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Verve Therapeutics' gene editing therapy may cure heart attacks once and for all

Trials underway to test the safety, but affordability and acceptance of gene therapy are major concerns

06-May-2022

Key points from article :

Even after decades of breakthroughs aimed at preventing heart attacks, they remain the world’s leading cause of death.

Verve Therapeutics is proposing to alter a person’s genome to stop the buildup of bad cholesterol.

“We’re on the cusp of potentially transforming that model to a one-and-done treatment,” - Sekar Kathiresan, CEO of Verve.

Initially targets those who’ve already had a heart attack.

Eventually aiming to give it to young people as a preventive measure.

Uses the Crispr DNA-editing tool to change a single letter of a person’s genome.

A lipid nanoparticle encases the editing system to protect it on its journey to the liver, where it turns off the desired gene.

Medicines focused on two genes: PCSK9 and ANGPTL3.

Some patients will need only one of the drugs; others will need both.

Encouraging signs in monkeys, cutting bad cholesterol levels by 59% after two weeks.

Verve is set to begin testing in humans within months.

Analysts forecast that this therapy will cost from $50,000 to $200,000.

Mentioned in this article:

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Sekar Kathiresan

Physician scientist, human geneticist and professor at Harvard Medical School.

Verve Therapeutics

Biotechnology company

Topics mentioned on this page:
Heart Disease, Gene Therapy