Gene-editing injection could improve cholesterol

Individuals who have inherited a genetic predisposition for high LDL cholesterol may benefit from a simple gene-editing technique, scientists say.

The work was carried out by a team led by Dr Andrew Bellinger of Verve Therapeutics in Boston, USA, and presented at the American Heart Association’s Scientific Sessions 2023 held in Philadelphia, USA.

In their report, the team state that lifelong exposure to circulating low-density lipoprotein cholesterol (LDL-C) “is a primary driver of atherosclerotic cardiovascular disease”.

People with a mutation in the PCSK9 gene are at greater risk, so the team developed ‘VERVE-101’, a treatment which alters DNA to inactivate this mutation.

“VERVE-101 is composed of a messenger RNA encoding an adenine base editor and a guide RNA that targets the PCSK9 gene, packaged within a lipid nanoparticle,” they write.

Findings from early stage studies in mice and non-human primates indicated that a single injection of VERVE-101 can alter the PCSK9 gene in the liver.

The treatment led to “substantial reductions in LDL-C that remained durable through more than two years of follow-up, supporting the potential for a permanent treatment effect,” say the authors.

It was then given to ten adult patients, with an average age of 54, in “the first reported results for any in vivo DNA base editing medicine administered to human trial participants”.

Similar effective results were seen in these patients, who were at high risk of early heart attack due to the PCSK9 gene mutation.

Dr Bellinger said: “Instead of daily pills or intermittent injections over decades to lower bad cholesterol, this study reveals the potential for a new treatment option – a single-course therapy that may lead to deep LDL-C lowering for decades.”

Vafai, S. B. et al. Safety and Pharmacodynamic Effects of VERVE-101, an Investigational DNA Base Editing Medicine Designed to Durably Inactivate the PCSK9 Gene and Lower LDL Cholesterol – Interim Results of the Phase 1b heart-1 Trial. American Heart Association Scientific Session, 12 November 2023


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