FDA Clears Single Dose Gene Therapy For Cholesterol
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In a major step forward for heart health, Verve Therapeutics has received approval from the U.S. FDA to begin human trials in the U.S. for its new cholesterol-lowering therapy, VERVE-102. This experimental medicine is designed to be a single-dose gene editing treatment that turns off the PCSK9 gene in the liver, which plays a key role in regulating LDL or "bad" cholesterol.
Why It Matters
Many people take cholesterol-lowering drugs like statins daily. However, staying on long-term medication is a challenge. Studies show that the biggest benefit in reducing heart attack risk comes not just from how much cholesterol is lowered—but for how long. A single treatment like VERVE-102 could help maintain low cholesterol levels for life, improving long-term heart health without the burden of daily pills.
Early Data Looks Promising
As part of its FDA application, Verve shared data from international clinical trials. Participants who received VERVE-102 experienced no serious side effects or abnormal lab results across all early dose groups. The trial, called Heart-2, is now expanding into the U.S., and results from the first set of participants are expected in mid-2025. Full dose trial data and a Phase 2 trial are planned for later in the year.
What’s Next
Verve is also working on similar gene-editing treatments targeting other genes that raise cholesterol and heart risk, including ANGPTL3 and Lp(a). These medicines aim to tackle difficult-to-treat inherited cholesterol disorders.