EDIT-401 achieved ~90% mean LDL-C reduction with single dose in non-human primatesEDIT-401 on track for human proof-of-concept data by end of 2026Strong cash position with operational runway into second quarter of 2027Company-sponsored webinar on EDIT-401 today at 8:00 a.m. ETCAMBRIDGE, Mass., Sept. 02, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (NASDAQ:EDIT), a pioneering gene editing company, today announced the nomination of its lead in vivo development candidate, EDIT-401, an experimental, potential best-in-class, one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels."Today's nomination of EDIT-401 as our lead in vivo development candidate is a significant milestone in our long-term vision to lead the field of in vivo programmable gene editing," said Gilmore O'Neill, M.B., M.M.Sc., President and Chief Executive Officer of Editas Medicine. "At Editas Medicine, we believe in vivo is the future of medicine. With EDIT-401, we're advancing our differentiated upregulation strategy to develop a next-generation medicine that can deliver lasting impact for patients."About EDIT-401EDIT-401 is an experimental, potentially transformative in vivo gene editing medicine, based on Editas' differentiated upregulation approach. EDIT-401 is designed to treat hyperlipidemia by directly editing the LDLR gene to increase LDLR protein expression and reduce LDL-C levels. This targeted approach has demonstrated a favorable preclinical profile in both efficacy data and tolerability and supports the potential of EDIT-401 to deliver meaningful clinical outcomes for patients underserved by current lipid-lowering therapies. Key highlights of EDIT-401 include:Robust efficacy data with ~90 percent mean reduction of LDL-C in preclinical non-human primate studies, while standard of care therapies demonstrate a 40-60% mean reduction of LDL-CPotential one-time treatment designed for lifelong benefitSizeable market potential with favorable health care system economicsAttractive business model expected to align with typical biopharma marginsCompelling preclinical data supporting rapid progression to human proof-of-concept data by end of 2026About Hyperlipidemia and Atherosclerotic Cardiovascular Disease (ASCVD)Atherosclerotic cardiovascular disease (ASCVD) is the leading cause of death worldwide and imposes a significant burden on the US healthcare system with national expenditures projected to reach over $300 billion in 2035. Elevated LDL-C is a major causal factor in ASCVD disease. Elevated LDL-C, also known as hyperlipidemia, is a highly prevalent disease affecting over 70 million patients in the United States alone. Substantial unmet need exists across multiple at-risk segments of patients ...Full story available on Benzinga.com