CRISPR Therapeutics advances top dose of gene editing treatment for high cholesterol

CRISPR Therapeutics presented one-year data from an early-stage trial of its gene editing treatment for high cholesterol, showing inconsistent durability. Despite the mixed results, the company plans to advance the highest dose tested. The findings were shared at the European Society of Cardiology meeting in Munich.

The one-year findings, presented at the European Society of Cardiology meeting in Munich, showed that the gene editing therapy produced inconsistent durability in lowering cholesterol among participants. This variability means some patients maintained reductions while others did not, leaving questions about how reliably the treatment holds up over time in an early-stage setting.

Despite the uneven results, CRISPR Therapeutics has decided to advance the highest dose tested in the study. This move suggests the company sees enough promise in the therapy's effect at that level to justify further investigation. The decision reflects a typical drug development path, where early data guide dose selection for larger trials, even when outcomes are not uniformly positive.