Tenaya Therapeutics Doses First Patient in RIDGE™-1 Phase 1b Clinical Trial of TN-401 for the Treatment of PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy

TN-401 AAV9-based Gene Therapy Designed to Deliver Fully Functional PKP2 Gene with the Aim of Increasing Protein Levels to Address Underlying Disease

RIDGE-1 Currently Enrolling at Six Centers; Observational Natural History and Seroprevalence Study of PKP2-associated ARVC Adults Continues Enrollment at 20 Clinical Sites in the U.S., UK and Europe

Initial Clinical Data for RIDGE-1 Anticipated in 2025

Excerpt from the Press Release:

SOUTH SAN FRANCISCO, Calif., Nov. 25, 2024 (GLOBE NEWSWIRE) — Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that the first patient has been dosed with TN-401 gene therapy in the RIDGE-1 Phase 1b clinical trial at the University of California, San Francisco. Tenaya currently anticipates sharing initial data from the RIDGE-1 trial in 2025.

TN-401 is being developed for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC, also known as arrhythmogenic cardiomyopathy or ACM) caused by mutations in the plakophilin-2 (PKP2) gene. PKP2 gene mutations result in insufficient levels of critical proteins needed to maintain the structural integrity and cell-to-cell signaling of heart muscle cells. TN-401 gene replacement therapy is designed to deliver a functional PKP2 gene into heart muscle cells using an adeno associated virus serotype 9 (AAV9) capsid. In preclinical studies, the new, healthy PKP2 gene was successfully integrated into heart cells where it produced the missing protein to slow or even reverse the course of disease. Compared to untreated in vivo knock-out models, TN-401 normalized heart rhythms, reversed disease progression and extended survival following a single dose.

“People living with ARVC frequently experience dangerous arrhythmias and are at risk for developing heart failure, cardiac arrest and sudden death. To minimize their risk, ARVC patients live with significant activity restrictions, take chronic medications, and require interventions that together negatively impact their quality of life but don’t address the underlying problem of a defective gene,” said Vasanth Vedantham, M.D., Ph.D., Professor of Medicine, Cardiac Electrophysiologist, Director of Cardiovascular Genetics at the University of California, San Francisco and an investigator for the RIDGE-1 Phase 1b clinical trial. “PKP2 genetic mutations are the most common single gene cause of ARVC and unlike existing treatments for ARVC, TN-401 gene therapy seeks to directly address the underlying cause of disease by delivering a fully functional copy of PKP2 to the heart.”

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