S2E6 Frank Bennett: Turning RNA Targeting into Lifesaving Medicines

In this episode of Let’s Talk About That, Anirvan Ghosh speaks with Frank Bennett, Executive Vice President and Chief Scientific Officer at Ionis Pharmaceuticals, and a founding architect of antisense oligonucleotide (ASO) therapeutics—one of the technologies that launched modern genetic medicine.For more than three decades, Frank has worked to turn the idea of targeting RNA into real medicines. At the center of the discussion is the discovery and development of nusinersen (Spinraza), the first approved therapy for spinal muscular atrophy (SMA)—a devastating genetic disease that destroys motor neurons, leads to progressive paralysis, and in its most severe form often claims the lives of infants before age two. For decades, there were no disease-modifying treatments.Frank explains how understanding RNA splicing unlocked a strategy to restore SMN protein production—and how early clinical data revealed something extraordinary: children gaining motor function once thought permanently lost.The episode also explores other diseases that can be targeted by ASOs and why Frank believes we are just at the beginning of what RNA-targeted medicines can achieve.