Editas Medicine revealed EDIT-401 as its lead in vivo development candidate, an innovative gene editing therapy designed to reduce LDL cholesterol by increasing LDL receptor expression through LDLR gene editing. Preclinical studies reported a 90% LDL-C reduction in non-human primates with a favorable efficacy and safety profile. This strategic pivot from rare diseases targets the large atherosclerotic cardiovascular disease market anticipated to exceed $300 billion in the U.S. by 2035, with clinical filings expected in mid-2026.