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Crispr Heart Disease Biotech Raises $94M in New Funds

Editing DNA

(LaCasadeGoethe, Pixabay)

19 Jan. 2021. A company applying Crispr gene-editing technology to treat inherited heart disease is raising $94 million in its second venture funding round. Verve Therapeutics in Cambridge, Massachusetts is a two year-old enterprise spun-off from Massachusetts General Hospital and the Broad Institute, a genetic research center affiliated with Harvard University and MIT.

Verve Therapeutics develops treatments for inherited forms of heart disease, starting with heterozygous familial hypercholesterolemia, or HeFH, a genetic heart disorder resulting in dangerously high levels of cholesterol, leading to heart failure, heart attack, or stroke at an early age. Unlike most treatments that aim to reduce cholesterol or triglycerides already in the blood, Verve first identifies people with higher genetic risk of HeFH — estimated at about 1 million in the U.S. — and edits the genes with mutations responsible for causing high cholesterol levels.

Gene editing in this case, says the company, needs to be very precise and granular, addressing pairs of nucleic acids called base-pairs that make up DNA. Humans have about 3 billion pairs of nucleic acids — adenine (A) with thymine (T), and cytosine (C) with guanine (G) — with the sequence of these nucleic acid pairs comprising a person’s DNA or genetic code. When mutations or errors occur in the these nucleic acids, the errors are transcribed into faulty instructions provided to cells with RNA and the proteins that result from those instructions.

For gene editing, Verve Therapeutics uses the technique known as Crispr, short for clustered regularly interspaced short palindromic repeats. Crispr is a genome-editing process based on bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA. Verve licenses its Crispr process from Beam Therapeutics, also a spin-off company from the Broad Institute.

Lead product in preclinical tests

Verve’s lead product, code-named Verve-101, is now in preclinical development as an HeFH therapy. Verve-101 is designed to edit genes producing proteins in the liver, particularly the PCSK9 gene to regulate cholesterol in the blood stream. PCSK9 controls the number of receptors for low-density lipoproteins or LDLs, the so-called bad cholesterol, produced in the liver. Verve-101 is given as an infusion of messenger RNA, packaged in lipid or natural oil nanoscale particles, where it’s taken up by the liver.

As reported by Science & Enterprise in June 2020, Verve Therapeutics presented tests of its therapy now called Verve-101 in lab monkeys at a virtual meeting of International Society for Stem Cell Research. The results show monkeys receiving single infusions of the gene-edited treatment had lower levels of PCSK9 and ANGPTL3 proteins in their blood, as well as LDLs and triglycerides, regulated by ANGPTL3 proteins. In addition, the study team found no evidence of off-target edits.

“Over the last year, we made significant progress toward our bold vision of eradicating coronary heart disease,” says Sekar Kathiresan, Verve’s CEO and co-founder in a company statement, “most recently supported by compelling non-human primate data, which validate in vivo liver base editing as a once-and-done approach to lower LDL cholesterol and treat coronary heart disease.”

The company’s second venture funding round is raising $94 million led by existing investors Wellington Management Company in Boston and life science venture company Casdin Capital in New York. Joining the round are existing investors GV, formerly Google Ventures, and Biomatics. Also joining the financing are new investors Redmile Group, Janus Henderson Investors, Cormorant Asset Management, Rock Springs Capital, Novo Holdings A/S, Logos Capital, Surveyor Capital, a Citadel company, and RA Capital Management. Proceeds from the round are expected to advance Verve-101 toward a clinical trial planned for 2022.

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