Major CMC Commercialization Gaps for Gene Therapy Products Manufactured for a Diverse Pediatric Subpopulation(s)

摘要

The development of cell and gene therapy products to address serious and life-threatening conditions that affect the pediatric population is growing exponentially. Although much is known about the Chemistry and Manufacturing Control (CMC) considerations for the adult population, the challenges of manufacturing complex products for younger populations are not yet fully understood. To manufacture cell and gene therapy products for pediatric subpopulations, special considerations need to be taken into account and there is a good reason to conclude that the relative risk due to drugs increases inversely by age.