UNISR - Universita Vita-Salute San Raffaele

Vita-Salute San Raffaele University (UniSR) combines its activities with the research conducted by San Raffaele Hospital (OSR), a leading research hospital performing cutting-edge translational research in human diseases and novel therapies. OSR has developed two EU-approved ATMPs -Strimvelis and Libmeldy- and is equipped with an infrastructure to develop cell and gene therapies from preclinical through clinical testing to market registration and clinical use. Research at UNISR covers several clinical areas (central and peripheral nervous systems pathologies, tumors, immune-mediated, infectious, genetic, cardiovascular, and metabolic diseases), thus offering a diversified range of use cases to be applied in redefining ATMPs’ regulatory framework. In this regard, an intramural research program funded by the Italian Association for Cancer Research (AIRC) has gathered several research and clinical units since 2019 to develop breakthrough ATMPs against liver metastases from colorectal and pancreatic cancers. The selected lead(s) are heading the clinical trial roadmap and are expected to open the way to their next-generation derivatives. UniSR has been actively involved in EU-wide interdisciplinary projects and multi-stakeholder consortia focused on healthcare and ATMP development and regulation (EUHA, T2EVOLVE, RESTORE).

Role within JOIN4ATMP

The UNISR team, led by Professor Alessandro Aiuti, is responsible for the implementation of WP4 and will transversely contribute to the accomplishment of different tasks within the overall project structure. In WP4, UNISR will chart new pathways for clinical trial and market approval, exploring innovative trial designs tailored for rare diseases, and working towards facilitating broader EU-wide patient access. Real-world data will be used to map hurdles in the commercial uptake of ATMPs, including pricing and reimbursement challenges. Specific activities will include: a definition of possible solutions to improve the interactions between ATMP developers and health technology assessment (HTA) bodies throughout ATMP development, assessment of the usability of real-world data for HTA evaluation and alternative pricing and reimbursement schemes. As part of the JOIN4ATMP consortium, UniSR will seek to achieve consensus on alternative evaluation and approval schemes for ATMPs in the EU, fostering a constructive regulatory-development dialogue with the regulatory bodies. UniSR, by recognizing the importance of public perception, will actively identify public attitudes towards ATMPs and strive to boost patients’ engagement towards ATMP development.

Main contact(s)

Aiuti, Alessandro

Deputy director clinical research; Join4ATMP Co-coordinator; WP4 Leader

Chiara Bonini

Group leader, Experimental hematology Unit; Full professor, UNISR; WP4 Co-leader

Fabio Ciceri

Head of Hematology and Bone Marrow Transplantation Unit, Full professor of Hematology UNISR; WP4 Co-leader

Francesco Marrafino

Join4ATMP Scientific Project Manager

Federica Prete

Head, EU Research Strategy and Policy Office, Research Development Area UNISR; Team Member

Francesca Severino

Senior Project Manager, EU Research Strategy and Policy Office, Research Development Area UNISR; Team Member

Maya Fedeli

Citizen Science & EU project communication officer, Research Policy Office Research Development Area UNISR; Team Member