Verve Therapeutics Adds $63M to Edit Heart Attack Risk Out of Genes
Summary
In a condition called familial hypercholesterolemia, a genetic defect makes it harder for these people to remove the bad form of cholesterol from their blood. A gene-editing therapy delivered via an engineered virus stays in the patient for months, which increases the risk of edits beyond the intended target, Kathiresan says. Kathiresan says in the past year the company has evaluated combinations of tools and targets to determine which offer the best shot at reducing cholesterol levels while posing the lowest risk. After that, he anticipates 18 months of preclinical research to support an application seeking FDA permission to begin tests of the gene-editing therapy in humans. Kathiresan is the scheduled keynote speaker on June 27 during the annual meeting of the International Society for Stem Cell Research, where he says he’ll discuss more details about Verve’s scientific findings.
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