Gene therapies and RNA drugs, Italia now has a research network
With the 320 million allocated by the NRP, an infrastructure of centres capable of ensuring all the steps for innovative therapies has been created: 95 scientific projects and 30 patents have been launched and 8 start-ups have been launched
Italia is officially joining the race in the promising frontier of new RNA drugs and gene therapies, a market that will exceed 42 billion dollars by 2030, with annual growth now in double figures and the certainty that personalised and precision medicine will increasingly be the treatment of the coming years. And it does so thanks to an investment of 320 million euro financed by the NRP - 41% earmarked for the South - to create a National Centre capable of bringing together and enhancing all the energies already present in the country. In almost four years, a 'Hub & Spoke' network has thus grown into 10 thematic poles, involving 44 leading institutions including universities and research centres of excellence such as the Tiget in Milan or the Bambino Gesù in Rome, and 58 partners. More than 2,000 people, researchers in primis, were involved, with over 700 hires (65% women) and the involvement of 200 PhD students. The maxi investment has resulted in over 1,000 scientific articles, more than 30 patents and 95 applied research projects funded through cascade calls, 8 innovative start-ups focused on unmet medical needs, and 3 research projects ready for scale-up.
An ecosystem made up of enhanced technologies and infrastructures that has already demonstrated the ability to translate laboratory discoveries into trials and possible new therapies for challenging diseases such as neuroblastoma, leukaemia, and rare autoimmune diseases with an impact already on dozens of patients, as emerged during an event in Rome in recent days that took stock with stakeholders. 'We have succeeded in activating Car-T therapies, immune system cells modified to be redirected to a specific target, in solid tumours, neoplasms of the central nervous system, acute leukaemias, for which there were no commercially available products, and also in autoimmune diseases, achieving a real rearrangement of the immune system,' warns Franco Locatelli, director of the Onco-Haematology and Cell and Gene Therapy Department of the Bambino Gesù paediatric hospital in Rome. The network on gene therapies and RNA drugs coordinated by the National Centre based in Padua can now count on enhanced infrastructures and cutting-edge technologies thanks to the Pnrr funds: among these, the new 'RNA Factory' in Naples, which has just been completed and authorised for the production of mRNA for Phase I and II trials, stands out. In Rome, the enhanced Gene Therapy Laboratory, a 700 square metre facility, works in synergy with the adjacent Pharmaceutical Workshop. In Modena the Clinical Trial Centre is being set up, the first in Italia for Phase 1 integrated with the Glp laboratory, supported by the 'Stefano Ferrari' Regenerative Medicine Centre. In Padua, the Organoid Centre is active to test the efficacy of drugs on 3D 'mini-organs', faithfully simulating the human response and avoiding the use of animals. The network expands with the Facility for the analysis of circulating biomarkers in Siena, the ReCaS-Bari Data Centre combined with the new RNA_Biomix Flagship, and the Centre of Competence in Milan, an advanced services hub created to ensure that new drugs are safe, effective and ready for approval by Aifa and Ema. Another important upgraded infrastructure is the 'Stefano Verri' Laboratory for Cell and Gene Therapy, which, together with the newly built Research Tower, has enabled the Tettamanti Foundation Research Centre at the San Gerardo Hospital in Monza to be upgraded. These major works are joined by pioneering automation technologies, such as the CFBox robotic isolator, born from the alliance between PBL Spa and the Bambino Gesù Children's Hospital. Other strategic partnerships have been established with AstraZeneca, Chiesi, Dompé and Stevanato Group. The future now is to evolve this network into an 'Open Innovation Hub', facilitating the entry of new entities and companies that will be able to access the infrastructure and advanced services. Public funds from the Pnric call (2021-27) will be used to finance the start-up of new projects starting in May and for the following 30 months.
