EDIT-401

October 8, 2026: Editas Medicine (NASDAQ: EDIT) said its Phase 1/2 Strive trial of EDIT-401 was cleared to start in Australia for patients with heterozygous familial hypercholesterolemia (HeFH) who need additional LDL-C lowering. The trial received HREC approval and completed the TGA Clinical Trial Notification process; the study has also been submitted for review in New Zealand. Editas said it remains on track to begin dosing in 2026.

EDIT-401 is an in vivo gene-editing therapy designed to edit LDLR to increase LDL receptor expression and lower LDL-C. Strive will test a single dose in two parts: Part 1 is an open-label single-ascending-dose, dose-finding study, and Part 2 is a randomized, placebo-controlled single-dose expansion study. Five sites have been selected across Australia and New Zealand.

Upcoming catalysts: initial safety and tolerability data are expected in Q1 2027, and topline safety and efficacy data from Part 1 are expected later in 2027.

The company also said new preclinical durability data will be presented in an oral session at the AHA Scientific Sessions 2026 on November 8, 2026. In non-human primates, EDIT-401 previously showed about 90%+ mean reductions in LDL-C, Lp(a), and ApoB, and new data showed about 90% LDL-C reduction maintained over 10 months.