Clinical TrialMay 26, 2026, 06:02 AM
Editas Medicine's EDIT-401 shows ~90% reduction in LDL-C, Lp(a), ApoB
AI Summary
Editas Medicine presented new preclinical data for EDIT-401, its lead in vivo development candidate, showing robust reductions of approximately 90% in LDL-cholesterol (LDL-C), lipoprotein(a) (Lp(a)), and apolipoprotein B (ApoB) in non-human primates. These results support its potential as a best-in-class medicine for hyperlipidemia through LDLR upregulation. The company plans to submit a Clinical Trial Notification (CTN) by mid-2026 to initiate a first-in-human clinical trial in Australia for patients with Heterozygous Familial Hypercholesterolemia (HeFH), with early human proof-of-concept data anticipated by the end of 2026.
Key Highlights
- Single dose of EDIT-401 achieved ~90% mean reduction in LDL-C in non-human primates.
- EDIT-401 achieved ~90% mean reduction in Lp(a) in non-human primates.
- EDIT-401 achieved ~90% mean reduction in ApoB in non-human primates.
- Reductions in LDL-C, Lp(a), and ApoB were highly correlated, supporting LDLR upregulation.
- EDIT-401 was well-tolerated in non-GLP toxicology at a therapeutically relevant dose of 1.5 mg/kg.
- Company plans to submit a Clinical Trial Notification (CTN) by mid-2026.
- Early in vivo human proof-of-concept data for EDIT-401 is expected by the end of 2026.
Price Impact
More from EDIT