Editas Medicine clears first-in-human trial of EDIT-401 in Australia

Editas Medicine (Nasdaq: EDIT) announced it has received Human Research Ethics Committee approval and completed the Clinical Trial Notification process with Australia's Therapeutic Goods Administration to initiate a Phase 1/2 clinical trial of EDIT-401, an experimental gene editing therapy targeting LDL cholesterol reduction.

The trial, called Strive, is designed to evaluate the safety, tolerability, and efficacy of a single dose of EDIT-401 in patients with Heterozygous Familial Hypercholesterolemia (HeFH). The company said it has selected five clinical trial sites across Australia and New Zealand. The trial has also been submitted for regulatory review in New Zealand.

Editas said it expects to begin patient dosing in 2026 and report initial safety and tolerability data in the first quarter of 2027. Topline safety and efficacy data from Part 1, the dose-finding portion of the trial, are expected later in 2027.

EDIT-401 is designed to edit the LDLR gene to increase LDLR protein expression and lower LDL-C levels. In preclinical studies conducted in non-human primates, the therapy demonstrated approximately 90% or greater mean reduction in LDL-C, Lp(a), and ApoB. New preclinical data showed that an approximately 90% LDL-C reduction was maintained over 10 months in an ongoing non-human primate study.

Editas will present these preclinical findings in an oral presentation at the American Heart Association Scientific Sessions 2026 on November 8, 2026, in Chicago, Illinois. The presentation, titled "EDIT-401: In Vivo Gene Editing to Enhance LDLR Function for Durable LDL-C Reduction," will be delivered by Dan Ory, M.D., Chief Medical Officer of Editas Medicine.

HeFH is an inherited disorder estimated to affect approximately 1.2 million people in the United States, according to the company's press release statement. Elevated LDL-C more broadly is said to affect over 70 million patients in the U.S.

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