Roots and studies in Turin, a career built around the world and now a new challenge with Tr1X, a US biotech company that studies and develops treatments for autoimmune and inflammatory diseases through the engineering of T cells. Maria Grazia Roncarolo is one of the world‘s leading experts in cell and gene therapy. Clinical trials of his therapy will start soon in the US and the goal is also to bring back to Italy the successes he is achieving abroad: «The pandemic has revolutionized the lives of many but has made us rediscover the importance of science. It was understood that scientific research applied to medicine creates innovation and provides the tools to deal with the unexpected».
Roncarolo graduated in Medicine at the University of Turin, then worked for many years in France, in California, then in Milan. Since 2014 she has moved permanently to the United States. But she remains very close to Turin: «During my specialty I moved to France also thanks to a scholarship obtained with La Stampa: I want to say this because it was the first funding I got in my life to do research. It allowed me to study in Lyon a new approach to treating genetic diseases with stem cells. Then I moved to Silicon Valley, where I worked in a cutting-edge biotech doing eight years of basic research. Meanwhile, I took a second specialty in immunology and allergology in Milan and then I returned to Italy, in 1997, because Don Luigi Maria Verzè and Susanna Agnelli called me to direct the San Raffaele Telethon institute for gene therapy. An experience that lasted ten years».
His path, he says, «has not been linear: many young researchers go abroad and never come back; instead I have had the good fortune and the opportunity to return and contribute to the creation of an institute that is of absolute excellence. Turin is my city, I remain strongly rooted here. It’s the only place in the world where I feel at home. I still have very strong ties with the Turin medical and scientific community”.
After having worked extensively on genetic engineering – in particular on regulatory cells capable of keeping our immune system in balance and understanding when it should activate to defend us against disease and when, instead, it should remain silent and not attack our body – now has developed a start-up based on an innovative idea: «When these cells are missing or don’t work, autoimmune and inflammatory diseases arise. The cells that my team discovered years ago and which we have called Tr1 are very important in maintaining this balance, not only for preventing these types of diseases but also for allergies. We tried to make a drug with these cells but it was very difficult to make. The winning idea of Tr1X is to take normal cells and make them look exactly like Tr1 through genetic engineering. They will then be administered to patients to reset the immune system and restore balance.”
The pre-clinical studies are completed, as well as the investment round: the next objective is to start the clinical study and the treatment of patients. «The first initial study will be carried out in California but I hope very soon to open the experimentation in Italy as well, which will give a new opportunity to people affected by these chronic diseases».