Pharmaceutical Compounding and Development of Advanced Therapy Medicinal Products: From Bench to Bedside

Dear Colleagues,

Advanced therapy medicinal products (ATMPs) are at the forefront of innovation and translational research and have transformed the clinical landscape. Based on gene, somatic-cell, and tissue-engineering therapy, these medicinal products have demonstrated potential to treat and cure patients for whom conventional medicine is not effective. Academic centers are major contributors to translational research and the development of ATMPs, such as therapies with viral vectors, chimeric antigen receptor (CAR) T cells, and tumor-infiltrating T cells (TILs). Despite their rapid development and increasing marketing authorization, challenges remain in terms of their development and clinical translation from bench to bedside, particularly within academia.

Regarding good manufacturing practices (GMP), the tech transfer of the production process may be challenging due to regulatory guidelines differing between competent authorities and the use of biological and non-compendial raw and starting materials. Furthermore, the advent of point-of-care or decentralized manufacturing practices for ATMPs requires harmonized practices across different manufacturing sites. Although a highly defined product is desired, batch standardization can be challenging since variations in the starting material (e.g., donor variability) and manufacturing process (e.g., duration, culture media) significantly impact product characteristics. Thus, particularly for point-of-care-manufactured ATMPs, demonstrating product comparability for products manufactured at different sites (potentially in different countries) is a challenge. Finally, pharmacokinetic–pharmacodynamic (PKPD) studies and elucidating the mechanism of action of ATMPs are complex, since these therapies are considered ‘living drugs’ and interact dynamically with their host.

The aim of this Special Issue is to highlight the development of ATMPs from a broad perspective, with a focus on pharmaceutical compounding and manufacturing processes. We invite authors to submit reviews and original articles on topics including the development of novel ATMPs; the optimization and validation of ATMP manufacturing processes; novel quality control tests; GMP technology transfer; point-of-care manufacturing; drug product characterization; comparability; hospital exemption; in vitro–in vivo correlation; PKPD studies; toxicology; formulation studies; cell metabolism; cell phenotype and subset analyses; regulatory aspects and commentaries.

Prof. Dr. Edwin Bremer
Dr. Bahez Gareb
Dr. Hugo Calderón
Guest Editors

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