CRISPR Therapeutics Advances Gene-Editing Treatments

The company shared new CTX310 trial results and outlined timelines for CTX320 and CTX340 programs.

June 27, 2025

Targeting the Heart of the Problem

CRISPR Therapeutics is moving fast on its mission to tackle cardiovascular disease through gene editing. The company just released an update on three programs—CTX310, CTX320, and CTX340—each designed to target a specific genetic driver of heart conditions.

CTX310: Strong Momentum from Early Results

CTX310 leads the way. It’s already in a Phase 1 trial, focused on patients with high triglycerides or LDL cholesterol. The latest data shows a clear dose response. At the highest dose tested, patients saw triglycerides drop by as much as 82%, and LDL levels fall by up to 86%. Liver enzyme levels stayed stable, and the safety data lined up with earlier results. That’s meaningful progress—and a strong signal that the program is headed in the right direction.

CTX320 and CTX340: Strategic Progress

Next up is CTX320, designed for patients with elevated lipoprotein(a), a genetically driven cardiovascular risk factor that affects millions worldwide. While the Phase 1 trial is still enrolling, CRISPR Therapeutics has pushed the next update to the first half of 2026. That change gives the team space to incorporate new insights emerging in the fast-moving Lp(a) space.

The company is also laying the groundwork for CTX340. This candidate targets angiotensinogen to treat resistant hypertension and is currently moving through studies needed to support clinical testing.

What’s Next

Each program is built on the company’s gene-editing platform and reflects a broader push to address complex, high-burden conditions at the source. The timelines may evolve, but the direction is clear: keep advancing, keep refining, and keep translating the science into therapies.