Crispr Heart Disease Start-Up Raises New $63M

(Bryan Brandenburg, Wikimedia Commons)

11 June 2020. A one year-old company developing a one-time gene-editing treatment for coronary artery disease is adding $63 million to its first venture funding round. Verve Therapeutics, in Cambridge, Massachusetts, a spin-off enterprise from research labs at Massachusetts General Hospital in Boston, raised $58.5 million when it first began.

Verve Therapeutics aims to provide a single therapy to fix coronary artery disease in patients, who now treat it as a chronic condition requiring continuous medications. Coronary artery disease, also known as atherosclerosis or hardening of the arteries, 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 current treatments that aim to reduce cholesterol already in the blood, Verve plans to edit the genes with mutations responsible for causing high cholesterol. The company’s technology is based on research by its scientific co-founder Sekar Kathiresan at Mass. General, a Harvard Medical School teaching hospital, 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 now full-time CEO at Verve.

The company also licenses Crispr gene-editing technology from the Broad Institute, a genetics research center affiliated with Harvard and MIT. Broad Institute is a pioneer in Crispr, short for clustered regularly interspaced short palindromic repeats, and advances in the technique using simpler, more precise, and efficient editing enzymes.

Verve’s treatments for coronary artery disease will address mutations in genes associated with cholesterol, such as the PCSK9 gene that produces a protein to help regulate cholesterol in the blood stream. PCSK9 controls the number of receptors for low-density lipoproteins or LDLs, produced in the liver. The more LDLs, also known as “bad” cholesterol, in the liver, the more high-density lipoproteins, or “good” cholesterol, are removed from blood, increasing the risk for coronary artery disease.

The company is first developing a Crispr-based therapy for people with coronary artery disease, but not responding to conventional drugs to lower cholesterol, and thus in most danger from the disease. From there, Verve expects to expand the treatments to larger segments of the population at higher risk of coronary artery disease. In addition, the company emphasizes that its treatments address genes in the liver considered somatic or adult genes, and are not passed on to future generations.

The new $63 million financing is the second part of the Verve’s first venture round, which began at the company’s launch in May 2019 with 58.5 million, as reported by Science & Enterprise. Leading this part of the round is current investor GV — formerly Google Ventures — with current investors ARCH Venture Partners, F-Prime Capital, and Biomatics Capital, joined by new investors Wellington Management and Casdin Capital.

The funding, says Kathiresan in a company statement, “will position us to rapidly advance our lead therapeutic candidate through IND-enabling studies and expand our pipeline of development programs. People with heart disease are in urgent need of new therapeutic options, and Verve’s research to date further underscores the transformative potential of gene editing as a therapeutic approach for these patients.” (IND stands for investigational new drug application, clearance from Food and Drug Administration to begin clinical trials.)

More from Science & Enterprise:

• Biotechs Apply Crispr Gene-Editing for Hemophilia
• Patient Receives First Crispr Genetic Eye Treatment
• Trial Underway Testing Crispr-Based Antibiotic
• Broad Inst, Biotech Partner on Immune-Safe Crispr
• First Blood Disorder Crispr Treatments Reported

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