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A Cambridge, Massachusetts biotech chasing the goal of curing heart disease just announced the first treatment it plans to test in people.
Verve Therapeutics’ lead experimental therapy, dubbed VERVE-101, aims to slash bad cholesterol levels and effectively cure patients with a serious genetic heart disease by editing a tiny piece of their DNA. Verve CEO and cofounder Dr. Sekar Kathiresan told Business Insider that’s the first part of his three-step strategy in transforming how heart disease is treated.
“Ultimately, this medicine, if effective and safe, will be a one-and-done for heart attack,” Kathiresan said.
Verve is set to be one of the leading — if not the first — biotechs to begin testing cutting-edge gene-editing technologies in humans to treat heart disease. Other biotechs have started testing CRISPR-based treatments for the genetic blood disorders sickle cell disease and beta-thalassemia.
Kathiresan, a prominent cardiologist, founded Verve while working as a researcher at the Broad Institute and Massachusetts General Hospital. After a lifetime of studying the heart and its related genetics, Kathiresan resigned from his positions to lead Verve, which was incubated by GV, formerly known as Google Ventures, from 2016 to 2018.
Over the last two years, Verve has raised more than $120 million in seed and Series A rounds led by GV. The company now has 53 employees, with plans to grow to about 80 workers in the next two years, Kathiresan said. Most of the hiring will be for clinical research and manufacturing roles, he added.
New data shows sustained cholesterol drop in monkeys, further building enthusiasm for gene-editing research
The biotech’s first experimental treatment was built using tools from the biotech Beam Therapeutics called base editing technology. It’s a more precise way to edit genes, allowing scientists to change a single letter of genetic code.
In this case, VERVE-101 aims to change an A to a G in the code of a gene involved in regulating cholesterol levels, called PCSK9. That single infusion should permanently lower someone’s bad cholesterol levels.
Verve’s first study will enroll patients with heterozygous familial hypercholesterolemia, a genetic heart disease that affects about 1.6 million people in the US and Europe, Kathiresan estimated.
These patients have abnormally high levels of LDL, or bad cholesterol from birth, which often result in overworked arteries. The ultimate result is young patients, often in their 30s or 40s, having heart attacks, strokes, and sometimes even dying.
The technology isn’t ready to be tested in people. Verve will focus in 2021 on completing the necessary toxicology studies and lab work to set the stage for human testing to begin in 2022, Kathiresan said.
Even as the technology remains a ways away from potentially reaching large numbers of patients, excitement is clearly building in the space.
Verve’s partner Beam, for instance, went public in February 2020 at a valuation of more than $1 billion. Less than a year later, Beam’s stock has increased more than 350%, with the biotech now worth well over $5 billion, despite having no treatments ready for testing in people.
Verve’s latest monkey data, released Tuesday morning, should further bolster enthusiasm. Initial data, presented in June 2020, showed the technology could effectively block genes to dramatically lower cholesterol levels in monkeys.
“This could be the cure for heart disease,” Dr. Michael Davidson, a cardiovascular surgeon not involved in the research, told The New York Times.
In Tuesday’s update, Verve showed that cholesterol drop of 61% was sustained six months out and counting, based on results from four monkeys. These are the first monkey studies to show a prolonged benefit from the base-editing technology, Kathiresan said.
The long-term goal is to develop a one-and-done treatment that can prevent heart attacks
The company’s long-term vision is to tackle cardiovascular disease, the leading cause of death in the US. The initial work in patients with this genetic heart condition is the first of three steps, Kathiresan said.
“Step two and step three are even more exciting,” he added.
The second phase will expand research to anybody with atherosclerotic heart disease, a group that Kathiresan estimated at about 24 million people in the US and Europe. That’s a condition in which plaque builds up in arteries, narrowing them and potentially blocking blood flow or causing them to break.
The third step is the most ambitious: testing a gene-editing program as a preventive therapy for anyone at risk of heart attack. This would require a sizable amount of safety data in humans, Kathiresan acknowledged — data that doesn’t yet exist at any level for base-editing treatments.
In chasing that ambitious vision, Verve will soon need to raise more capital. Kathiresan declined to say if Verve plans take advantage of a red-hot IPO market and go public this year.
“We’re in the process of thinking through what those additional raises are going to be like,” he said. “That’s probably all I want to say right now on that topic.”