CRISPR Therapeutics (NASDAQ: CRSP) on Wednesday disclosed one-year follow-up data for its investigational gene therapy CTX310, demonstrating sustained lipid-lowering effects in a Phase 1a clinical trial.
The open-label, dose-escalation study evaluated single intravenous infusions of CTX310 at doses ranging from 0.1 to 0.8 mg/kg in 15 participants with homozygous familial hypercholesterolemia, severe hypertriglyceridemia, heterozygous familial hypercholesterolemia, or mixed dyslipidemias. All participants completed at least one year of follow-up as of the data cutoff date.
At the highest dose tested, CTX310 achieved a mean 79% reduction in ANGPTL3 levels from baseline, with a maximum reduction of 89%. Triglycerides declined by a mean 48% (maximum 78%), while low-density lipoprotein cholesterol fell by a mean 53% (maximum 84%). The therapy was generally well tolerated, with no dose-limiting toxicities or treatment-related serious adverse events reported. Three participants experienced Grade 2 infusion-related reactions, all of which resolved, and one participant showed a transient elevation in liver enzymes shortly after dosing without further abnormalities.
The company is now evaluating a fixed flat dose equivalent to 0.8 mg/kg in a Phase 1b trial focused on patients with severe hypertriglyceridemia. CRISPR Therapeutics expects to provide an interim update on the trial in the second half of 2026.
The results were presented at the European Society of Cardiology Congress 2026 and published concurrently in The New England Journal of Medicine.
