Objective

Every rare disease deserves a treatment. To address this challenge, Healx developed a scalable and hypothesis-free drug discovery model. Integrating patient group expertise and multi-omic data with an AI-based platform enables novel ways of identifying treatment opportunities for rare diseases. This session will discuss: • How the Healx AI-enabled drug repurposing approach has been used to deliver a new drug repurposing candidate for Fragile X at a fraction of the cost and time of traditional drug discovery. • The Healx AI enable combination discovery technology and the progress made so far to identify drug combinations for Fragile X. • The advantages and challenges of developing repurposed drug combinations in general, from an IP and regulatory perspective.