Base editing makes its clinical trial debut

A new gene editing method called base editing, which could make gene editing more precise, is being clinically tested for the first time, Good Day BIO reports.

The company behind the testing is Verve Therapeutics, which is dosing the first patient in a trial of a drug that targets hereditary high cholesterol. Verve Therapeutics is a member of the Biotechnology Innovation Organization (BIO).

The treatment is performed in a way where the patient takes one pill “to make a single spelling change in liver DNA to permanently turn off a disease-causing gene,” thereby reducing “disease-driving low-density lipoprotein cholesterol (LDL-C),” Verve Therapeutics says.

Heterozygous familial hypercholesterolemia is an illness affecting 1 in 500 people. The disease raises LDL cholesterol levels and increases the risk of coronary artery disease.

What is base editing?

According to Nature, in the process of CRISPR-Cas9 genome editing, “the Cas9 enzyme breaks both strands of DNA at the site that is to be edited.”

DNA is often correctly reassembled by the cell. However, mistakes can occur. On the other hand, base editing only removes one strand of DNA, which minimizes potential errors, Nature explains.

In addition to giving hope to millions of people that live with this genetic disorder, the clinical trial for Verve Therapeutics’ VERVE-101 could also reveal whether base editing offers “safer and more controllable therapies for genetic diseases.”

“The dosing of the first human with such an investigational base editing medicine represents a significant achievement by our team and for the field of gene editing,” says CEO of Verve Therapeutics Sekar Kathiresan.

Regulatory obstacles for gene-edited drugs, particularly those relating to new manufacturing methods, are preventing the industry from reaching its full potential. Regulators also need to keep up with scientific advancements, according to experts on a panel discussing gene editing at the BIO International Convention in June.

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