National Institutes of Health, Wednesday Afternoon Lecture Series

Basic research, technological advances and clinical observations can converge to produce major therapeutic breakthroughs. Examples from lung cancer, spinal muscular atrophy and hemophilia illustrate how curiosity-driven science contributed to medicines including osimertinib, risdiplam and emicizumab. The lecture follows discoveries across the decades between initial biological insights and effective treatments, examining the persistence, unexpected findings and collaboration across disciplines that made these advances possible. It considers how biological, clinical and technological research connect in the development of new medicines.