Dissertation
Novel analytical approaches for the characterization of cell-based medicinal products and their formulation
Cell-based medicinal products (CBMPs), such as CAR T cells, are highly complex biopharmaceutical products offering various treatment options for serious illnesses such as cancer or autoimmune diseases.
- Author
- A. Rösch
- Date
- 10 June 2025
- Links
- Thesis in Leiden Repository
CBMPs consist of cells, which are derived from the patient or a donor and are subsequently modified and processed. Developers are facing various challenges regarding the production and quality control of CBMPs. Establishing stable formulations is of high importance to maintain product safety and potency. Cryopreservation, i.e., storage of cells at temperatures below -145 °C, is usually applied for long-term storage of CBMPs, which requires a constant supply of liquid nitrogen. Moreover, CBMPs are typically cryopreserved with dimethyl sulfoxide (DMSO) although it is damaging to cells at ambient temperature and can lead to side effects in patients.In this thesis, research is described investigating alternatives to the currently used cryopreservation of cells with dimethyl sulfoxide (DMSO). In addition, analytical techniques were evaluated to assess the quality of CBMPs. Furthermore, the impact of cell processing and handling on the viability of cells, i.e., survival, was evaluated to define appropriate process steps and holding times. The research described in this thesis supports the development of stable CBMP formulations and their analytical characterization, thus bringing novel cell therapy products closer to patients.