SOUTH SAN FRANCISCO - Tenaya Therapeutics (NASDAQ:TNYA) presented interim data from its RIDGE-1 Phase 1b/2 trial evaluating TN-401 gene therapy for arrhythmogenic right ventricular cardiomyopathy caused by PKP2 mutations.
All six patients receiving a single infusion of TN-401 showed reductions in premature ventricular contractions: 60% in Cohort 1 and 67% in Cohort 2.
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The treatment was well tolerated. Transient troponin or transaminase elevations were the most common adverse events. One Grade 3 liver enzyme elevation was attributed to a medication error.
The European Medicines Agency granted TN-401 PRIME designation. PKP2-associated ARVC affects about 70,000 people in the U.S.
