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Single CTX310 infusion silences ANGPTL3, cuts LDL and triglycerides after one year At the European Society of Cardiology congress, August 28, researchers presented 12‑month data from the phase 1 trial of CTX310. The drug delivers CRISPR‑Cas9 components to the liver in a single infusion to knock out the ANGPTL3 gene. In the four participants who received the highest dose of 0.8 mg/kg, LDL‑cholesterol was on average 52.5% lower than baseline after one year, and triglycerides were 47.8% lower. These changes persisted for the full 12‑month period. CTX310 uses a lipid nanoparticle to ferry mRNA for Cas9 and a guide RNA targeting ANGPTL3 into hepatocytes; once inside, Cas9 cuts the DNA, and the repaired gene loses function. ANGPTL3 normally inhibits enzymes that clear fats from the blood, so its loss improves lipid levels. The phase 1 study enrolled 15 adults with severe lipid disorders unresponsive to maximal tolerated therapy, split into five dose groups. Safety and tolerability were the primary outcomes, while ANGPTL3 editing and lipid changes were secondary measures, as listed in the trial registry. 🔗 Read original →