Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.

ABSTRACT: Advanced therapy medicinal products (ATMPs) are medicines for human use based on genes, cells or tissue engineering. After clear successes in adults, the nascent technology now sees increasing pediatric application. For many still untreatable disorders with pre- or perinatal onset, timely intervention is simply indispensable; thus, prenatal and pediatric applications of ATMPs hold great promise for curative treatments. Moreover, for most inherited disorders, early ATMP application may substantially improve efficiency, economy and accessibility compared with application in adults. Vindicating this notion, initial data for cell-based ATMPs show better cell yields, success rates and corrections of disease parameters for younger patients, in addition to reduced overall cell and vector requirem

SUBMITTER: Lederer CW 

PROVIDER: S-EPMC9031205 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature