Editas Advances Gene-Editing Program Into First Human Study For High Cholesterol

(RTTNews) - Editas Medicine, Inc. (EDIT) has received regulatory and ethics approval in Australia to begin the first-in-human Phase 1/2 clinical trial of EDIT-401, an experimental gene editing therapy designed to treat hyperlipidemia, or high levels of LDL cholesterol.

The company remains on track to start dosing patients later this year.

Hyperlipidemia is a major risk factor for heart disease, heart attack, and stroke. The initial trial will focus on patients with Heterozygous Familial Hypercholesterolemia (HeFH), an inherited condition that causes very high LDL cholesterol levels from an early age. An estimated 1.2 million people in the United States are living with HeFH, many of whom remain undiagnosed.

EDIT-401 is an in vivo gene-editing medicine designed to directly modify the LDLR gene, which plays a key role in removing LDL cholesterol from the bloodstream. By increasing LDL receptor activity, the therapy aims to deliver long-lasting reductions in LDL cholesterol levels with a single treatment.

The Phase 1/2 Strive trial will evaluate the safety, tolerability, and effectiveness of a single dose of EDIT-401. The study consists of two parts. Part 1 is an open-label, single ascending-dose, dose-finding study designed to identify appropriate dose levels, while Part 2 is a randomized, placebo-controlled expansion study that will further assess the therapy's safety and efficacy. Editas has selected five clinical trial sites across Australia and New Zealand.

Preclinical studies have shown encouraging results. According to the company, EDIT-401 achieved approximately 90% or greater average reductions in LDL cholesterol, lipoprotein (a), and ApoB in non-human primates. Additional data demonstrated that LDL cholesterol reductions of about 90% were maintained for up to ten months in an ongoing study.

Editas expects to report initial safety and tolerability data from the trial in the first quarter of 2027, with topline safety and efficacy results from the dose-finding portion of the study expected later in 2027.

The company also plans to present new preclinical durability data for EDIT-401 at American Heart Association (AHA) Scientific Sessions 2026, in November.

The approval marks an important milestone for Editas Medicine as it advances its gene-editing technology into human testing for cardiovascular disease, expanding beyond rare genetic disorders into a much larger patient population.

EDIT has traded between $1.66 and $4.33 over the past year. The stock closed Wednesday's trading at $2.75, up 0.73%. In pre-market trading, the stock is at $2.80, up 1.82%.