A single Gene-Editing treatment had a reduction in high cholesterol

The trial of gene-editing the LDL cholesterol in patients with inherited heart disease is open to a new study, and no one has tried to lower it

No one has ever tried to lower cholesterol using gene editing. “This is a strategy that could be revolutionary,” said Karol Watson, a cardiologist at UCLA, during the news conference. We need to make sure it’s safe.

The experiment was carried out in 10 participants with an inherited condition that causes extremely high LDL, or “bad,” cholesterol levels, which can lead to heart attack at an early age. Despite being on cholesterol-lowering medications, the volunteers were already suffering from heart disease. They joined a trial run by a Massachusetts–based company.

Critics also criticized the findings. Two serious adverse events in the trial, including a death, have raised safety concerns, and Verve’s share price plummeted by nearly 40% following the results’ release despite their promise.

The results of a phase 1b trial were reported by Verve at the meeting of the American Heart Association in Philadelphia. The trial will continue in the US next year after receiving approval from the FDA.

The treatment resulted in some side effects, including a brief flu-like symptom, as well as a temporary increase in liver enzymes which returned to normal within days.

Review of a recent research by Torres on a genetic-medicines trial: the case of non-rare gene editing in mice and monkeys

“The sentiment for editing when there are viable alternatives is going to be a challenge. Time will tell if non-rare is viable, according to a post on X written by Michael Torres, co- founder of genetic-Medicines company ReCode.

There is more to be done in terms of addressing the key aspects of this technology, according to the director of a gene therapist in Milan, Italy. “The delivery by nanoparticles is still in early stages in terms of tolerability.”

Gene-editing approaches carry the risk of ‘off target’ edits elsewhere in the genome. In animal studies, the Verve team found no off-target editing in mice, and no evidence of the changes in the PSCK9 gene becoming heritable in monkeys. Verve aims to select the best therapeutic dose from the trial next year and to launch a phase 2 trial in 2025.

The FDA mandates that the firm follow trial participants for 14 years. “This is a gene editing study — you are changing the genome forever. The importance of safety will be paramount, because there are currently safe and effective strategies available for lowering cholesterol.

The first patient was treated just six months ago, and researchers are still following all of the participants. The results were presented at the annual meeting of the American Heart Association.