S2E2 Gilmore O'Neill: Rethinking Medicine in the Age of Gene Editing

In this episode of Let’s Talk About That, Anirvan Ghosh is joined by Gilmore O’Neill, President and CEO of Editas Medicine, for an in-depth conversation at the frontier of genetic medicine, neuroscience, and biotech leadership.Gilmore traces his journey from clinical neurology at Mass General to a career in biopharma driven by frustration with the lack of effective treatments for devastating neurological diseases. He reflects on formative years at Biogen, where first-principles thinking—deep attention to human biology, PK/PD, and disease pathology—helped enable breakthroughs such as Spinraza for spinal muscular atrophy and transformative therapies in multiple sclerosis.The discussion then turns to the rise of genetic medicines, from antisense oligonucleotides to gene therapy and, ultimately, gene editing. Gilmore offers a clear, accessible explanation of how CRISPR-based in vivo gene editing differs from traditional gene delivery—and why Editas is focused on single-dose, durable therapies that can be delivered with minimal healthcare infrastructure.A major focus is Editas’s lead program targeting LDL cholesterol, where in vivo gene editing has shown unprecedented reductions in LDL levels in preclinical models. Gilmore outlines the scientific rationale, the public health implications of treating common diseases with genetic medicines, and what it will take to translate this technology safely into humans.A thoughtful conversation on innovation, rigor, and reimagining medicine beyond chronic treatment.