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 requirements, illustrating that early application may resolve key obstacles to the widespread application of ATMPs for inherited disorders. Here, we provide a selective review of the latest ATMP developments for prenatal, perinatal and pediatric use, with special emphasis on its comparison with ATMPs for adults. Taken together, we provide a perspective on the enormous potential and key framework parameters of clinical prenatal and pediatric ATMP application.

Consorzio per Valutazioni Biologiche e Farmacologiche 70122 Bari Italy

Department of Pharmacology and Toxicology Faculty of Pharmacy in Hradec Králové Charles University 50005 Hradec Králové Czech Republic

Empa Swiss Federal Laboratories for Materials Science and Technology 9014 St Gallen Switzerland

Institute of Translational Pharmacology IFT National Research Council 90146 Palermo Italy

Pediatric Clinic Department of Clinical Surgical Diagnostic and Pediatric Sciences Fondazione IRCCS Policlinico San Matteo University of Pavia 27100 Pavia Italy

The Molecular Genetics Thalassemia Department The Cyprus Institute of Neurology and Genetics Nicosia 2371 Cyprus

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