US regulators laid out new guidelines for approving custom-made treatments for individual patients, a move that could bring the promise of gene editing to more people with devastating diseases.
Food and Drug Administration Commissioner Marty Makary and Vinay Prasad, a top agency official, detailed their criteria for clearing personalized treatments for diseases so rare they may only affect a handful of people in an article published on Wednesday. Among other things, they want to see that a drug targets the underlying cause of the condition, like a genetic mutation, and that the patient improved after treatment.