Signal CRISPR moves from rare blood disorders towards common cardiovascular risk

Summary

Despite funding constraints, the range of diseases addressed by CRISPR keeps widening. Casgevy, a CRISPR-based therapy approved by the US Food and Drug Administration in December 2023 for sickle cell disease and beta thalassaemia, is now offered at 50 treatment sites across North America, Europe and the Middle East. Combined with AI and advanced sequencing, CRISPR-Cas could extend gene editing into epigenomic expression. In a Cleveland Clinic study, CRISPR-Cas9 therapy cut LDL cholesterol by 50% on average, with effects seen within two weeks and lasting 60 days, and lowered triglycerides by 55%. The approach could offer an alternative to daily pills or monthly injections, and the study is set to expand in 2026.