Project UpdateMay 14, 2026, 06:21 AM
Editas Medicine EDIT-401 Shows >=90% LDL-C Reduction in Preclinical Data
AI Summary
Editas Medicine reported new preclinical data for its lead candidate, EDIT-401, at the ASGCT 2026 Annual Meeting. A single dose of EDIT-401 achieved a 90% or greater mean LDL cholesterol reduction in non-human primates, with durable effects for approximately six months and a promising safety profile. The data supports EDIT-401's potential as a one-time treatment for hyperlipidemia and its advancement towards clinical development, also highlighting the broader potential of the company's upregulation strategy.
Key Highlights
- EDIT-401 achieved >=90% mean LDL-C reduction in non-human primates.
- LDL-C lowering was durable for ~6 months across 1.5-3.0 mg/kg doses.
- Reduction achieved with 10-40% functional LDLR allele editing.
- Showed >=6-fold mean increase in hepatic LDLR protein.
- Promising preclinical safety profile at 1.5 mg/kg dose.
- Highest delivery in hepatocytes; undetectable oocyte delivery.
- Data suggests no dose adjustments needed for HeFH patients.