One-Time CRISPR Therapy Halves “Bad” Cholesterol

CRISPR Therapy Halves “Bad” Cholesterol

    Advances in gene editing shows how quickly CRISPR-based therapies are moving from rare-disease, into broader applications. The Phase 1 trial involved CTX310, a CRISPR-Cas9 gene-editing therapy that targets the ANGPTL3 gene in liver cells. By “turning off” ANGPTL3, the treatment dramatically reduced LDL (“bad”) cholesterol by ~50% and triglycerides by ~55%, with effects seen within two weeks and sustained for at least 60 days. 

    Safety results from the initial trial were promising. Participants experienced no serious side effects linked to the CRISPR treatment, and only mild infusion-related reactions were reported. The most significant takeaway is the therapy’s durability. A single treatment may produce long-lasting reductions in harmful blood lipids. Early data indicate substantial editing of the ANGPTL3 gene, up to nearly 90% in some patients, which directly correlates with significant decreases in LDL cholesterol and triglycerides.

    If larger studies confirm both long-term safety and effectiveness, this approach could transform how cardiovascular disease is managed. Instead of relying on lifelong daily medications, patients might be able to receive a one-time gene-editing therapy that permanently lowers their risk. Still, several challenges remain, including determining long-term effects, monitoring for possible off-target edits, and ensuring the therapy is accessible and affordable for the people who need it most.

Main Article: https://newsroom.clevelandclinic.org/2025/11/08/cleveland-clinic-first-in-human-trial-of-crispr-gene-editing-therapy-shown-to-safely-lower-cholesterol-and-triglycerides?

Additional article: https://newsroom.heart.org/news/first-in-human-trial-of-crispr-gene-editing-therapy-safely-lowered-cholesterol-triglycerides?