Lexeo’s FA gene therapy candidate LX2006 uses a highly cardiotropic AAVrh.10 vector to deliver functional frataxin to the heart. Interim data show consistent, dose-responsive left ventricular mass index reductions and “striking” troponin declines, with patients having abnormal baseline LVMI returning to normal range and biopsy revealing meaningful increases in cardiac frataxin expression (from around 2% toward a 5% normal threshold) at low vector doses.

The PKP2-ACM program LX2020 delivers a functional plakophilin-2 gene to address inherited arrhythmogenic cardiomyopathy. Early cohorts demonstrated dose-dependent increases in plakophilin-2 expression, a 22% reduction in nonsustained ventricular tachycardia at the first time point, and up to a 60% reduction in two high-dose patients at nine months, contrasting natural history trends of rising VT risk.

Lexeo is finalizing alignment with regulators on an accelerated approval pathway, targeting an effect size greater than 10% LVMI improvement and an earlier assessment time point than 12 months. A parallel natural history study will support crossover enrollment and confirm there is no spontaneous LVMI improvement; full clinical updates are expected in Q4 with pivotal FA results due in early 2026.