The FDA Opened the Door For Rare Disease Patients — Here’s What It Takes to Walk Through It
The Plausible Mechanism Framework is a breakthrough. Turning it into treatments that reach families will take five things the framework doesn't provide.
Steven Ringel
Steven Ringel is the founder and CEO of Nome Therapeutics, the operating system for personalized therapeutics. Nome uses AI to help rare disease families determine whether a custom therapy is scientifically viable for their specific genetic mutation, then coordinates the development process end to end.
Before founding Nome, Stevie led precision medicine business units at Tempus, GeneDx, and Sema4, and worked in healthcare strategy at Bain & Company. He also founded a 501(c)(3) to advance gene therapy for his own progressive inherited retinal condition, which he and his sister share. That dual perspective, as both a patient and an operator who has built precision medicine businesses, anchors how Nome approaches every family it serves.