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Start-Up to Use Crispr for Heart Disease, Gains $59M

Editing DNA

(LaCasadeGoethe, Pixabay)

7 May 2019. A new biotechnology enterprise plans to apply genetic analysis and the gene-editing technology Crispr to reduce the risk of coronary artery disease. The company, Verve Therapeutics in Cambridge, Massachusetts, starts up today staked to $58.5 million in its first venture funding round.

Verve Therapeutics is a spin-off business from Massachusetts General Hospital and the Broad Institute — a genetic research center affiliated with Harvard University and MIT — in Boston. Verve plans to develop treatments for coronary artery disease, also known as atherosclerosis or hardening of the arteries. Coronary artery disease often results from a build-up of cholesterol plaques on the arteries feeding the heart, and is a major risk factor for heart attacks and other cardiac diseases. Unlike most treatments that aim to reduce cholesterol already in the blood, Verve plans to identify people with higher genetic risk of coronary artery disease and edit the genes with mutations responsible for causing high cholesterol.

The company is licensing discoveries from the lab of its scientific co-founder Sekar Kathiresan at Mass. General on genetic links to families with a history of heart disease. Among the discoveries in Kathiresan’s lab are genetic mutations that protect against heart disease. Kathiresan is also co-director of the Broad Institute’s program on medical and population genetics. He is stepping down from his academic positions in July to become Verve’s full-time CEO.

Verve Therapeutics is licensing as well advances in the gene editing technology Crispr from the Broad Institute for certain cardiovascular disease targets. Crispr, short for clustered regularly interspaced short palindromic repeats, is a technique for editing genomes based on bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA. The actual editing of genomes with Crispr uses enzymes directed by RNA to the genes in question. Verve is gaining access to Broad Institute’s work with the enzymes Cas9 and Cas12a, also known as Cpf1 that Broad says is simpler and more precise than Cas9.

In addition, Verve is partnering with another start-up, Beam Therapeutics, also in Cambridge, gaining access to that company’s technology for using Crispr to repair base-pair errors in genes. As reported by Science & Enterprise at the company’s founding in May 2018, Beam Therapeutics is also licensing gene-editing technologies from Broad Institute. Verve and Beam plan to apply Beam’s base-pair repair techniques to cardiovascular diseases, and equally divide profits or losses from products from this collaboration.

Verve plans to develop treatments initially for adults already diagnosed with coronary artery disease, but not responding to cholesterol-lowering drugs. The treatments will target the liver, with tissue containing the target genes — somatic or adult genes, not passed on to future generations — and accessible for editing to repair the responsible mutations, thus reducing cholesterol levels and the risk for heart disease. The company says preclinical studies with animals show this approach can reduce cholesterol levels and lower disease risks.

“Coronary artery disease is a true pandemic and a growing health crisis,” says Kathiresan in a company statement. “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 Therapeutics is receiving $58.5 million in its first venture funding round. GV — formerly Google Ventures — is leading the round, with participation by venture investors ARCH Venture Partners, F-Prime Capital, and Biomatics Capital.

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