EP 24 Samuel M. Young, Jr., PhD

In this episode of Rare Awareness Radio, host Richard Juknavorian sits down with Dr. Sam Young, Director of the Gene Therapy Center at the University of North Carolina at Chapel Hill. From his early days as a Princeton football player turned molecular biology student, to training under Nobel laureates and leading pioneering labs across the U.S. and Europe, Sam has built a career at the cutting edge of gene therapy. His story is one of resilience, curiosity, and a willingness to tackle “generational questions” that few others dare to pursue. Together, we explore: The evolution of gene therapy from early setbacks to transformative breakthroughs. Why packaging limits in AAV vectors matter—and how new adenoviral approaches may unlock treatments for large, complex genes. The urgent challenges in treating rare neurological conditions like Ataxia-Telangiectasia and CACNA1A-related disorders. The importance of integration between scientists, clinicians, and patient advocates to bring new therapies forward. Dr. Young reminds us that the real heroes are always the patients and families, and that the responsibility of science is to create the world not as it is, but as it should be. 🎧 Tune in for an inspiring conversation about science, persistence, and hope for the rare disease community. For additional information on Sam Young, please visit - https://www.med.unc.edu/pediatrics/people/samuel-m-young-jr-phd/ To learn more about the CACNA1A Foundation, please visit - https://www.cacna1a.org/