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Verve Therapeutics said Tuesday its one-time treatment that uses the “base editing” form of CRISPR to remove a cholesterol-associated gene in monkeys has demonstrated durable and significant reductions in LDL “bad” cholesterol for six months.

Based on these new data, Verve intends to advance the treatment, called VERVE-101, into clinical development for people born with heterozygous familial hypercholesterolemia, or HeFH, a genetic heart disease that causes lifelong, high LDL levels


“These individuals have sky-high LDL starting at birth and they have a tremendously high exposure to cumulative LDL, which is their primary problem,” said Sekar Kathiresan, Verve’s co-founder and CEO. “Verve-101 will look to be a definitive therapy for them — a one-and-done permanent lowering of cholesterol, or a genetic medicine for a genetic disease.”

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