Gene therapies, how Italy can become a global hub for research

A $141 million round, one of the highest ever raised in Italy in the biotech sector, coming just twelve months after a previous $61 million investment, confirms that 'made in Italy' gene therapy is now a global reference. Leading the operation on AAVantgarde Bio are three major international funds, flanked by foreign investors and, for the first time, also Italians. For Alberto Auricchio, director of Tigem and co-founder of the company, this is proof that 'when there is true innovation, supported by solid data, the market responds even in unfavourable economic times'.

The strength of the Neapolitan biotech lies in the two platforms capable of overcoming one of the historical limitations of gene therapy: transferring very large genes, hitherto incompatible with traditional vectors. 'With our system we use two vectors to build a single drug, doubling the transport capacity,' Auricchio explains. A technology that becomes a platform because it can be applied to numerous diseases and organs. The eleven patients treated with Usher 1B syndrome show an encouraging safety profile and early signs of efficacy, while parallel studies in China and the US have used the same logic to restore hearing to young people with hereditary deafness.

The new capital will now make it possible to speed up: phase 3 for Usher 1B is on its way and the first patient with Stargardt's disease will be treated between the end of 2025 and the beginning of 2026, after the OK from the FDA and the UK authorities. Plans also continue on other forms of retinitis pigmentosa linked to very large genes. Naples remains the clinical epicentre, thanks to the historic collaboration between Tigem and Professor Francesca Simonelli's ophthalmology clinic at the University of Campania Luigi Vanvitelli: 'Having integrated research and clinic makes it possible to bring discoveries rapidly to the patient. Italy must strengthen this supply chain,' Auricchio notes.

The issue of sustainability remains crucial. Ultra-rare diseases require high investments and risks that are difficult for industry to absorb; this is why Auricchio calls for more agile regulatory paths and 'platform' development, sharing studies and processes for several similar diseases. This is an approach that the Telethon Foundation has already brought to the table with Aifa, in the belief that dedicated models can also be adopted at European level.

It is precisely Telethon, Italy's driving force in research into rare genetic diseases for 35 years, that is relaunching its commitment this year with its Christmas campaign and 36th marathon on the RAI networks, from 13 to 21 December. Thousands of volunteers will be in the Italian squares on 20 and 21 December with the traditional Chocolate Hearts, also available online: a symbolic and concrete gesture to support scientific paths that require continuity, time and stable investments. The Telethon Foundation recalls how, since its inception, it has invested more than 740 million euros in research, contributing to the development of therapies now available in the European Union for Ada-Scid and metachromatic leukodystrophy, and accompanying the regulatory process of new treatments, including Wiskott-Aldrich syndrome and Usher 1B.