A new experimental cholesterol treatment is being tested as a potential one-and-done option instead of a lifelong routine of pills or repeated injections.

 

A new experimental cholesterol treatment is being tested as a potential one-and-done option instead of a lifelong routine of pills or repeated injections. The goal is to reduce LDL cholesterol by making a precise genetic change that lowers the activity of PCSK9, a key regulator of LDL levels in the bloodstream.

This treatment, called VERVE-102, is delivered as a single IV infusion. It uses a form of base editing (a refined gene-editing approach) intended to switch off PCSK9 in the liver, so the body can clear LDL cholesterol more effectively over time.

In the company’s early update from the Heart-2 clinical trial (14 participants reported in the initial release), LDL cholesterol dropped in a dose-dependent way. The average LDL-C reduction reported was around 53%, and the largest individual reduction reported was 69% in one participant at the higher dose level.

Safety-wise, these early results reported no treatment-related serious adverse events, with ongoing follow-up still needed to understand durability and longer-term risk. Some coverage also noted that these were early trial results and not necessarily peer-reviewed at the time of reporting.

If future studies confirm both safety and lasting benefit, this kind of approach could shift cholesterol care toward long-term control from a single treatment, especially for people at very high cardiovascular risk, including those with inherited cholesterol disorders

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