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One and done?
The interim Phase I trial data was only from 35 people, but results look good so far.
An experimental gene-editing therapy that aims to lower bad cholesterol for the long-term after a single infusion is off to a positive start in an early clinical trial.
Researchers running a Phase I safety trial for the drug, dubbed VERVE-102, published interim results from just 35 patients this week in the New England Journal of Medicine. Though the numbers are small and the analysis is preliminary, VERVE-102 appeared safe, with no serious adverse events reported from the treatment, even at the largest doses. The most significant finding was a temporary, mild increase of a liver enzyme that suggested minor injury in the liver, where the drug works.
