American scientists have injected into the liver of a living person a serum they developed for gene editing. With this new drug, you can reduce the level of bad cholesterol in the body.
Researchers from the American biotech company Verve Therapeutics injected a gene-editing serum into the liver of a living patient in order to lower cholesterol levels. This is a turning point in the history of gene editing, which could potentially save millions of people from cardiovascular diseases and heart attacks, according to MIT Technology Review.
How the serum works
The new drug was named VERVE-101. Previously, its effectiveness was tested on monkeys, and the results were encouraging. Now the researchers have moved on to human clinical trials — the serum was injected into the patient’s liver from New Zealand.
Specialists Verve Therapeutics claim that their development is able to edit genes in such a way that the liver begins to produce less “bad” cholesterol, which in excess amounts leads to blockage of the arteries.
Unlike classical methods of combating high cholesterol, which include strict diets, physical activity and medication, VERVE-101 has a long-lasting effect and works much more effectively.
“VERVE-101 is a first—in—class gene editing drug that we have developed so that with just one DNA editing in the liver, the disease-causing gene can be permanently disabled,” said Sekar Kathiresan, founder of Verve Therapeutics.
Now the company hopes to attract about 40 adult patients with familial heterozygous hypercholesterolemia, a hereditary genetic disease that causes dangerously high cholesterol levels, to the trials. With their help, scientists hope to assess the safety of their drug.