‘Unprecedented’: Single dose of new gene-editing therapy lowers cholesterol, triglycerides
A single dose of a new CRISPR-Cas9 gene-editing therapy significantly reduced low-density lipoprotein (LDL) cholesterol and triglyceride levels in high-risk heart patients, according to new first-in-human data presented at the American Heart Association’s Scientific Sessions 2025 conference. The findings were simultaneously published in The New England Journal of Medicine.[1]
The therapy in question, CTX310 from publicly-traded CRISPR Therapeutics, works by switching off ANGPTL3 proteins in the liver. It is delivered intravenously. The idea to target this protein came after researchers noted that some people are born with natural mutations that turn off ANGPTL3. This individuals are consistently associated with low cholesterol and triglyceride levels.
Overall, 15 patients with known lipid disorders received a one-time infusion of the therapy. Within two weeks, their LDL cholesterol and triglyceride levels had dropped significantly; the reductions lasted for at least 60 days. These findings impressed the study’s authors, who were not necessarily expecting such a substantial impact
“This is really unprecedented,” lead author Luke J. Laffin, MD, a cardiologist with Cleveland Clinic, said in a statement. “If confirmed in larger trials, this one-and-done approach could transform care for people with lifelong lipid disorders and dramatically reduce cardiovascular risk.”
“Adherence to treatment is a significant issue for patients. In fact, half of the patients who are treated with cholesterol-lowering drugs stop taking them within a year,” senior author Steven Nissen, MD, chief academic officer of Cleveland Clinic’s Heart, Vascular and Thoracic Institute, said in a separate statement. “Although it is early in the study of gene-editing therapies, the efficacy demonstrated in the current trial is highly promising and represents a potential new frontier for drug development.”
CRISPER Therapeutics shares a statement
CRISPER Therapeutics, which funded this analysis, issued its own statement celebrating the early success.
"The publication and presentation of these Phase 1 results mark an important milestone for CRISPR Therapeutics and for the field of in vivo gene editing,” Naimish Patel, MD, chief medical officer of CRISPR Therapeutics, said. “For the first time, we’ve shown that a single-course in vivo CRISPR treatment can safely and durably lower ANGPTL3, leading to clinically meaningful reductions in triglycerides and LDL. These data provide strong support for continued advancement of CTX310 and our broader cardiovascular gene-editing portfolio.”
Additional context
From a safety perspective, CTX310 was found to be relatively safe. However, it was still associated with two serious adverse events and three infusion-related reactions.
“Longer follow-up in larger patient populations is required to assess late-emerging or low-incidence safety signals,” the authors wrote.
The authors also noted that there is still much more work to be done before this therapy can gain regulatory approvals.
“Further studies are needed to understand patient-specific predictors of treatment effects, editing efficiency and optimized dose-administration strategies,” they wrote.
Click here to read the full analysis in The New England Journal of Medicine. Click here for additional details about the American Heart Association’s Scientific Sessions 2025 conference.
Michael has more than 19 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.