FDA Creates New Pathway for Personalized Gene Therapy

“The FDA launched a new pathway to market for personalized gene therapy procedures in November.”

The FDA announced a regulatory pathway specifically designed for one-of-a-kind gene therapies tailored to individual patients. Earlier this year, a baby with a rare genetic disorder became the first person to receive a personalized CRISPR gene-editing treatment directly modifying faulty genes in his liver. By November, his mother reported he was starting to walk and hitting developmental milestones. The new FDA framework aims to make such individualized treatments more accessible as the technology matures.