CAMBRIDGE, Mass. — October 8, 2026

Editas Medicine, Inc. announced approval to initiate its Phase 1/2 Strive clinical trial evaluating EDIT-401 in patients with heterozygous familial hypercholesterolemia (HeFH) who require additional LDL-cholesterol (LDL-C) reduction. The trial received Human Research Ethics Committee approval and completed Australia’s Clinical Trial Notification process. The study has also been submitted for review in New Zealand, with enrollment there dependent on approval.

EDIT-401 Targets LDL-Cholesterol Through Gene Editing

EDIT-401 is an investigational in vivo gene-editing therapy designed to edit the LDLR gene to increase LDL receptor protein expression and lower LDL-C levels. In preclinical studies involving non-human primates, EDIT-401 demonstrated mean reductions of approximately 90% or greater in several atherogenic lipoproteins, including LDL-C, lipoprotein(a) and apolipoprotein B (ApoB). Additional preclinical data indicate that approximately 90% LDL-C reduction was maintained over 10 months in an ongoing study.

Strive Trial to Assess Safety and Efficacy

The Strive study will evaluate the safety, tolerability and efficacy of a single dose of EDIT-401. Part 1 is an open-label, single-ascending-dose study designed to identify an appropriate dose, while Part 2 will be a randomized, placebo-controlled expansion study. Editas has selected five clinical trial sites across Australia and New Zealand. The company expects to report initial safety and tolerability data in the first quarter of 2027, with Part 1 enrollment completion and topline safety and efficacy results anticipated later in 2027.

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Preclinical Data Set for AHA Scientific Sessions

Editas plans to present new preclinical findings on the durability of LDL-C reduction at the American Heart Association (AHA) Scientific Sessions 2026 in Chicago on November 8. The presentation will discuss EDIT-401’s potential to support sustained lipid reduction through gene editing. HeFH is an inherited condition associated with elevated LDL-C from an early age and increased cardiovascular risk. EDIT-401 remains investigational, and its safety and efficacy in humans have yet to be established.

Source::Editas Medicine, press release