Single CRISPR infusion cuts stubborn LDL cholesterol for a year

By Adrian Cole · September 22, 2026, 6:42 AM · 3 mins read

A single dose of CTX310, a CRISPR-Cas9 therapy, dropped LDL cholesterol by more than half in patients who did not respond to standard drugs. The effect lasted at least a year.

One year after a single infusion, patients with hard-to-treat high cholesterol saw their LDL drop by 52.5%. Just a few years ago, this would have sounded impossible. The treatment behind this result is CTX310, a CRISPR-Cas9 gene-editing therapy tested in a Phase 1 trial at the Cleveland Clinic.

Fifteen people took part. All had lipid metabolism disorders that did not respond to usual treatments. They got CTX310 in doses from 0.1 to 0.8 mg/kg. Before the gene-editing, each patient received corticosteroids and antihistamines to lower the risk of immune reactions.

CTX310 is designed as a one-time in vivo CRISPR-Cas9 therapy targeting ANGPTL3 in the liver, with early data showing up to 80% reduction in ANGPTL3 protein at higher doses.
Nasdaq

CTX310 works by targeting and shutting down the ANGPTL3 gene in the liver. This gene controls blood fat levels. By turning it off, the therapy sharply lowers both LDL cholesterol and triglycerides. Both are major risk factors for heart disease. The trial showed a 47.8% drop in triglycerides along with the big LDL reduction. All this came from just one treatment.

The results lasted. The first improvements showed up two months after the infusion. They stayed steady for the full year. People who got the highest dose saw the biggest benefits, showing a clear link between dose and effect. According to a Nasdaq review of CRISPR Therapeutics data, higher doses brought LDL and triglyceride reductions close to 50%, with the effect holding at 12 months.

Safety is always a concern with gene-editing. In the first year, there were no serious side effects tied to CTX310. Still, the story is not finished. The FDA requires all trial participants to be followed for 15 years to watch for any long-term problems from this powerful approach.

As of September 2026, CTX310 has advanced from Phase 1a to Phase 1b, with ongoing studies now focusing on patients with severe hypertriglyceridemia and mixed dyslipidemia. The company expects new subgroup data by the end of 2026, reflecting the early stage of clinical development.
Investing.com transcriptIndustry reporting

For people whose cholesterol does not budge with standard drugs, CTX310's early results point to a possible breakthrough. The lasting, dose-linked drops in LDL and triglycerides after just one treatment open a new path for gene-editing in metabolic disease. The long-term safety picture is not yet clear, but the lack of serious side effects in the first year is a good sign. If these results hold up in bigger, longer studies, CTX310 could change the game for patients left out by current cholesterol-lowering options.

CRISPR Therapeutics chose ANGPTL3 as the target for CTX310 because natural loss-of-function mutations in this gene are linked to lower heart risk. This reasoning is detailed in company conference transcripts. The ongoing Phase 1b trial now includes patients with severe hypertriglyceridemia, showing the therapy may help a wider group with metabolic problems.

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Adrian Cole

Adrian Cole is the Founder and Editor-in-Chief of GenoMethods, where he writes about bioengineering, genome and cell engineering, synthetic biology, computational biology and emerging research methods. His editorial approach focuses on how technologies actually work, how they are validated and where the evidence stops supporting the claim.