Safe, fast and accessible gene and cell therapies
Treating age-related macular degeneration and preventing blindness remains a complex task today. Thanks to exceptional funding from the Gelbert Foundation, a new project aims to introduce gene and cell therapy into clinical routine to enable better care. It brings together the experimental ophthalmology (OpEx) research group at the UNIGE Faculty of Medicine and the HUG, led by Prof. Gabriele Thumann and Dr Martina Kropp, and a research group from the Leibniz Institute for Photonic Technologies specialising in spectroscopy and imaging (Leibniz-IPHT), led by Prof. Iwan Schie. If their results are conclusive, they could be extended to other domains in personalized medicine.
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Gene therapies that are using cells involve genetic modification of therapeutic cells before transplanting them to restore the functions of diseased organs. Although their potential is immense, in many areas such as cancers or diseases causing loss of sight, the complexity of production and quality control has so far limited their use. The challenge lies in the fact that each cell behaves differently, just as each human being is unique and, consequently, the different batches of drugs made up of living cells are also different. This is unlike industrially manufactured pills, which are all identical. Nevertheless, we must also be able to guarantee a therapeutic effect for the cells. Thanks to the support of the Gelbert Foundation, the OpEx research group in Geneva and the Leibniz-IPHT in Germany will be able to develop new quality control technologies in order for these advanced therapies to become part of clinical routine.
Quality control of gene and cell therapies has evolved from laboratory methods. Not very suitable for the clinical context, it remains laborious and leads to the destruction of many of the therapeutic cells. “Our idea is to set up a control system at the very heart of the clinic, at the patient's bedside, to analyse the exact cells that will be administered to the patient just before administration,’ emphasises Gabriele Thumann. “We need to be fast and efficient, and above all, this control must not destroy the cellular drug, while guaranteeing the reliability and quality of the modified cells.”
Characterising without destroying
To do this, the team is working on the development of an innovative technology based on Raman spectroscopy. ‘This method makes it possible to observe and characterise the molecular composition and external structure of single cells without destroying them,’ explains Martina Kropp. ‘It is already used in fixed tissue in laboratory analyses but has never been tried and tested in personalized medicine and living cells.’
Initially, the cells viability will be evaluated using a cell product derived from non-viral cellular gene therapy developed by the OpEx group to treat age-related macular degeneration. ‘With a fast and reliable analysis of living cells, our approach aims to make these therapies accessible to everyone and to reduce public scepticism towards them,’ the scientists say. ’Its therapeutic potential is truly exceptional.’
7 Apr 2025