Statins could be replaced by gene therapy within years, scientists say

Daily statins could be replaced by gene therapy which cuts cholesterol with a single dose within years, scientists claim

  • Statin use could be replaced ‘within the decade’ by gene-editing Verve-101 drug
  • Drug alters DNA inside cholesterol-regulating cells to shut down production
  • Nine have so far been successfully treated by Verve-101 with trials set to expand

Daily statins taken by millions to ward off heart attacks and strokes could be replaced ‘within the decade’ by a gene-editing jab, scientists claim.

The remarkable new drug manipulates the DNA inside cholesterol-regulating cells to shut down production of the substance after a single dose.

It was unveiled yesterday at the American Heart Association’s annual Scientific Sessions in Philadelphia.

Trial results showed the one-off injection cut ‘bad’ LDL cholesterol by more than 50 per cent.

Los Angeles-based cardiologist Professor Karol Watson predicted ‘a brave new world’ in heart treatment: ‘This trial showed with [gene] editing you can durably lower LDL.’

Daily use of statins could become a thing of the past due to a revolutionary new gene-editing drug

Experts behind the treatment, called Verve-101, believe its effect is long-lasting and possibly permanent.

Admitting research is at an early stage, they said their findings throw open the possibility that high cholesterol could effectively be ‘cured’ or prevented from becoming a problem in the first place.

‘This is a potential game-changer in treating heart disease,’ said Dr Sekar Kathiresan, one of the cardiologists behind Verve-101.

He added: ‘We’ve shown a one-time therapy could be the future.’

So far nine patients with genetic condition familial hypercholesterolaemia – which causes sky-high LDL levels that trigger heart attacks – have been successfully treated.

Trials will soon be extended to wider groups, including those with existing heart disease. ‘We hope the treatment will be available by the end of the decade,’ added Dr Kathiresan.

The drug is based on CRISPR technology which can delete and rewrite sections of DNA inside cells.

Unlike other CRISPR-based treatments, which can cost upward of £1million a dose, it is possible to mass-manufacture Verve-101, added Dr Kathiresan.

Nine patients have so far been treated successfully, with trials soon extending to larger groups

He said: ‘We expect it will cost no more than other one-off heart procedures like stent or heart bypass operations.’

Statins are one of the most prescribed drugs in the UK: about eight million adults take them, and more than 71million prescriptions for statins are dished out each year.

The drugs can be remarkably effective, reducing heart attack risk by up to 50 per cent and stroke risk by a third – while costing as little as £15 per patient per year.

Professor Watson said the biggest concern about the new treatment was safety: ‘If you’re editing the human genome, it’s a big deal, so we have to be sure it’s uber-safe.’

Dr Andrew Bellinger, the chief scientific and medical officer at Verve Therapeutics, said that large-scale trials featuring thousands of heart disease patients were planned to start in 2025.

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