Most gene therapies developed to date have gone after rare, inherited diseases. But a newly launched company is going after one of the most widespread ailments and causes of death on earth.
Cambridge, Massachusetts-based Verve Therapeutics announced its launch Tuesday for the purpose of using gene-editing technology to discover and develop therapies to permanently reduce the risk of coronary artery disease. The company is using technologies that it has gained access to through partnerships with single-letter gene-editing company Beam Therapeutics and Alphabet life sciences research company Verily Life Sciences, as well as licensing deals with Harvard University and the Broad Institute, which is run collaboratively by Harvard and MIT.
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In terms of financing, the company has raised a $58.5 million Series A round, led by GV and with participation from biopharma venture capital firms ARCH Venture Partners, F-Prime Capital and Biomatics Capital.
“Our genetic understanding of coronary artery disease, combined with increasing sophistication of gene editing technologies, have aligned to create a transformative moment in the treatment of this disease,” Verve CEO Sekar Kathiresan said in a statement. “Gene editing offers the possibility of introducing protective gene variants to adults at risk of the disease through a one-time therapy.”
The company said its therapeutics involve editing adult cells, which are not passed to offspring, focusing initially on patients with life-threatening artery disease, subsequently widening its focus to include larger patient populations. Technologies like CRISPR and base editors have the potential to change disease outcomes. Existing treatments like statins, the company said, face challenges like poor adherence, high cost and limited access in low- and middle-income countries, which according to World Health Organization figures account for 80 percent of coronary artery disease deaths.
But if the experience with other gene therapies provides precedent, cost could be an issue. The only gene therapy currently approved is Spark Therapeutics’ Luxturna (voretigene neparvovec), for biallelic RPE65 mutation-associated retinal dystrophy – with a list price of $850,000 for both eyes. Luxturna differs from gene-edited therapies in that it uses a viral vector to correct defective genes, as opposed to gene editing.
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Nevertheless, gene therapies are difficult to research, costly to put through clinical trials, rarely carry the possibility that patients will pay for them perennially and serve limited populations, wrote New York Law School professor Jacob Sherkow, who specializes in intellectual property and life sciences, in a December 2017 paper published in the Yale Journal of Biology and Medicine. Notwithstanding the fact that coronary artery disease represents a much larger patient population than genetic retinal disease or beta-thalassemia, Sherkow wrote that there is no reason to suspect CRISPR-based therapies won’t carry price tags comparable to their viral vector-based counterparts.
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