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World J Immunol. Jul 24, 2021; 11(1): 1-10
Published online Jul 24, 2021. doi: 10.5411/wji.v11.i1.1
New era of personalized medicine: Advanced therapy medicinal products in Europe
Javier García-González, Sonia Marhuenda-Castillo, Sheila Romero-Carretero, Jesús Beltrán-García
Javier García-González, Strategy and Market Access, Pharmaceutical Industry, Madrid 28012, Spain
Sonia Marhuenda-Castillo, Sheila Romero-Carretero, Clinical Research, International CRO, Madrid 28003, Spain
Jesús Beltrán-García, Department of Physiology, Faculty of Medicine and Dentistry, University of Valencia, Valencia 46010, Spain
Jesús Beltrán-García, Center for Biomedical Research in Rare Diseases Network (CIBERER), Carlos III Health Institute, Valencia 46010, Spain
Jesús Beltrán-García, INCLIVA Institute of Sanitary Research, Valencia 46010, Spain
Author contributions: García-González J wrote the original draft; Marhuenda-Castillo S and Romero-Carretero S contributed to the original draft writing and prepared the summarized table; Beltrán-García J conceptualized, established, and directed the draft execution.
Conflict-of-interest statement: The authors have no conflicts of interest.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Jesús Beltrán-García, PhD, Academic Research, Department of Physiology, Faculty of Medicine and Dentistry, University of Valencia, Av. de Blasco Ibáñez, 15, Valencia 46010, Spain. jesus.beltran@ext.uv.es
Received: January 14, 2021
Peer-review started: January 14, 2021
First decision: February 15, 2021
Revised: February 24, 2021
Accepted: June 7, 2021
Article in press: June 7, 2021
Published online: July 24, 2021
Abstract

Advanced therapy medicinal products are human medical therapies based on genes, cells, or tissues, and due to their characteristics, they offer new innovative opportunities for the treatment of diseases and injuries, especially for diseases beyond the reach of traditional approaches. These therapies are at the forefront of innovation and have historically been very controversial, although in the last decade they have gained prominence while the number of new advanced therapies has increased every year. In this regard, despite the controversy they may generate, they are expected to dominate the market in the coming decades. Technologies based on advanced therapies are the present and future of medicine and bring us closer to the long-awaited precision medicine. Here we review the field as it stands today, with a focus on the molecular mechanisms that guided the different advanced therapies approved by the European Medicines Agency, their current status, and their legal approval.

Keywords: Advanced therapy, Advanced therapy medicinal products, Cell therapy, Gene therapy, Tissue therapy, Chimeric antigen receptor T cell

Core Tip: The advanced therapy medicinal products (ATMPs) have opened a new world of possibilities in the prevention and treatment of a large number of diseases that have not been effectively treated to date. ATMPs are based on the use of novel and sophisticated technologies adapted to each patient (personalized medicine), as well as on the discovery of novel and reliable biomarkers for the prediction and monitoring of clinical response. In this work we describe the latest advances in ATMPs approved by the European Medicines Agency, the molecular mechanisms that guided their medical benefits, how they work, the current status, and how they can change the medicine field forever, approaching precision personalized medicine.