Editas Medicine Pivots to In Vivo Gene Editing, Names New Lead Candidate

CAMBRIDGE, MA. Sep 2, 2025 – Following a dramatic restructuring and a strategic pivot to in vivo gene editing, Editas Medicine has nominated a new lead development candidate, EDIT-401. The announcement, made by the company on Tuesday, provides a clear glimpse into its future direction, with a new focus on a large cardiovascular market.

EDIT-401 is a one-time gene therapy designed to significantly reduce LDL cholesterol (LDL-C) levels. In preclinical studies conducted in nonhuman primates, the asset showed a remarkable 90% mean reduction, far surpassing the 40% to 60% reduction typically seen with current standard-of-care therapies. The company aims for the therapy to provide a lifelong benefit to patients after a single treatment.

The drug works by editing the LDLR gene to increase the expression of the LDLR protein, which is responsible for clearing LDL-C from the bloodstream. According to the Massachusetts-based biotech, this approach has demonstrated a “favorable” profile for both efficacy and tolerability in preclinical testing.

This new direction marks a significant change from the company’s previous focus on rare genetic diseases like sickle cell disease. By pursuing LDL-C reductions, Editas is targeting a much broader market in atherosclerotic cardiovascular disease, which is projected to cost the U.S. healthcare system more than $300 billion by 2035.

While still in the early stages, Editas has set a clear timeline for the program. The company plans to submit its investigational new drug (IND) or a clinical trial application (CTA) to the FDA by mid-2026. This would allow for the potential delivery of human proof-of-concept data by the end of next year.

To support this new focus, Editas has funneled most of its resources toward EDIT-401. The company’s current cash and equivalents, at approximately $178.5 million as of June 30, 2025, are expected to fund operations into the second quarter of 2027. This financial runway follows a series of strategic moves, including the discontinuation of its prior lead program, reni-cel, and a layoff of 65% of its staff last year.