Base editing, a method to make gene editing even more precise, is being tested in the clinic for the first time, with Verve Therapeutics dosing the first patient in a clinical trial of a drug targeting hereditary high cholesterol.
The treatment: The patient takes just 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),” says BIO member Verve Therapeutics.
The disease: Heterozygous familial hypercholesterolemia is a common genetic disorder affecting 1 in 500, which increases LDL cholesterol levels and is associated with a higher risk of coronary artery disease.
The technology: “In CRISPR–Cas9 genome editing, the Cas9 enzyme breaks both strands of DNA at the site that is to be edited,” according to Nature. The cell usually stitches the DNA back together correctly, but errors can happen. However, “base editing” only cuts one strand, reducing possible errors.
It’s a potential game-changer: The clinical trial for Verve Therapeutics’ VERVE-101 drug not only provides hope for millions worldwide with this genetic disorder—it could show whether base editing offers “safer and more controllable therapies for genetic diseases.”
What they’re saying: “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 Verve Therapeutics CEO Sekar Kathiresan.
The context: Regulatory challenges for gene-edited drugs, especially regarding new manufacturing techniques, are “keeping the game-changing sector from realizing its full potential,” Bio.News reports—and regulators need to keep up with the science, said experts on a gene editing panel at the BIO International Convention in June.
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