Clinical TrialMay 15, 2026, 06:02 AM
Tenaya Therapeutics TN-401 gene therapy shows 64% mean PVC reduction
AI Summary
Tenaya Therapeutics presented positive interim data from its RIDGE-1 Phase 1b/2 clinical trial of TN-401 gene therapy for arrhythmogenic right ventricular cardiomyopathy (ARVC). All six patients treated with TN-401 showed meaningful reductions (mean 64%) in premature ventricular contractions (PVCs), and the therapy was well tolerated at both dose levels. Post-dose biopsies confirmed TN-401 activity in heart muscle cells, and the European Medicines Agency (EMA) granted TN-401 PRIority MEdicine (PRIME) designation.
Key Highlights
- All patients achieved a mean 64% decrease in daily premature ventricular contraction (PVC) count.
- TN-401 gene therapy was well tolerated at both 3E13 vg/kg and 6E13 vg/kg doses.
- Cohort 1 showed a mean PVC reduction of 60%, while Cohort 2 showed 67%.
- Post-dose biopsies provided evidence of TN-401 activity in heart muscle cells.
- European Medicines Agency (EMA) granted TN-401 PRIority MEdicine (PRIME) designation.
- Patient 2's NSVT burden dropped from 78 counts to zero at Week 52.
- Mean TN-401 DNA levels were 3.4 vg/dg (Cohort 1) and 3.8 vg/dg (Cohort 2).