Imagine if your unique genetic code could be the blueprint for your own personalized medicine. You're about to discover how scientists are now crafting gene therapies specifically for just one patient at a time.
The FDA recently announced a new regulatory pathway for "N-of-1" gene therapies, treatments designed for a single patient with an ultra-rare genetic disease. This marks a significant shift towards highly individualized medicine.
Historically, drug development targets large patient populations to ensure commercial viability and broad impact.