Gene Therapy Breakthroughs in Genetic Cardiomyopathies
In this episode of the Heart Failure Beat, Dr. Eric Adler of UC San Diego Health joins the show to discuss his landmark gene therapy trial for Danon disease, recently published in the New England Journal of Medicine. Dr. Adler recounts the powerful bedside-to-bench-to-bedside story that led to the development of a groundbreaking treatment for this severe, genetic cardiomyopathy. He explains the mechanics and evolution of gene therapy, its promise in treating monogenic heart failure syndromes, and the early but encouraging clinical outcomes—like dramatic reductions in troponins and heart mass. The episode also explores regulatory and safety challenges, delivery limitations, and the critical role of patient advocacy. Whether you’re a heart failure specialist or a translational researcher, this conversation offers compelling insights into the future of precision therapies in cardiology.
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Check out Episode Highlights
References, Links and Disclosures
Dr. Adler Disclosures: Chief Medical Officer and Head of Research at Lexeo Therapeutics. Shareholder and Royalties at Lexeo Therapeutics and Rocket Pharmaceuticals. Founder and Shareholder of Papillion Therapeutics.
Greenberg, B., Taylor, M., Adler, E., Colan, S., Ricks, D., Yarabe, P., Battiprolu, P., Shah, G., Patel, K., Coggins, M., Carou-Keenan, S., Schwartz, J. D., & Rossano, J. W. (2025). Phase 1 study of AAV9.LAMP2B gene therapy in danon disease. New England Journal of Medicine, 392(10), 972–983. https://doi.org/10.1056/nejmoa2412392
Argiro, A., Bui, Q., Hong, K. N., Ammirati, E., Olivotto, I., & Adler, E. (2024). Applications of gene therapy in cardiomyopathies. JACC: Heart Failure, 12(2), 248–260. https://doi.org/10.1016/j.jchf.2023.09.015
