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

期刊

PHARMACEUTICS
卷 14, 期 4, 页码 -

出版社

MDPI
DOI: 10.3390/pharmaceutics14040793

关键词

gene therapy; somatic cell therapy; tissue-engineered medicinal product; CAR T cell; CAR NK cell; hematopoietic stem and progenitor cell; mesenchymal stromal cell

资金

  1. European Union [777554]
  2. European Regional Development Fund
  3. Republic of Cyprus through the Research and Innovation Foundation [EXCELLENCE/1216/0092, EXCELLENCE/0421/0086, 2014-2021]

向作者/读者索取更多资源

Advanced therapy medicinal products show great promise in pediatric applications, with potential to improve treatment outcomes for untreatable disorders and inherited diseases. Early application in children may provide better results and reduce overall requirements.
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.

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