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Cell and gene therapies offer transformative potential for treating diseases that cause disability and premature mortality, such as age-related macular degeneration (AMD) and various cancers. By transplanting genetically modified cells to restore or replace damaged tissue, these therapies open entirely new avenues for regenerative medicine. Yet two major challenges remain: ensuring public acceptance in the face of incomplete information and establishing robust quality assurance for cell therapy medicinal products (CTMPs).

Quality control is critical, as patient-derived cell isolates are inherently variable, and this variability increases with genetic modification. Although regulatory frameworks for CTMP characterization have been proposed, current quality control practices are still insufficient for routine implementation. This project therefore develops an innovative analytical technique capable of assessing CTMPs from minimal sample volumes, such as transfected retinal pigment epithelial (RPE) cells, within clinically relevant timeframes.

The approach is based on Raman spectroscopy (RS), a label-free optical technique that captures the molecular “fingerprint” of living cells through their light-scattering patterns. Raman spectroscopy has already proven effective in oncology for characterizing solid tumors and leukemia. Here, it will be adapted and refined for non-destructive, real-time quality assessment of living therapeutic cells.

The research follows a stepwise validation strategy: first applying the method to RPE cell lines, then to porcine RPE cells, and finally to human RPE cells. As proof of principle, 50 CTMPs composed of non-virally transfected human RPE cells will be analyzed to confirm that Raman-based evaluation reliably predicts therapeutic quality and efficacy, meeting the standards of clinical practice.

By ensuring both the efficiency of genetic modification and the viability of therapeutic cells, this technology aims to make personalized regenerative treatments safer, faster, and more accessible. Beyond technical innovation, the project also emphasizes public engagement to improve understanding and acceptance of cell and gene therapies, fostering trust in these groundbreaking medical approaches.