CTX310 Phase 1 results published in NEJM show dose-dependent triglyceride and LDL reductions after one infusion

CRISPR Therapeutics/CTX310 (ANGPTL3)

CRISPR Therapeutics' in-body CRISPR therapy CTX310 produced dose-dependent drops in triglycerides and LDL cholesterol after a single infusion in 15 patients with uncontrolled dyslipidemia. At the top dose, mean LDL fell about 49% and triglycerides about 55%, with no serious adverse events reported.

• CTX310 switches off the ANGPTL3 gene in the liver with CRISPR-Cas9 delivered in lipid nanoparticles.
• At 60 mg: mean 73% reduction in ANGPTL3, 55% drop in triglycerides, 49% drop in LDL (max 87% and 84%).
• One infusion showed durable effects; average follow-up 60 days.
• No Grade 3 transaminase elevations or serious adverse events; infusion reactions were mild.