Our CEO, Neil Kumar joined a panel discussion at the 2026 Milken Global Conference titled Rewriting the Rules: Scaling Innovation in Rare Disease. During the discussion, Neil argues that failure to address the needs of patients with rare genetic disease represents a profound market failure. He sights that despite scientists now identifying three new causal genetic variants per day, most companies with traditional business models remain fixated on large markets while countless rare conditions remain unaddressed.
From the Milken Global institute’s website:
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.