Gene Editing’s Breakthrough: One-Time Therapies Slash Cholesterol, Transform Care
Published Nov 16, 2025
On 2025-11-08 CRISPR Therapeutics reported Phase I results from a 15‐participant, single‐dose in‐vivo ANGPTL3 gene‐editing trial in the UK, Australia and New Zealand showing ~50% reductions in LDL cholesterol and triglycerides lasting at least 60 days; one participant with preexisting heart disease died but the death was not attributed to the treatment and no serious adverse events were linked to the therapy, and Phase II is planned for 2026. Separately, VERVE‐102 (PCSK9 base editing) delivered average LDL reductions of 53% (up to 69% in the highest dose) after one dose and was well tolerated. These early, durable effects move gene editing into common cardiovascular/metabolic markets and prioritize Phase II and long‐term follow‐up data, regulatory pathways, manufacturing scale‐up and pricing/reimbursement strategies.