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 Calderon
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Pharmaceutics is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2900 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

• ATMPs
• manufacturing process
• pharmaceutical compounding
• product development
• product characterization
• process optimization
• tech transfer
• analytical procedures
• qualification
• validation
• mechanism of action
• CAR T cells
• TILs
• (viral) vectors
• cell banking
• regulatory aspects
• hospital exemption
• donor variability
• comparability
• point-of-care
• allogenic/autologous
• reconstitution/preparation for administration