Unknown Heart, Stroke & Blood NIHR-supported project Digestion, Kidneys & Other Organs

A Phase 1b Single Ascending Dose Study to Evaluate the Safety of VERVE-201 in Patients with Refractory Hyperlipidemia

Funder NIHR (part of DHSC)
Recipient organisation NIHR Wellcome Trust Manchester Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Mar 2025 — Ongoing

In plain English

AI plain-English summary

A single dose of the experimental medicine VERVE-201 will be tested in people for the first time, to see if it can safely lower cholesterol by editing a gene in their DNA. This matters because millions of people with refractory hyperlipidemia cannot reach their recommended cholesterol targets with existing drugs. High levels of LDL-C, or “bad” cholesterol, narrow arteries and raise the risk of heart attacks and strokes. Current treatments—statins, injections, and lifestyle changes—often fail to bring cholesterol low enough for these patients. VERVE-201 takes a different approach: it makes a permanent change to the DNA of the ANGPTL3 gene, switching it off. Turning off this gene is known to reduce LDL-C levels. If the trial shows VERVE-201 is safe, it could lead to a one-time treatment that frees patients from daily pills or regular injections. For people who have exhausted other options, a single dose might keep their cholesterol low for years, reducing their risk of cardiovascular events. This is a phase 1b safety study, so the immediate goal is not efficacy but establishing that the medicine does not cause serious harm. Participants will be followed for up to 15 years to track long-term effects.

View original technical description
Verve Therapeutics is funding this research to see if VERVE-201 can safely lower cholesterol in patients who have not been able to achieve their recommended cholesterol goal. High cholesterol occurs when high levels of a type of fat called low-density lipoprotein cholesterol (LDL-C), sometimes referred to as “bad” cholesterol, circulate in the blood. If there is too much LDL-C in your blood, it could cause your arteries to narrow and increase the risk of suffering a heart attack or stroke. VERVE-201 is designed to lower the levels of LDL-C in the blood. VERVE-201 does this by making a change to the DNA in a gene called “ANGPTL3” to turn the gene off. Turning off the ANGPTL3 gene is known to lower LDL-C levels. VERVE-201 is an investigational medicine not yet approved for use by any health authorities. This is the first time VERVE-201 is being tested in humans. The primary goal of this study is to determine whether VERVE-201 is safe. Participants will be in the study for approximately one year but will be asked to participate in a long-term follow-up study for up to 15 years.

Researchers

Handrean Soran (Principal Investigator)

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