FDA Announces Regulations for Custom Gene Therapies Amid Rare Disease Drug Debate

The FDA has released new rules for bespoke gene therapies, anticipating a surge in applications for personalized treatments, which may revolutionize rare disease medicine. Meanwhile, FDA Commissioner Marty Makary defended its rejection of certain rare disease drugs, highlighting ongoing regulatory tensions. The mixed signals to disease advocates underscore the complexity of balancing innovation with safety and approval standards, making this a crucial area to monitor in upcoming policy shifts.