CRISPR Therapy CTX310 Shows Promise for Hard-to-Treat Dyslipidemia

A first-in-human CRISPR-Cas9 gene-editing therapy, CTX310, targeting angiopoietin-like protein 3 (ANGPTL3), has shown encouraging results in patients with refractory dyslipidemia, according to a phase I study presented at the AHA 2025 Scientific Sessions and published in the New England Journal of Medicine.

Given as a one-time IV infusion, CTX310 produced mean reductions of 48.9% in LDL cholesterol and 55.2% in triglycerides at the highest dose over 60 days. Other lipids—including apolipoprotein B and non-HDL cholesterol—also declined significantly. Importantly, no dose-limiting toxicities or serious treatment-related adverse events were reported.

The therapy uses lipid nanoparticles to deliver CRISPR components to the liver, inducing a loss-of-function mutation in ANGPTL3—a mechanism inspired by people with natural mutations who have lifelong low cholesterol and reduced ASCVD risk.

Experts called the results “a new frontier” in lipid management but urged caution and long-term safety follow-up, especially for potential off-target or liver effects. If proven durable and safe, CTX310 could one day offer a “one-and-done” alternative to chronic statins, PCSK9 inhibitors, or ANGPTL3 antibodies like evinacumab.

Key Takeaway:
Early data show that CTX310 can profoundly lower LDL-C and triglycerides after a single dose, potentially transforming lipid therapy—pending confirmation of long-term safety and efficacy.