Using AI to De-Risk Rare Disease Drug Development

Healx, which developed an AI platform to repurpose drugs for rare diseases, is among the first generation of companies built on an AI drug discovery engine. The company’s lead experimental therapy, which the U.S. Food and Drug Administration granted Fast Track designation to in October 2024, is in mid-stage development for neurofibromatosis type 1, a rare genetic condition that causes tumors to grow on nerves. A deal at the end of 2024 shows expanded uses for its platform technology as Sanofi entered into an agreement to see if the company could find new indications for a late-stage asset that it had discontinued. We spoke to Tim Guilliams, co-founder and CEO of Healx, about the origin of the company, its lead asset in development for neurofibromatosis type 1, and how he views the company’s progress to date.