Editas Medicine reported new preclinical data at ASGCT 2026 showing EDIT-401 achieved ≥90% mean LDL-C reduction with durable effect through ~6 months in non-human primates, supporting advancement toward first-in-human trials.
Key takeaways
Single dose of EDIT-401 achieved ≥90% mean LDL-C reduction across all dose groups (1.5-3.0 mg/kg) in non-human primates.
LDL-C reduction durable through ~6 months with 10-40% functional editing of LDLR alleles and ≥6-fold hepatic LDLR protein increase.
Promising safety profile: no adverse observations at 1.5 mg/kg dose; highest delivery in hepatocytes, undetectable in oocytes.
EDIT-401(mu) data in mice supports no dose adjustment needed for HeFH patients.
Data presented in oral and poster sessions at ASGCT and TIDES USA 2026 conferences.
Strengthens confidence in EDIT-401 as potential one-time treatment for hyperlipidemia.