Editas pivots to tackle high cholesterol with gene editing breakthrough

Editas Medicine has unveiled EDIT-401, a preclinical gene editing therapy targeting high LDL cholesterol, showing a 90% LDL-C reduction level in animal tests and aiming for human trials by late 2026.

Why it matters: With standard treatments lowering LDL-C by only 40–60%, Editas' 90% reduction potential offers a major leap, if human trials confirm safety and durability. The drug works by editing the LDLR gene to boost receptor expression.

Backstory: Editas shifted from rare diseases like sickle cell to in vivo therapies after workforce cuts and leadership changes in 2024. These changes were prompted by the company´s failure to secure a partner and laid off 65% of staff.

Big picture: Cardiovascular disease, driven by high LDL-C, is projected to cost over $300B in the U.S. by 2035. A one-time, lifelong treatment could redefine how cholesterol is managed globally.

Zoom in: Backed by $178.5M in cash, Editas expects to fund operations through Q2 2027. With this runway, the company is taking a high-stakes gamble on cardiovascular gene editing as Editas is expending the majority of its resources on this single asset.

What’s next: The company aims to file for FDA clearance in mid-2026 and show proof-of-concept data by year-end for EDIT-401.