19th Annual Meeting of the Safety Pharmacology Society: regulatory and safety perspectives for advanced therapy medicinal products (cellular and gene therapy products)

ABSTRACT

This report summarizes and discusses talks delivered at an educational course offered during the 2019 Annual Meeting of the Safety Pharmacology Society on advanced therapy medicinal products (ATMPs) and cell gene therapeutic products (CGTPs). ATMPs and CGTPs comprise gene and cell therapy medicinal products, tissue-engineered products, or the incorporation of one of these products into a medical device. Cited examples of ATMPs are autologous CD34⁺ cells encoding for the β^A-T87Q-globin gene, CAR (chimeric antigen receptor)-T cell immunotherapy medicines, genome editing products, and engineered heart muscle patches constructed from induced human pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) for remuscularization of the failing human heart. The nonclinical assessment of efficacy and safety of ATMPs for undertaking human clinical trials requires innovative, product-specific strategies. In order to succeed in gaining marketing approval for these novel medicines, sponsors should establish well-defined collaborative relationships with the appropriate regulatory authorities.

KEYWORDS:

• Advanced therapy medicinal products (ATMPs)
• cellular and gene therapy products (CGTPs)
• CAR-T-cells
• tissue-engineered heart repair
• regulatory development

Author contributions

I Cavero and H Holzgrefe conceived, designed, and prepared a preliminary draft of the manuscript. D Seimetz and WH Zimmerman carefully reviewed the original draft, correcting imprecisions and adding additional novel information during two manuscript revisions. D Koziel contributed a text on the FDA and the EMA differences in the regulation of ATMPs and CGMPs. The final version was thoroughly reviewed for language, journal style, and submission requirements and approved by all authors before submission.

Declaration of interest

WH Zimmerman is the founder, shareholder, and advisor of Repairon GmbH. The other authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.