San Raffaele-Telethon, so Italia becomes the laboratory of genomic medicine

The first European collaboration of the 'Danaher Beacon' programme was born in Milan and carries with it an ambitious promise: to accelerate the development of gene and cell therapies that are reshaping precision medicine. In fact, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) joins the international network of centres of excellence chosen by the Danaher biotech group, through its subsidiary Cytiva, to test technologies and processes designed to make the production of advanced therapies faster, more scalable and sustainable. The project will focus on a number of crucial technological nodes: the optimisation of lentiviral vectors, tools used to transfer genetic material into cells, the evolution of gene editing techniques, and the automation of blood stem cell manipulation procedures. The shared goal is to make cell and gene therapies more reproducible, scalable and accessible, overcoming one of the main obstacles that currently slows down their delivery to patients.

For the Telethon Foundation, joining the programme represents scientific recognition but also a strategic investment in the future of the sector. "The value of this collaboration is very high because it means that our institute is recognised for its excellence and is projected onto the frontier of innovation," explains Ilaria Villa, the Foundation's director general. "Here we will test new ways of technological development around advanced personalised therapies on a global scale. The central issue remains the so-called scalability: many gene therapies have demonstrated clinical efficacy, but their production remains complex, expensive and difficult to replicate on a large scale. 'Today, the biotechnology industry struggles to advance precisely because these processes are so complex,' notes Villa. 'Collaborations such as this one help to investigate routes that are scientifically robust but also acceleratable and more economically sustainable.

The Beacon model was created precisely with this objective in mind: to transform a number of research centres of excellence into technological experimentation laboratories where industry and academia can test new solutions. "We become a beacon," says Villa. "We do not just replicate industrial processes, but we push knowledge forward by experimenting with tools and technologies together with a partner who recognises the quality of our research. The programme will have a multi-year horizon and will also focus on automating processes that are still manual today in the handling of haematopoietic stem cells. Improved yields, safety and replicability of gene editing systems could result in faster development times and reduced costs, two decisive factors for patient access to therapies. According to Villa, the initiative also has symbolic value for the European research system. "Europe is still struggling to be central in biotechnology," he notes. "The fact that a major group chose an Italia institute for this investment, however, shows that there is global expertise.

There remains the economic challenge of advanced therapies, which are often associated with very high costs. On this front too, Telethon claims a special role. 'We are a non-profit organisation that works with industrial standards but without the logic of profit,' Villa emphasises. 'This allows us to be a laboratory of innovation where new processes can be tested and then become standards for the entire sector. For the foundation, which over the years has brought to market a number of gene therapies developed in its own laboratories, the ultimate goal remains unchanged: to transform research into concrete cures. "Behind all this work," Villa concludes, "is the mission to make therapies truly accessible to patients who need them. In the medium term, the results will be measured through very concrete indicators: reduction in the loss of biological material during the production phases, increased yields of viral vectors, and greater automation of laboratory procedures. Seemingly technical improvements that, in the gene therapy chain, can however make the difference between a promising scientific discovery and a treatment that is actually available to patients. A step that can bring genomic medicine closer to large-scale clinical practice in Europe.