Editas clears Australian requirements for first-in-human EDIT-401 cholesterol trial
Published in Biology, Drugs development & discovery.
October 8, 2026 — Editas Medicine announced that its phase 1/2 Strive trial of EDIT-401 received Australian ethics approval and completed the Therapeutic Goods Administration’s Clinical Trial Notification process. The study targets heterozygous familial hypercholesterolemia and remains under review in New Zealand.
EDIT-401 is an experimental in vivo gene-editing medicine designed to increase LDL receptor expression by editing the LDLR gene, with the aim of lowering LDL cholesterol.
The trial will evaluate a single dose. Its initial open-label portion will explore ascending doses, followed by a randomized, placebo-controlled expansion. Editas expects patient dosing to begin in 2026 and initial safety and tolerability results in the first quarter of 2027; these remain projected milestones.
The company also reports that approximately 90% LDL cholesterol reduction persisted for ten months in an ongoing non-human-primate study, with more data planned for November 8 at the American Heart Association meeting. Those animal findings do not establish human efficacy, long-term safety or cardiovascular benefit. Today’s announcement authorizes research initiation, rather than marketing the treatment.
Source: Editas Medicine announcement, October 8, 2026.