UCGSci Studio: How a Drug Customized for One Boy Sparked a Revolution in Medicine

In late February 2026, the U.S. Food and Drug Administration (FDA) released new guidelines that could transform the way gene therapies are developed for patients with the rarest diseases. UCGSci Studio presents an overview of the new regulatory framework and the story of KJ Muldoon, the first patient in the world to receive a CRISPR therapy designed exclusively for him. What happens when a medicine needs to be made for only one patient in the world? In late February 2026, the U.S. Food and Drug Administration (FDA) published draft guidance—the first systematic answer to that question,...