VERV - Verve Therapeutics: An Update On Human CRISPR

2024-04-03 14:15:00 ET

Summary

• Verve is targeting patients with genetically-driven high cholesterol levels, and they are making a single A-to-G change in the gene for the PCSK9 protein.
• Verve announced their results yesterday, and it's a glass-half-full story. They say that they've dosed 13 patients so far, six of them at the 0.45 mg/kg level.
• The bad news is that the sixth patient showed elevated ALT liver enzyme levels along with severe thrombocytopenia after their dose.

I wrote here almost two years ago about Verve Therapeutics ( VERV ) and their plans to try human trials of a single-base editing CRISPR therapy. That post will give you plenty of background, but the short version is that Verve is targeting patients with genetically-driven high cholesterol levels, and they are making a single A-to-G change in the gene for the PCSK9 protein. That one is by now a well-known cardiovascular drug target, and there is abundant evidence that inactivating or depleting that protein can have a strong effect on cholesterol levels (and particularly levels of LDL). That evidence starts with a few rare human cases of natural knockout mutations, and goes on through several modes of drug therapy....

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