Gene editing treatment for inherited high cholesterol: early human trial

An in vivo base-editing therapy lowered PCSK9 and LDL cholesterol in adults with heterozygous familial hypercholesterolemia after a single infusion.

This ongoing phase 1 dose-escalation trial reported interim results in 6 adults given single intravenous YOLT-101, finding no grade 3 or higher adverse events and dose-dependent durable reductions in PCSK9 and LDL-C, with 74.4% and 52.3% reductions at 24 weeks in the 0.6 mg/kg cohort (n=3).

These are interim phase 1 results from only 6 participants, with the largest efficacy signal coming from a 3-person highest-dose cohort and short-term follow-up focused mainly on safety and biomarker change.