05/15/2026
This morning at , interim data that demonstrate consistent reductions in arrhythmias for adults with PKP2-associated ARVC from the RIDGETM-1 Phase 1b/2 trial of investigational TN-401 were presented. The data also showed clear evidence from heart biopsies of TN-401 transduction and expression, and a promising safety profile at both doses.
John Giudicessi, M.D., Ph.D., a genetic cardiologist who oversees the clinical cardiac gene therapy section of the Windland Smith Rice Comprehensive Sudden Death Program at Mayo Clinic and a principal investigator for the RIDGE-1 clinical trial, said the following about the results:
“The most prominent and troubling clinical feature of PKP2-related ARVC is potentially life-threatening heart rhythm problems or ventricular arrhythmias, so the substantial reduction in ventricular arrhythmia events observed across the first six adults to receive TN-401 gene therapy is exciting. While additional follow-up is needed to appropriately assess TN-401’s full potential, the emerging safety profile and early evidence of activity observed are promising. A gene therapy approach offers the potential of transformative treatment by correcting the genetic defect underlying PKP2-related ARVC, whereas existing care – which includes the use of implantable defibrillators, heart failure or anti-arrhythmic medicines, or ablation of parts of the heart muscle – does not address the underlying cause of disease or slow its progression.”
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