CRISPR Therapeutics（CRSP.US）公布基因编辑疗法CTX310治疗ANGPTL3的一年期随访数据：最高剂量组ANGPTL3平均降低79%、甘油三酯降低48%、LDL降低53%

Fed chair Warsh kicks off Jackson Hole keynote address
ZUG, Switzerland and BOSTON - CRISPR Therapeutics (NASDAQ:CRSP) presented one-year follow-up data from its Phase 1a clinical trial of CTX310, an investigational gene editing therapy targeting ANGPTL3, according to a company press release statement.
The data, presented today at the European Society of Cardiology Congress 2026 and published in The New England Journal of Medicine, showed that a single intravenous infusion of CTX310 at the highest dose produced mean reductions from baseline of 79% for ANGPTL3, 48% for triglycerides, and 53% for low-density lipoprotein at one year. Maximum reductions reached 89% for ANGPTL3, 78% for triglycerides, and 84% for LDL.
The Phase 1a trial was an open-label, dose-escalation study evaluating single-course doses ranging from 0.1 to 0.8 mg/kg in 15 participants with conditions including 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.
The therapy was generally well tolerated with no dose-limiting toxicities or treatment-related serious adverse events. Three participants experienced Grade 2 infusion-related reactions, all of which resolved. One participant had a transient elevation in liver enzymes shortly after treatment, with no additional liver function test elevations throughout the remainder of the trial.
The company is advancing CTX310 in a Phase 1b clinical trial examining a fixed flat dose equivalent to 0.8 mg/kg. CRISPR Therapeutics expects to provide an update on the Phase 1b trial in the second half of 2026, focused on severe hypertriglyceridemia patients.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.