Italian Gene Therapy Success
08/06/21
Groundbreaking gene therapy treatment, developed at the prestigious Bambino Gesu hospital in Rome, has successfully cured three children of a rare type of leukemia. The automated system that was used is part of Italy's CAR-T project.

Italy is at the forefront of developing gene therapy in the world. Only six of the ten cell and gene therapies initially approved are still on the market, and of those three are from Italy, which makes the Italian contribution to the existing approved therapies significant.

Dr Luigi Naldini, the director of the San Raffaele Telethon Institute for Gene Therapy, explains Italy’s enormous success in the field of cell and gene therapies.

"We certainly have a strong clinical capacity, especially in the early stages of clinical testing, and a strong connection in some institutions between clinicians and scientists. Many biomedical institutions in Italy can fully support clinical translation themselves. In other places, that can be a challenge; a lot of the time, research institutes perform substantial preclinical development, but their location is far removed from the clinical sites. The culture in Italy is to bring the research and clinical work together. That is an engine for translation.

Another factor in Italy’s success is the role of public funding, particularly from charities like the Telethon Foundation, which supports research on genetic diseases – the main platform for cell and gene therapies. In Italy, Telethon plays a unique role because global funding has not always been reliable or constant. In time, Telethon has therefore emerged as a prime source of high-quality funding and has really driven cell and gene therapy research.

Finally, prior to the current hype surrounding cell and gene therapy, it was for a long time met with great scepticism and very little interest from industry. During that time, Europe managed to build a strong research community driven by EU-funded consortia, with charities and academia supporting the translational work. That has been the only surviving strategy for the field. When the first clinical results came, they came from that preassembled community of scientists and clinicians, supported by public, EU or charity funding. That was instrumental to the delivery of the first products."