Gene therapy for heart disease

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Tenaya Therapeutics Presents Interim Data from RIDGE™ Natural History and Seroprevalence Study of Adults with PKP2-associated ARVC at Heart Rhythm 2025
Globenewswire· 2025-04-24 18:07
Core Insights - Tenaya Therapeutics announced interim data from the RIDGE study, the largest natural history study of adults with arrhythmogenic right ventricular cardiomyopathy (ARVC) due to PKP2 gene mutations, highlighting the high burden of arrhythmias and severe disease progression despite standard treatments [1][3][4] Group 1: Study Overview - The RIDGE study has enrolled over 175 patients across approximately 20 clinical sites in the U.S., UK, France, Germany, Italy, and Sweden, focusing on clinical characteristics and medical history of PKP2-associated ARVC patients [3][4] - Interim data from 144 adults with PKP2-associated ARVC were analyzed as of February 2025 [3] Group 2: Key Findings - 83% of participants with Holter data had a premature ventricular contraction (PVC) count of 500 PVCs/day or greater, indicating a higher risk of life-threatening ventricular arrhythmias [4] - 49% of participants with available Holter monitoring data had a history of ventricular tachycardia [4] - 60% of patients with MRI data showed disease progression, as indicated by measures of right and left ventricular function and heart tissue health [4] - Electrocardiogram results indicated that a majority of patients had T-wave inversions, suggesting ventricular strain or myocardial injury [4][5] Group 3: Treatment Insights - Current treatment approaches, including medications and surgical interventions, have proven insufficient in addressing the high burden of arrhythmias and disease progression in PKP2-associated ARVC patients [3][4] - Tenaya is developing TN-401 gene therapy, which aims to deliver a functional PKP2 gene to heart muscle cells using an adeno-associated virus serotype 9 (AAV9) [2][10] Group 4: Future Directions - Enrollment is ongoing for Tenaya's Phase 1b clinical trial, RIDGE-1, to assess the safety, tolerability, and activity of a one-time dose of TN-401, with initial safety and biopsy data expected in the second half of 2025 [6][11] - The RIDGE study is expected to serve as a control arm for assessing the efficacy of TN-401 gene therapy in PKP2-associated ARVC [7]