Core Insights - CRISPR Therapeutics announced positive Phase 1 clinical trial results for CTX310, a CRISPR/Cas9 gene-editing therapy targeting ANGPTL3, demonstrating significant reductions in triglycerides and LDL cholesterol after a single intravenous infusion [1][2][11] Clinical Data Summary - The Phase 1 trial showed a mean reduction in circulating ANGPTL3 of -73% (maximum -89%), triglycerides (TG) by -55% (maximum -84%), and low-density lipoprotein (LDL) by -49% (maximum -87%) at the highest dose [1][12] - Among participants with elevated baseline TG (>150 mg/dL), a mean reduction of 60% in TG was observed at therapeutic doses [1][12] - CTX310 was well tolerated with no serious adverse events related to treatment and no significant changes in liver transaminases [1][7][9] Safety and Tolerability - The trial included 15 participants who received ascending doses of CTX310, with all participants completing at least 28 days of follow-up [7][9] - Adverse events were generally mild to moderate, with one allergic reaction and infusion-related reactions in three participants, all of which resolved [8][9] Efficacy Highlights - The results indicate that CTX310 has the potential to provide durable lipid-lowering effects following a single-course IV administration, which could transform treatment for patients with severe dyslipidemia [11] - The study's findings support the advancement of CTX310 into Phase 1b clinical trials, focusing on severe hypertriglyceridemia and mixed dyslipidemia [11] Company Overview - CRISPR Therapeutics is a leading gene editing company focused on developing transformative medicines for serious diseases, with a diversified pipeline that includes CTX310 and other investigational programs targeting cardiovascular diseases [14]