy. In preclinical non-human primate studies, EDIT-401 achieved robust efficacy data with a greater than or equal to90% mean LDL-C reduction. These data strengthen our conviction that EDIT-401 represents a novel therapeutic approach with the potential to significantly improve outcomes for people living with high LDL cholesterol,” said CSO Linda Burkly.