Phase I Trial of CRISPR Cholesterol Drug Shows Half Reduction
Trial presented at American Heart Association meeting in New Orleans, published in The New England Journal of Medicine.
Drug targets ANGPTL3 gene in liver, mimicking a genetic mutation in 1 in 250 US people.
Trial locations: UK, Australia, New Zealand.
Participants aged 31 and 68, baseline LDL 155 mg/dL, triglycerides 192 mg/dL.
One participant died months after infusion.
Phase 2 trials planned to test safety of gene edit for heart attacks and strokes.
Company cautions about long-term safety and costs, per Dr. Luke Laffin.
2 months ago