Verve Therapeutics said Tuesday it used a next-generation form of CRISPR to edit the genes of someone with high cholesterol. The first-ever move sent VERV stock flying.
It's the first time base editing has been used in a human being. Base editing doesn't involve cutting both strands of DNA's double helix. Experts say that's an improvement over CRISPR, which does require a cut in order to correct genes. Cutting the double helix runs the risk the strands will sew themselves up incorrectly. That could lead to off-target side effects.
Verve licensed the technology from Beam Therapeutics. Beam is also partnered with Pfizer to test out its base-editing approach. But Verve was the first to put the technology into a human. The company tested the gene-editing technology in a person with inherited high LDL cholesterol.
The drug "is a first-in-class gene-editing medicine that we have designed to make a single spelling change in liver DNA to permanently turn off a disease-causing gene," Chief Executive Sekar Kathiresan said in a written statement. "The dosing of the first human with such a base-editing medicine represents a spectacular achievement by our team and for the field of gene editing."
On today's stock market, VERV stock rose soared 8.4% to 22.20. Beam stock also jumped 4.8% to 52.88.
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VERV Stock: Rivaling Amgen, Regeneron
Success in high cholesterol could send VERV stock flying. Studies estimate the condition affects one in 250 people both in the U.S. and globally. People with the condition — known formally as heterozygous familial hypercholesterolemia — are more likely to suffer heart attacks and heart disease at a young age.
There are treatments on the market from biotech companies Amgen and Regeneron Pharmaceuticals. These shots called Repatha and Praluent block proteins known as PCSK9, which play a role in regulating blood cholesterol. Verve says just 20% of people achieve desired LDL cholesterol levels with chronic treatment.
In comparison, Verve hopes to use base editing to turn off the gene responsible for making the PCSK9 proteins. This will change the way cardiovascular disease is treated by lowering LDL cholesterol "as low as possible for as long as possible after a single treatment," Verve Chief Medical and Scientific Officer Andrew Bellinger said in a written statement.
Verve hopes to enroll 40 patients with heterozygous familial hypercholesterolemia in a larger study. Verve plans to test several doses. In later portions of the study, some patients who received lower doses could get a higher second dose, Verve said.
Interim data are expected in 2023. The results could impact both VERV stock and shares of Beam.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
