One-time gene-editing infusion cut dangerous blood fats by 63% in a small early trial
CRISPR Therapeutics, which does research in San Francisco, says its CTX310 switches off a fat-raising gene in the liver. Only 10 patients were treated, and long-term effects are unknown.
A single infusion of an experimental gene-editing treatment lowered triglycerides, a type of fat in the blood, by an average of 63% after 30 days in 10 adults with very high levels, according to results presented Nov. 9 at the American Heart Association’s meeting in Chicago. The treatment, CTX310, is made by CRISPR Therapeutics, which has research operations in San Francisco and Boston, the company says.
How it works
CTX310 uses CRISPR-Cas9, a tool that cuts DNA at a chosen spot, to switch off a gene called ANGPTL3 in liver cells. That gene helps control how the body handles fats. Tiny fat bubbles called lipid nanoparticles carry the tool to the liver. The protein the gene makes fell 65% on average, the company said.
Very high triglycerides raise the risk of pancreatitis, a painful swelling of the pancreas, and heart disease, said Dr. Ashish Sarraju, the Cleveland Clinic cardiologist who presented the data. Many patients stay high despite current drugs, the company says. Researchers hope one treatment could work without ongoing medicine, according to Cleveland Clinic.
The results
All 10 patients had diabetes, and their median starting level was above 1,000 milligrams per deciliter. Three had had pancreatitis before. Non-HDL cholesterol, a measure of artery-clogging fats, fell 51%. The seven patients followed for at least 90 days kept their lower levels. There were no serious side effects tied to the treatment, and one infusion reaction cleared up, the company said.
What is not known
This is a Phase 1b trial, an early study focused on safety, with no comparison group. It does not show how long the effect lasts or whether it prevents pancreatitis or heart attacks; Cleveland Clinic says more research is needed on durability. CTX310 is not approved. The Food and Drug Administration has given it Regenerative Medicine Advanced Therapy status, part of a program to speed development of such treatments for serious conditions. CRISPR Therapeutics paid for the study.
Source: Cleveland Clinic news release, Nov. 9, 2026 and CRISPR Therapeutics news release.