Verve banks $63M to move gene-editing heart attack treatment toward clinic

Verve Therapeutics launched last year with $58.5 million to create a one-and-done treatment to replace the array of drugs we use to treat heart disease. Now, it’s gearing up to move its first program into the clinic, and it’s snagged another $63 million to do it.

The company is working on new treatments that edit the human genome to permanently lower a person’s risk of coronary artery disease, which can ultimately lead to a heart attack. Since launch, it has pushed multiple types of gene therapies aiming at multiple targets through monkey studies, CEO Sekar Kathiresan, M.D., told Fierce Biotech.

The new capital, drawn from the likes of GV, Arch Venture Partners, F-Prime and Casdin Capital, will fund IND-enabling studies for Verve’s lead prospect and move it into a phase 1 study in the next couple of years. It’s still choosing between candidates for the program, with plans to pick one by the end of the year, Kathiresan said.

Beyond that, Cambridge, Massachusetts-based Verve is working on eight genes where loss-of-function mutations—mutations that “break” the gene—protect people from heart attacks. Its lead program targets one of the eight, and follow-on programs will do the same.

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Verve’s treatments would edit adults' genomes to mimic these protective mutations and lower “bad” LDL cholesterol throughout people's lives, reducing their risk of heart attacks.

With technology licensed from the Broad Institute and Harvard University, as well as partnerships with Beam Therapeutics and Verily, Verve is developing treatments using CRISPR Cas9 and Cas12, as well as base editing, to turn off cholesterol-raising genes in the liver.

“We’re not wedded to any specific technology, we just really want to find the best editing system that will maximize efficacy and minimize any safety issues,” Kathiresan said.

It will focus first on the most vulnerable patients before expanding into larger patient groups.

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“Imagine a patient who’s had a heart attack and still has sky-high cholesterol levels despite the standard-of-care therapies,” he said. “That’s the group we would focus on initially. Assuming we prove out efficacy and safety in that group, we can then expand to larger populations in the cardiovascular disease space.”