Milken Institute 4d ago

Watch all 2026 Global Conference session replays: https://milkeninstitute.org/events/global-conference-2026/program?utm_medium=social&utm_source=youtube&utm_campaign=gc26_post-event-content&utm_content=video-description Historically, the rare disease community has met persistent barriers when forced to follow clinical and regulatory pathways designed for common diseases. Such barriers are reinforced by a fractured funding environment and a challenging investment case. With more than 10,000 rare diseases identified, applying one-disease-at-a-time models is prohibitively slow, costly, and ultimately unsustainable. Breakthroughs in genetic therapies and AI are now enabling a fundamental reimagining of how rare diseases are diagnosed, studied, and treated. Panelists will examine how innovators are rewriting the rules—from AI-powered diagnostics and drug-repurposing screens to platform-based therapies and batch regulatory-approval models that address multiple diseases at once. Together, these approaches will facilitate more rapid, cost-efficient, and scalable progress for the global rare disease community. Becky Quick Anchor, "Squawk Box," CNBC; Co-founder, CNBC Cures Nicola Blackwood Chair, Health Data Research Service David Fajgenbaum Co-founder and President, Every Cure Michael Hund CEO, EB Research Partnership Neil Kumar Founder and CEO, BridgeBio Pharma William H. Lewis Chair and CEO, Insmed