Clinical Development of Gene Therapies

What makes gene therapy so different from traditional drug development? How do we address safety when each patient only receives a single dose? And why are statisticians so essential in this process? In this episode of The Effective Statistician, I sit down with Oscar Segurado, an MD, PhD, an expert in gene and cell therapies, to answer these questions and more. Oscar draws on decades of experience in academia, med tech, and biopharma—including his work on the blockbuster drug Humira—to guide us through the complexities of clinical development in gene therapy. We discuss everything from early clinical trials to commercialization, uncovering the unique challenges of treating rare diseases like hemophilia A and spinal muscular atrophy. Whether you’re a statistician, data scientist, or just curious about the future of medicine, this episode offers valuable insights into the clinical development of gene therapies and the vital role statisticians play in advancing these groundbreaking treatments.