William H. Lewis

Rewriting the Rules: Scaling Innovation for Rare Diseases | Global Conference 2026

Becky Quick, Nicola Blackwood, David Fajgenbaum, Michael Hund, Neil Kumar, William H. Lewis

Panelists including David, Will Osborn, and Michael discussed how AI-driven repurposing of generic drugs and patient-led "venture cure" models are accelerating the discovery of treatments for the over 300 million people globally affected by rare diseases. The dialogue highlighted transformative regulatory shifts, such as the UK's newborn sequencing trials and FDA-authorized umbrella CRISPR studies, which aim to bypass traditional trial inefficiencies while addressing barriers like pricing models that stifle global drug launches. Ultimately, the event concluded that converging data infrastructure with proactive patient advocacy is essential to de-risk development and deliver rapid therapeutic interventions for currently untreatable conditions.