New era of personalized medicine: Advanced therapy medicinal products in Europe

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.