FDA publishes Information and Reviews of Biological Research Projects

Recommendation

7/8 May 2024

Annex 2 & Co. - GMP Compliance for Biopharmaceuticals - Live Online Training
Regulatory Requirements and Practical Implementation

The field of cell- and tissue-based products in particular has gained considerably in importance in recent years. The FDA is also paying increased attention to these topics. This is reflected on the one hand in the number of guidelines published in this area, but also in the fact that the FDA publishes information on FDA projects in the area of biological research.

For example, an overview of the activities on "Investigating the Effects of Cell-Materials Interactions on the Safety and Effectiveness of Cell-Based Products" was published in the CBER newsletter.

In this article, Dr Kyung E. Sung of the Office of Tissues and Advanced Therapies, Division of Cellular and Gene Therapies
Cellular and Tissue Therapy Branch provides a general overview of her research programme, which uses multidisciplinary approaches combining microphysiological systems and biomaterials. The aim is to investigate the effects of interactions between living cells and biomaterials used in the production and characterization of cell and tissue products. The primary objectives of this laboratory research are:

• the development of microphysiological 3D systems that reliably measure the functionality of multipotent stroma cells (MSCs) and induced pluripotent stem cells (iPSCs) under physiologically relevant conditions, and
• understanding the function and behaviour of cellular products in specific micro-environments to predict the safety and efficacy of manufactured CTPs

Another project in the field of biological research is the Evaluation of Therapeutic Approaches Based on Genome Editing and Stem Cell Technologies by Dr Zhaohui Ye, Office of Tissues and Advanced Therapies, Division of Cellular and Gene Therapies, Gene Transfer and Immunogenicity Branch, which aims to develop sensitive and unbiased methods for the detection of genetic mutations after editing with regard to the safety of genome editing technologies. In silico methods may not be sufficient to predict all potential off-target modifications, especially in newer technologies for which safety profiles do not yet exist. Therefore, this project uses a clonal, genome-wide sequencing strategy to detect genetic modifications in iPSC clones that have been successfully subjected to on-target genome editing. This strategy succeeds in overcoming genome-wide sequencing limitations in detection sensitivity, which is a significant advantage since unintended changes often occur randomly and with low frequency.