Editas Medicine receives approval to initiate Phase 1/2 trial of EDIT-401

Editas Medicine announced on October 8, 2026 that it received approval to initiate the Phase 1/2 Strive trial of EDIT-401 in patients with Heterozygous Familial Hypercholesterolemia in Australia.¹ The trial received Human Research Ethics Committee approval and completed the Clinical Trial Notification process with Australia's Therapeutic Goods Administration, and has also been submitted for review in New Zealand.¹ If New Zealand grants approval, patients in both countries will be eligible to enroll in the Strive trial.¹

EDIT-401 is designed as an in vivo gene editing treatment for hyperlipidemia. It works through Editas' upregulation approach, directly editing the LDLR gene to increase LDLR protein expression and lower LDL-cholesterol levels.¹ In preclinical studies, the therapy produced roughly 90 percent or greater mean reductions in several atherogenic lipoproteins, including LDL-C, Lp(a), and ApoB, in non-human primates.¹ New data to be presented at the American Heart Association Scientific Sessions in November 2026 showed that an approximately 90 percent LDL-C reduction was sustained over 10 months in an ongoing non-human primate study.¹

The Strive trial will assess safety, tolerability, and efficacy of a single dose of EDIT-401, structured in two parts: a single ascending dose, open-label, dose-finding Part 1, followed by a randomized, placebo-controlled Part 2 expansion study.¹ Five clinical trial sites have been selected across Australia and New Zealand.¹

Editas expects to report initial safety and tolerability data in the first quarter of 2027, with enrollment in Part 1 completed and topline safety and efficacy results available later that year.¹