In its first tough test, CRISPR base editing slashes cholesterol levels in monkeys

A form of CRISPR widely expected to be safer and possibly more effective that the original has aced its first substantive test. When CRISPR “base editing” was used to knock out two cholesterol-associated genes in monkeys, the animals’ blood levels of heart-disease-causing LDL (“bad”) cholesterol and triglycerides plunged as much as 60% and 65%, respectively, Sekar Kathiresan, co-founder and CEO of Verve Therapeutics, announced on Saturday at the (virtual) meeting of the International Society for Stem Cell Research.

The results, from Verve’s experiments in 14 cynomolgus monkeys (a.k.a. crab-eating macaques), are the first published data showing successful CRISPR base editing in a non-human primate; there have been similar successes in mice. They are therefore good news not only for Verve, which was founded last year to develop CRISPR-based cures for cardiovascular disease, but also for Beam Therapeutics, a two-year-old company
developing CRISPR base editors for a long list of diseases. Verve licensed Beam’s “adenine base editor” for its experiment.

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