Editas Medicine, Inc. announced on October 8, 2026, that it has received approval to initiate the Phase 1/2 Strive clinical trial of EDIT-401 in patients with heterozygous familial hypercholesterolemia (HeFH). The trial has received Human Research Ethics Committee approval and completed the Clinical Trial Notification process with Australia’s Therapeutic Goods Administration. Additionally, the trial has been submitted for review in New Zealand, with plans to enroll patients in both countries if approved.
EDIT-401 is an experimental, potentially best-in-class in vivo gene editing medicine designed to treat hyperlipidemia. The therapy utilizes Editas’ differentiated upregulation approach to directly edit the LDL receptor (LDLR) gene, aiming to increase LDLR protein expression and reduce LDL-cholesterol (LDL-C) levels. In preclinical studies involving non-human primates, EDIT-401 demonstrated approximately 90 percent or greater mean reduction in multiple atherogenic lipoproteins, including LDL-C, lipoprotein(a), and apolipoprotein B. New preclinical data also indicated that an LDL-C reduction of approximately 90 percent was maintained over a 10-month period.
The Strive trial is designed to evaluate the safety, tolerability, and efficacy of a single dose of EDIT-401. The trial consists of two parts: Part 1 is a single ascending dose, dose-finding, open-label trial, and Part 2 is a single-dose randomized, placebo-controlled expansion study. The Company has selected five clinical trial sites across Australia and New Zealand. Editas expects to report initial safety and tolerability data in the first quarter of 2027 and plans to complete enrollment in Part 1 with topline safety and efficacy data available later in 2027.
Separately, the Company announced that new preclinical data demonstrating the durability of LDL-C reduction with EDIT-401 will be presented at the American Heart Association (AHA) Scientific Sessions 2026, scheduled for November 6-9, 2026, in Chicago, Illinois. The presentation is scheduled for Sunday, November 8, 2026, from 3:30 to 4:45 p.m. CT.
